Children's Medical Center Corporation

United States of America

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IPC Class
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca 150
A61K 39/00 - Medicinal preparations containing antigens or antibodies 121
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides 107
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A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy 93
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1.

BOSTON CHILDREN'S HOSPITAL

      
Application Number 1885171
Status Registered
Filing Date 2025-05-05
Registration Date 2025-05-05
Owner The Children's Medical Center Corporation (USA)
NICE Classes  ?
  • 41 - Education, entertainment, sporting and cultural services
  • 44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
  • 45 - Legal and security services; personal services for individuals.

Goods & Services

Educational services, namely, conducting seminars, lectures, classroom and clinical instruction in the fields of medicine and health. Medical services, namely, inpatient and outpatient, surgical and non-surgical, medical and healthcare treatment services; hospital services; physician services; medical clinic services; providing medical diagnostic and medical treatment services; providing general health, wellness, and medical information to the public; providing information in the field of healthcare and wellness via a website links; emergency medical response services; mental health services; behavioral health services; physical therapy; providing breastfeeding information and lactation consulting services; providing neonatal medical treatment services; medical radiology and imaging services; nutrition and lifestyle wellness counseling; speech and hearing therapy; charitable services, namely, providing medical services to underdeveloped countries; dentistry services; occupational therapy services. Providing health care case management services, namely, coordinating social and personal support services for children; providing personal support services for pediatric patients and their families; providing personal support services for patients dealing with pediatric health and wellness concerns; providing emotional counseling and emotional support services for pediatric patients and their families.

2.

CELLS WITH INCREASED IMMUNO-REGULATORY PROPERTIES AND METHODS FOR THEIR USE AND MANUFACTURE

      
Application Number 18977377
Status Pending
Filing Date 2024-12-11
First Publication Date 2025-10-30
Owner The Children's Medical Center Corporation (USA)
Inventor Fiorina, Paolo

Abstract

The present invention is directed to compositions and methods to increase the expression of PD-L1 and/or IDO-1 in a population of cells, the modulated cells expressing increased PD-L1 and/or IDO-1, and methods related to the immunosuppressive effects obtained by cells expressing increased PD-L1 and/or IDO-1.

IPC Classes  ?

  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61K 38/44 - Oxidoreductases (1)
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61K 40/10 - Cellular immunotherapy characterised by the cell type used
  • A61K 40/22 - Immunosuppressive or immunotolerising
  • A61K 40/41 - Vertebrate antigens
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
  • A61P 37/00 - Drugs for immunological or allergic disorders
  • C07K 14/555 - Interferons [IFN]
  • C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
  • C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
  • C12N 9/02 - Oxidoreductases (1.), e.g. luciferase

3.

METHODS AND COMPOSITIONS RELATING TO ANTI-PD1 ANTIBODY REAGENTS

      
Application Number 19264986
Status Pending
Filing Date 2025-07-10
First Publication Date 2025-10-30
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Alt, Frederick W.
  • Tian, Ming
  • Cheng, Hwei-Ling

Abstract

Described herein are novel anti-PD1 antibody reagents (e.g., antibodies, antigen-binding fragments thereof, and/or chimeric antigen receptors). Also decribed herein antibody-drug conjugates or kits comprising the disclosed antibody reagents, as well as methods of treating cancer by administereing the disclosed antibody reagents.

IPC Classes  ?

  • C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
  • A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies

4.

ANTI-NUCLEOPHOSMIN 1 ANTIBODY AND ANTIBODY CONJUGATE COMBINATION THERAPIES

      
Application Number 18860597
Status Pending
Filing Date 2023-04-27
First Publication Date 2025-10-23
Owner
  • The Children's Medical Center Corporation (USA)
  • Cambridge Enterprise Limited (United Kingdom)
Inventor
  • Tzelepis, Konstantinos
  • Flynn, Ryan A.
  • George, Benson M.

Abstract

Provided herein are antibodies specific for nucleophosmin 1 (NPM1), which are capable of binding to nucleophosmin 1 located on the surface of cells, as well as antibody-drug conjugates (ADCs) comprising such antibodies. Also provided herein are methods of treating cancers in which NPM1 is expressed on the surface of cancer cells, by administering to a subject an NPM1-binding antibody or antibody conjugate described herein and a chemotherapeutic drug.

IPC Classes  ?

  • A61K 51/10 - Antibodies or immunoglobulinsFragments thereof
  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
  • A61P 35/02 - Antineoplastic agents specific for leukemia
  • C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans

5.

USE OF TOLL-LIKE RECEPTOR 2 (TLR-2) AGONIST FOR MODULATING HUMAN IMMUNE RESPONSE

      
Application Number 18972689
Status Pending
Filing Date 2024-12-06
First Publication Date 2025-10-23
Owner
  • Children's Medical Center Corporation (USA)
  • Dana-Farber Cancer Institute, Inc. (USA)
Inventor
  • Dowling, David J.
  • Levy, Ofer
  • Borriello, Francesco
  • Scott, David A.
  • Brightman, Spencer E.

Abstract

Provided herein are Toll-like receptor 2 (TLR2) agonists for use in enhancing human immune response and/or as adjuvants in vaccines. The TLR2 agonists include thiophenes, imidazoles, or phenyl-containing compounds, which may be compounds of Formulae (I), (II), (III), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. The compounds described herein are used as enhancers of an immune response (e.g., innate and/or adaptive immune response), and are useful in treating and/or preventing a disease, as adjuvants in a vaccine for the disease, (e.g., proliferative disease, inflammatory disease, autoimmune disease, infectious disease, or chronic disease). Also provided in the present disclosure are pharmaceutical compositions, kits, methods, and uses including or using a compound described herein. Provided herein are Toll-like receptor 2 (TLR2) agonists for use in enhancing human immune response and/or as adjuvants in vaccines. The TLR2 agonists include thiophenes, imidazoles, or phenyl-containing compounds, which may be compounds of Formulae (I), (II), (III), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, prodrugs, and compositions thereof. The compounds described herein are used as enhancers of an immune response (e.g., innate and/or adaptive immune response), and are useful in treating and/or preventing a disease, as adjuvants in a vaccine for the disease, (e.g., proliferative disease, inflammatory disease, autoimmune disease, infectious disease, or chronic disease). Also provided in the present disclosure are pharmaceutical compositions, kits, methods, and uses including or using a compound described herein.

IPC Classes  ?

  • A61K 39/39 - Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
  • A61K 31/18 - Sulfonamides
  • A61K 31/381 - Heterocyclic compounds having sulfur as a ring hetero atom having five-membered rings
  • A61K 31/4025 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil not condensed and containing further heterocyclic rings, e.g. cromakalim
  • A61K 31/4164 - 1,3-Diazoles
  • A61K 31/438 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring being spiro-condensed with carbocyclic or heterocyclic ring systems
  • A61K 31/4535 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a heterocyclic ring having sulfur as a ring hetero atom, e.g. pizotifen
  • A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
  • A61K 31/739 - Lipopolysaccharides
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 37/04 - Immunostimulants

6.

PEDIATRIC EMERGENCY KIT AND METHODS OF USE THEREOF

      
Application Number 19037541
Status Pending
Filing Date 2025-01-27
First Publication Date 2025-10-23
Owner The Children’s Medical Center Corporation (USA)
Inventor Ngo, Peter

Abstract

Disclosed is a pediatric emergency kit for esophageal tamponade procedures in pediatric patients and methods of use thereof. The pediatric emergency kit includes at least one esophageal balloon catheter for the tamponade of blood flow, and an inflation hand pump equipped with a pressure gauge for inflating a balloon on the balloon catheter. The pediatric emergency kit can be configured to include multiple compartments, optionally, with a first container/compartment containing at least the esophageal balloon catheter and a second container/compartment containing at least the inflation handpump, and optionally a second esophageal balloon catheter. Optionally, both the first and second containers/compartments contain a syringe, a 3-way luer lock stopcock, and/or a guidewire. Also provided are instructions and/or methods of using the kit for correct placement and inflation of the esophageal balloon to effectively manage arterio-esophageal fistulas.

IPC Classes  ?

  • A61F 17/00 - First-aid kits
  • G06K 19/06 - Record carriers for use with machines and with at least a part designed to carry digital markings characterised by the kind of the digital marking, e.g. shape, nature, code

7.

ZINC FINGER CCCH-TYPE CONTAINING 14 (ZC3H14) MUTANTS AND METHODS OF USE

      
Application Number 18291414
Status Pending
Filing Date 2022-07-21
First Publication Date 2025-10-23
Owner
  • The Children's Medical Center Corporation (USA)
  • Dana-Farber Cancer Institute, Inc. (USA)
Inventor
  • Zon, Leonard I.
  • Insco, Megan L.

Abstract

The disclosure provides polypeptides, polynucleotides, compositions, kits and methods useful for modulating RNA molecules including degrading disease-causing RNA(s) or stabilizing RNA(s) to treat diseases.

IPC Classes  ?

  • C12N 9/22 - Ribonucleases
  • A61K 38/00 - Medicinal preparations containing peptides
  • A61P 35/00 - Antineoplastic agents
  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals

8.

SIMPLE NATURAL LIGHT MIMICS

      
Application Number US2025025422
Publication Number 2025/222155
Status In Force
Filing Date 2025-04-18
Publication Date 2025-10-23
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Do, Michael, Tri H.
  • Morquette, Charles, Philippe

Abstract

An artificial illumination light source to mimic natural light' s engagement of an organism's visual pigments using a small set of wavelengths is provided. In the case in which the organism is a mouse or human, mimicking daylight' s engagement of the visual pigments in the eye may be accomplished with as few as two peak wavelengths, including a first "short" wavelength and a second "medium" wavelength. The artificial illumination source may include two discrete narrow-band light sources to generate the two peak wavelengths. The peak wavelengths may be generated with unequal intensities to further mimic natural light, including daylight.

IPC Classes  ?

  • A61B 5/06 - Devices, other than using radiation, for detecting or locating foreign bodies
  • A61B 3/06 - Subjective types, i.e. testing apparatus requiring the active assistance of the patient for testing light sensitivity, e.g. adaptationSubjective types, i.e. testing apparatus requiring the active assistance of the patient for testing colour vision
  • H05B 47/155 - Coordinated control of two or more light sources

9.

SURFACE CALR CHEMICAL INDUCERS

      
Application Number 18874961
Status Pending
Filing Date 2023-06-07
First Publication Date 2025-10-16
Owner
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
  • PRESIDENT AND FELLOWS OF HARVARD COLLEGE (USA)
Inventor
  • Zon, Leonard
  • Rodrigues, Cecilia Pessoa

Abstract

ROS signal upregulates surface CALR and promotes macrophage-HSC interactions, safeguarding the development of stem cells that are stressed or damaged. Described herein are methods of controlling hematopoiesis, e.g., reducing hematopoiesis and/or improving the quality control mechanisms of hematopoiesis, relating to the use or administration of at least one CALR agonist.

IPC Classes  ?

  • A61K 31/706 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
  • A61K 31/53 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
  • A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca

10.

SOFT BRACES TO PREVENT INJURY TO A JOINT OR BODY SEGMENT

      
Application Number 19003564
Status Pending
Filing Date 2024-12-27
First Publication Date 2025-10-16
Owner
  • PRESIDENT AND FELLOWS OF HARVARD COLLEGE (USA)
  • CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Kiapour, Ata
  • Popov, Dmitry
  • Granberry, Rachael M.
  • Nathanson, Danielle L.
  • Eckert-Erdeim, Asa M.
  • Walsh, Conor James

Abstract

A soft brace to prevent injury to one or more target joints or body segments is disclosed. The soft brace includes one or more tensile elements configured to limit motion of one or more target joints based on placement of the one or more tensile elements relative to the one or more target joints such that the placement and tension of each of the one or more tensile elements provides resistance against motion of the one or more target joints; one or more soft tissue anchors positioned on a body around the one or more target joints, the one or more anchors being configured to anchor one or more of the one or more tensile elements to the body to provide force distribution relative to the one or more target joints; and wherein at least one of the one or more tensile elements is routed in parallel with the approximate center of rotation of at least one of the one or more target joints.

IPC Classes  ?

  • A61F 5/01 - Orthopaedic devices, e.g. long-term immobilising or pressure directing devices for treating broken or deformed bones such as splints, casts or braces
  • A63B 71/08 - Body-protectors for players or sportsmen

11.

COMPOSITIONS AND METHODS FOR TREATING SELENOPROTEIN-ASSOCIATED DISEASES

      
Application Number US2025024380
Publication Number 2025/217586
Status In Force
Filing Date 2025-04-11
Publication Date 2025-10-16
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Beggs, Alan H.
  • Moghadaszadeh, Behzad

Abstract

The present invention features compositions and methods for treating selenoprotein-associated diseases. Non-limiting examples of selenoproteins include selenoprotein N (SELENON).

IPC Classes  ?

  • C12N 15/09 - Recombinant DNA-technology
  • C12N 7/00 - Viruses, e.g. bacteriophagesCompositions thereofPreparation or purification thereof
  • C12N 7/01 - Viruses, e.g. bacteriophages, modified by introduction of foreign genetic material
  • C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • A61K 35/76 - VirusesSubviral particlesBacteriophages
  • A61P 21/00 - Drugs for disorders of the muscular or neuromuscular system
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans

12.

COMPOSITIONS COMPRISING MODIFIED PHOSPHOLIPIDS AND USES THEREOF

      
Application Number 18716692
Status Pending
Filing Date 2022-12-06
First Publication Date 2025-10-16
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Kohane, Daniel S.
  • Li, Yang

Abstract

Provided herein are compositions comprising compounds of Formula (I), and salts, co-crystals, tautomers, stereoisomers, solvates, hydrates, polymorphs, and isotopically enriched derivatives thereof; for example, in the form of a particle (e.g., liposome). Also provided are methods, uses, pharmaceutical compositions, and kits involving the compounds and/or compositions described herein, for methods for delivering an agent described herein (e.g., therapeutic agent, diagnostic agent), or for treating and/or preventing a disease in a subject, and methods of synthesizing these compositions.

IPC Classes  ?

  • A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
  • A61K 31/137 - Arylalkylamines, e.g. amphetamine, epinephrine, salbutamol, ephedrine
  • A61K 31/445 - Non-condensed piperidines, e.g. piperocaine
  • A61K 31/529 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim forming part of bridged ring systems
  • A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
  • A61K 31/704 - Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin, digitoxin
  • A61K 47/24 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids

13.

MUTANT AND MISLOCALIZED CELL SURFACE NUCLEOPHOSMIN 1 AS A DIAGNOSTIC AND THERAPEUTIC TARGET OF HUMAN DISEASE

      
Application Number 18860335
Status Pending
Filing Date 2023-04-27
First Publication Date 2025-10-09
Owner
  • The Children's Medical Center Corporation (USA)
  • Cambridge Enterprise Limited (United Kingdom)
Inventor
  • Flynn, Ryan A.
  • George, Benson M.
  • Tzelepis, Konstantinos

Abstract

Provided herein are antibodies specific for nucleophosmin 1 (NPM1), which are capable of binding to wild-type and/or mutant nucleophosmin 1 located on the surface of cells. These antibodies, as well as antibody conjugates comprising these antibodies, are useful for the detection and treatment of cancers in which NPM1 is expressed on the surface of cancer cells.

IPC Classes  ?

  • C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
  • A61P 35/00 - Antineoplastic agents
  • C12N 9/24 - Hydrolases (3.) acting on glycosyl compounds (3.2)
  • G01N 33/574 - ImmunoassayBiospecific binding assayMaterials therefor for cancer

14.

COMPOSITIONS AND METHODS FOR TARGETED PROTEIN DEGRADATION

      
Application Number 18863427
Status Pending
Filing Date 2023-05-05
First Publication Date 2025-10-09
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor Orkin, Stuart H.

Abstract

Described herein are compositions and methods for targeted erythroid-specific protein degradation. The compositions described comprise a binding domain for a erythroid protein of interest, e.g., BCL11A, and a binding domain for a erythroid enriched E3 ubiquitin ligase. Compositions and methods described can be used to promote fetal hemoglobin and treat hemoglobinopathy disorders.

IPC Classes  ?

  • C07K 16/40 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against enzymes
  • C12N 15/62 - DNA sequences coding for fusion proteins
  • C12N 15/864 - Parvoviral vectors

15.

INJECTABLE HYDROGEL BASED ON LIPOSOME SELF-ASSEMBLY FOR SUSTAINED RELEASE OF SMALL HYDROPHILIC MOLECULES

      
Application Number US2025022428
Publication Number 2025/212563
Status In Force
Filing Date 2025-04-01
Publication Date 2025-10-09
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Kohane, Daniel, S.
  • Aizik, Gil

Abstract

The present disclosure provides liposome gels. The liposome gels comprise gelling liposomes and may also comprise non-gelling particles (e.g., liposomes). The present disclosure also provides compositions comprising the liposome gels and a therapeutic agent or diagnostic agent. The compositions provide a means of controlled and prolonged delivery of the therapeutic and diagnostic agents. The present disclosure further provides kits comprising the compositions, methods of treating or preventing a disease or disorder, and methods of preparing the compositions.

IPC Classes  ?

  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
  • A61K 9/1271 - Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers

16.

COMPOSITIONS AND METHODS FOR STABILIZING MRNA

      
Application Number US2025023146
Publication Number 2025/213014
Status In Force
Filing Date 2025-04-04
Publication Date 2025-10-09
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor Chen, Kaifu

Abstract

Provided herein are methods for modifying mRNA, thereby stabilizing the mRNA. In particular, polypeptides of the disclosure may be used to modify mRNA with 2'-O-methylations.

IPC Classes  ?

  • C12N 9/10 - Transferases (2.)
  • C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
  • C12N 9/12 - Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
  • C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression

17.

SLC6A1 GENE THERAPY CONSTRUCTS

      
Application Number US2025021572
Publication Number 2025/207784
Status In Force
Filing Date 2025-03-26
Publication Date 2025-10-02
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Lee, Hing Cheong
  • Rotenberg, Alexander

Abstract

Provided herein are SLC6A1 gene therapy constructs, as well as SLC6A1 promoters, compositions comprising the constructs and promoters, and methods of using the same.

IPC Classes  ?

  • C12N 15/864 - Parvoviral vectors
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61P 25/00 - Drugs for disorders of the nervous system
  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals

18.

METHODS FOR CELL DELIVERY IN DECELLULARIZED HEART VALVES USING INJECTABLE HYDROGELS

      
Application Number US2025022135
Publication Number 2025/208090
Status In Force
Filing Date 2025-03-28
Publication Date 2025-10-02
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Mayer, John
  • Vetter, Valentine
  • Masoumi, Nafiseh
  • Tibbit, Mark
  • Gansevoort, Merel

Abstract

Provided herein are methods of preparing an engineered heart valve for implantation into a subject in need (e.g., a pediatric patient). For example, a porcine pulmonary valve can be decellularized and then recellularized with cells from the subject. Hydrogels can be used to facilitate cell delivery and growth within the sub-endothelial layer (e.g., spongiosa) of a heart valve, which is essential for long-term success of the implantation.

IPC Classes  ?

  • A61L 27/36 - Materials for prostheses or for coating prostheses containing ingredients of undetermined constitution or reaction products thereof
  • A61L 27/52 - Hydrogels or hydrocolloids
  • A61L 27/50 - Materials characterised by their function or physical properties

19.

NUCLEIC ACID NANOSWITCH CONSTRUCTION METHODS

      
Application Number 19054483
Status Pending
Filing Date 2025-02-14
First Publication Date 2025-10-02
Owner
  • Children's Medical Center Corporation (USA)
  • President and Fellows of Harvard College (USA)
Inventor
  • Yang, Darren
  • Mulhall, Eric M.
  • Zhao, Hongyu
  • Ward, Andrew
  • Hansen, Clinton H.
  • Wong, Wesley Philip

Abstract

This disclosure provides methods for generating functionalized nanoswitches, as well as the functionalized nanoswitches themselves, and methods of use thereof.

IPC Classes  ?

  • C07H 21/04 - Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression
  • C12Q 1/6804 - Nucleic acid analysis using immunogens
  • C12Q 1/6853 - Nucleic acid amplification reactions using modified primers or templates
  • C12Q 1/686 - Polymerase chain reaction [PCR]
  • G01N 33/542 - ImmunoassayBiospecific binding assayMaterials therefor with immune complex formed in liquid phase with steric inhibition or signal modification, e.g. fluorescent quenching

20.

INTERVENTIONAL DEVICE WITH ADJUSTABLE CONFIGURATION

      
Application Number 19081947
Status Pending
Filing Date 2025-03-17
First Publication Date 2025-10-02
Owner Children`s Medical Center Corporation (USA)
Inventor
  • Saeed, Mossab Y.
  • Del Nido, Pedro J.
  • Vasilyev, Nikolay V.
  • Ad, Niv
  • Payne, Christopher J.

Abstract

A device for performing intervention procedures includes an elongated body having a first end and a second end. The elongated body includes a hollow tool channel extending through the elongated body from the first end to the second end. The tool channel is configured to receive an intervention tool and a length-adjusting element disposed at the first end or the second end of the elongated body. The length-adjusting element is configured to enable a length of the elongated body to be adjusted. The elongated body includes an angle-adjusting element disposed along the length of the elongated body. The angle-adjusting element is configured to enable the elongated body to be bent. The device includes an imaging system disposed at the first end or the second end of the elongated body. The imaging system includes an imaging device and an illumination device.

IPC Classes  ?

  • A61M 25/01 - Introducing, guiding, advancing, emplacing or holding catheters
  • A61B 1/00 - Instruments for performing medical examinations of the interior of cavities or tubes of the body by visual or photographical inspection, e.g. endoscopesIlluminating arrangements therefor
  • A61B 1/005 - Flexible endoscopes
  • A61B 1/06 - Instruments for performing medical examinations of the interior of cavities or tubes of the body by visual or photographical inspection, e.g. endoscopesIlluminating arrangements therefor with illuminating arrangements
  • A61B 1/07 - Instruments for performing medical examinations of the interior of cavities or tubes of the body by visual or photographical inspection, e.g. endoscopesIlluminating arrangements therefor with illuminating arrangements using light-conductive means, e.g. optical fibres
  • A61B 5/318 - Heart-related electrical modalities, e.g. electrocardiography [ECG]

21.

RESONANCE RAMAN SPECTROSCOPY FOR EVALUATING MITOCHONDRIAL FUNCTION

      
Application Number 19175877
Status Pending
Filing Date 2025-04-10
First Publication Date 2025-10-02
Owner
  • Children's Medical Center Corporation (USA)
  • Pendar Technologies, LLC (USA)
Inventor
  • Smith, Lois
  • Cakir, Bertan D.
  • Romfh, John Padraic
  • Chen, Peili
  • Vakhshoori, Daryoosh

Abstract

Apparatuses and methods for using Raman Resonance Spectroscopy to evaluate metabolic and oxygenation status of the eye are disclosed herein. In some embodiments, metabolic mapping of the eye may be performed by aligning a Raman spectrum and a recorded spatial image of the eye.

IPC Classes  ?

  • G01N 21/65 - Raman scattering
  • A61B 3/10 - Objective types, i.e. instruments for examining the eyes independent of the patients perceptions or reactions
  • A61B 3/12 - Objective types, i.e. instruments for examining the eyes independent of the patients perceptions or reactions for looking at the eye fundus, e.g. ophthalmoscopes
  • G01J 3/44 - Raman spectrometryScattering spectrometry

22.

METHODS AND COMPOSITIONS FOR TAUOPATHY DIAGNOSIS AND TREATMENT

      
Application Number 18036020
Status Pending
Filing Date 2021-11-12
First Publication Date 2025-09-25
Owner Children`s Medical Center Corporation (USA)
Inventor
  • Steen, Judith Aj
  • Steen, Hanno
  • Wesseling, Hendrik
  • Kumar, Mukesh
  • Mair, Waltraud
  • Beerepoot, Pieter

Abstract

This disclosure relates to methods for diagnosing and treating a tauopathy, e.g., Alzheimer's disease, in a subject, the methods comprising, in part, identifying one or more post-translation modifications (PTMs) in the subject.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

23.

METHODS FOR NONINVASIVE MONITORING OF BRONCHOPULMONARY DYSPLASIA

      
Application Number 18712787
Status Pending
Filing Date 2022-11-22
First Publication Date 2025-09-25
Owner The Children`s Medical Center Corporation (USA)
Inventor
  • Steen, Hanno
  • Ahmed, Saima

Abstract

Provided herein are methods for diagnosing and treating a pulmonary disease, e.g., bronchopulmonary dysplasia (BPD) using proteomics analysis.

IPC Classes  ?

  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • G01N 30/88 - Integrated analysis systems specially adapted therefor, not covered by a single one of groups

24.

CARDIOMYOCYTES AND COMPOSITIONS AND METHODS FOR PRODUCING THE SAME

      
Application Number 18709798
Status Pending
Filing Date 2022-11-11
First Publication Date 2025-09-25
Owner
  • President and Fellows of Harvard College (USA)
  • The Children's Medical Center Corporation (USA)
Inventor
  • Liu, Jia
  • Aoyama, Junya
  • Li, Qiang
  • Liu, Ren
  • Lee, Richard T.
  • Garbern, Jessica

Abstract

Disclosed herein are methods for generating mature cardiomyocytes and compositions including mature cardiomyocytes. Also disclosed herein are methods for enhancing electrophysiological maturation of cardiomyocytes.

IPC Classes  ?

  • C12N 5/077 - Mesenchymal cells, e.g. bone cells, cartilage cells, marrow stromal cells, fat cells or muscle cells
  • A61K 35/34 - MusclesSmooth muscle cellsHeartCardiac stem cellsMyoblastsMyocytesCardiomyocytes

25.

THERAPEUTIC MODULATION OF GENES IN MYELOID LINEAGE CELLS AND USES THEREOF IN OPHTHALMOLOGY

      
Application Number 18861150
Status Pending
Filing Date 2023-04-28
First Publication Date 2025-09-18
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Sun, Ye
  • Wang, Tianxi

Abstract

Provided herein are methods for reducing the activation of myeloid lineage cells of a subject by targeting the expression and/or activity of factors that contribute to myeloid cell activation with a therapeutic agent. As a result of these methods, the activity of myeloid lineage cells that contribute to neovascularization in the retina of a subject may be reduced. The methods are useful for the treatment and/or prevention of various retinal diseases and injuries, including retinopathy of prematurity (ROP), age-related macular degeneration (AMD), retinitis pigmentosa, or diabetic retinopathy.

IPC Classes  ?

  • C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 27/02 - Ophthalmic agents
  • C12N 15/86 - Viral vectors

26.

COMPOSITIONS AND METHODS FOR DELIVERING NUCLEIC ACIDS TO PODOCYTES

      
Application Number US2025020011
Publication Number 2025/194082
Status In Force
Filing Date 2025-03-14
Publication Date 2025-09-18
Owner
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
  • BLACKFAN CIRCLE INNOVATIONS, LLC (USA)
Inventor
  • Hildebrandt, Friedhelm
  • Saida, Ken
  • Buerger, Florian
  • Schneider, Ronen
  • Steinsapir, Andrew
  • Goncalves, Kevin A.

Abstract

Provided herein are materials and methods for efficient and/or tropic delivery of transgenes to podocytes, and methods of treating podocytopathies associated with a genetic defect.

IPC Classes  ?

  • C12N 15/864 - Parvoviral vectors
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
  • A61K 35/76 - VirusesSubviral particlesBacteriophages

27.

THERAPEUTIC MODULATION OF TREM2 AND USES THEREOF IN RETINAL DEGENERATION

      
Application Number US2025020105
Publication Number 2025/194147
Status In Force
Filing Date 2025-03-14
Publication Date 2025-09-18
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Sun, Ye
  • Wang, Tianxi

Abstract

Provided herein are methods for treating retinal degeneration in a subject by targeting the expression and/or activity of Trem2 with a therapeutic agent. Also provided herein are methods for treating retinal degeneration or retinopathies in a subject by administering a Gasdermin D inhibitor to a subject. Also provided herein are methods for treating retinopathies in a subject by targeting the expression and/or activity of Aim2 with a therapeutic agent. The methods are useful for the treatment and/or prevention of various forms of retinal degeneration or retinopathies.

IPC Classes  ?

  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61K 40/17 - MonocytesMacrophages
  • A61K 40/24 - Antigen-presenting cells [APC]
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 27/02 - Ophthalmic agents
  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
  • C12N 15/79 - Vectors or expression systems specially adapted for eukaryotic hosts
  • A61K 31/7048 - Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin
  • A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
  • A61K 35/15 - Cells of the myeloid line, e.g. granulocytes, basophils, eosinophils, neutrophils, leucocytes, monocytes, macrophages or mast cellsMyeloid precursor cellsAntigen-presenting cells, e.g. dendritic cells

28.

METHODS FOR DIAGNOSIS AND TREATMENT OF ALZHEIMER'S DISEASE

      
Application Number 18549676
Status Pending
Filing Date 2022-02-28
First Publication Date 2025-08-28
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Steen, Hanno
  • Steen, Judith
  • Van Zalm, Patrick W.

Abstract

Provided herein are methods for diagnosing and treating Alzheimer's disease in a subject comprising determining the expression level of three, four or five members of a panel of proteins in a biological sample obtained from the subject.

IPC Classes  ?

  • C12Q 1/48 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving transferase
  • A61K 31/13 - Amines, e.g. amantadine
  • A61K 31/27 - Esters, e.g. nitroglycerine, selenocyanates of carbamic or thiocarbamic acids, e.g. meprobamate, carbachol, neostigmine
  • A61K 31/445 - Non-condensed piperidines, e.g. piperocaine
  • A61K 31/55 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
  • C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
  • C12Q 1/32 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving oxidoreductase involving dehydrogenase
  • C12Q 1/527 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving lyase
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • G01N 33/92 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving lipids, e.g. cholesterol

29.

2'-6'-PIPECOLOXYLIDIDE AS A SENSORY-SELECTIVE ANESTHETIC

      
Application Number US2025016039
Publication Number 2025/175174
Status In Force
Filing Date 2025-02-14
Publication Date 2025-08-21
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Kohane, Daniel S.
  • Ostertag-Hill, Claire A.

Abstract

A method of inducing local anesthesia in a subject includes administering to the subject in need thereof 2',6'-pipecoloxylidide (PPX) or a pharmaceutically acceptable salt thereof in an amount effective to induce local anesthesia in the subject. An active molecule that is metabolized to PPX in the subject is not administered to the subject with the PPX.

IPC Classes  ?

  • A61K 31/4458 - Non-condensed piperidines, e.g. piperocaine only substituted in position 2, e.g. methylphenidate
  • A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
  • A61K 31/05 - Phenols
  • A61P 23/02 - Local anaesthetics
  • A61P 23/00 - Anaesthetics

30.

METHODS FOR TREATING AND PREVENTING NEUTROPHIL-DERIVED NET TOXICITY AND THROMBOSIS

      
Application Number 19069515
Status Pending
Filing Date 2025-03-04
First Publication Date 2025-08-21
Owner CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Wagner, Denisa D.
  • Fuchs, Tobias A.
  • De Meyer, Simon
  • Martinod, Kimberly
  • Brill, Alexander
  • Thomas, Grace M.

Abstract

Embodiments of the technology described herein are based upon the discoveries that neturophil extracellular traps (NETs) provide a stimulus for thrombus formation and that NETs are present in stored blood products. Accordingly, some embodiments relate to methods of treating and preventing toxicity of NETs and thrombosis caused by NETs. Additional embodiments are directed towards methods of treating stored blood products to prevent transfusion-related injuries.

IPC Classes  ?

  • A61K 38/46 - Hydrolases (3)
  • A61K 31/155 - Amidines (), e.g. guanidine (H2N—C(=NH)—NH2), isourea (HN=C(OH)NH2), isothiourea (HN=C(SH)—NH2)
  • A61K 31/166 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the carbon atom of a carboxamide group directly attached to the aromatic ring, e.g. procainamide, procarbazine, metoclopramide, labetalol
  • A61K 38/49 - UrokinaseTissue plasminogen activator
  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61M 1/02 - Blood transfusion apparatus
  • A61M 1/34 - Filtering material out of the blood by passing it through a membrane, i.e. hemofiltration, diafiltration

31.

METHODS FOR MAPPING IMMUNE CELL CLONES IN SITU

      
Application Number US2025015663
Publication Number 2025/174923
Status In Force
Filing Date 2025-02-13
Publication Date 2025-08-21
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Moffitt, Jeffrey, R.
  • Yang, Evan
  • Carroll, Michael

Abstract

The technology described herein is directed to methods of preparing nucleic acid probes that can discriminate between closely related RNAs for use in fluorescent in situ hybridization techniques, i.e., FISH and MERFISH.

IPC Classes  ?

  • C12Q 1/6841 - In situ hybridisation
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12Q 1/6876 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
  • G16B 25/00 - ICT specially adapted for hybridisationICT specially adapted for gene or protein expression
  • G16B 30/10 - Sequence alignmentHomology search
  • G16B 10/00 - ICT specially adapted for evolutionary bioinformatics, e.g. phylogenetic tree construction or analysis
  • G06N 3/00 - Computing arrangements based on biological models
  • G01N 33/48 - Biological material, e.g. blood, urineHaemocytometers
  • C12N 5/078 - Cells from blood or from the immune system
  • C07K 14/705 - ReceptorsCell surface antigensCell surface determinants

32.

SYNTHETIC NUCLEIC ACIDS AND CELLS FOR IMMUNE CELL DIFFERENTIATION

      
Application Number US2025015936
Publication Number 2025/175104
Status In Force
Filing Date 2025-02-14
Publication Date 2025-08-21
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Schlaeger, Thorsten M.
  • Schuster-Kubaczka, Caroline M.

Abstract

Provided herein is a synthetic nucleic acid encoding at least one promoter, a transgene, and at least one inhibitory nucleic acid sequence. Cells, stables cell lines, and viruses expressing the same are also provided herein.

IPC Classes  ?

  • A61K 35/545 - Embryonic stem cellsPluripotent stem cellsInduced pluripotent stem cellsUncharacterised stem cells
  • A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
  • C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • C12N 15/86 - Viral vectors

33.

APOM FUSION CONSTRUCTS AND METHODS OF USE IN TREATING INFLAMMATORY DISEASES

      
Application Number US2025015976
Publication Number 2025/175130
Status In Force
Filing Date 2025-02-14
Publication Date 2025-08-21
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Hla, Timothy T.
  • Swendeman, Steven L.

Abstract

Described herein are ApoA1 and ApoM fusion proteins and methods of use thereof for treatment of vascular and inflammatory disorders. Also described herein are high-yield protocols to produce active ApoA1-ApoM fusion proteins from bacteria.

IPC Classes  ?

  • C07K 14/775 - Apolipopeptides
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • C12N 15/63 - Introduction of foreign genetic material using vectorsVectorsUse of hosts thereforRegulation of expression

34.

INTERPRETABLE SEQUENTIAL MULTIPLE-INSTANCE LEARNING FOR MEDICAL IMAGING

      
Application Number US2025013804
Publication Number 2025/166017
Status In Force
Filing Date 2025-01-30
Publication Date 2025-08-07
Owner
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
  • PRESIDENT AND FELLOWS OF HARVARD COLLEGE (USA)
Inventor
  • Li, Michael, Lingzhi
  • Wang, Hsin-Hsiao, Scott
  • Luo, Xiaolong

Abstract

A significant challenge in medical diagnostics is the often- sequential nature of various diagnostic modalities, for example, x-rays and ultrasounds. Systems and methods for using a machine learning model to analyze a sequence of scans to account for sequential information are provided herein. A system may receive a sequence of scans. The system may determine a subsequence of scans including a subset of scans from the sequence of scans. The system may determine an incremental prediction, using a machine learning model, based on the subsequence of scans. When the system determines all scans of the sequence are included in the subsequence, the system may determine an overall prediction based on one or more of the incremental predictions.

IPC Classes  ?

  • G16H 50/20 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for computer-aided diagnosis, e.g. based on medical expert systems
  • G06N 3/04 - Architecture, e.g. interconnection topology
  • G06N 3/08 - Learning methods
  • G16H 30/40 - ICT specially adapted for the handling or processing of medical images for processing medical images, e.g. editing
  • G06T 7/00 - Image analysis
  • G16H 10/60 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for patient-specific data, e.g. for electronic patient records

35.

COMPOSITIONS AND METHODS FOR TREATING AND/OR PREVENTING A VIRAL INFECTION

      
Application Number 18854929
Status Pending
Filing Date 2023-04-07
First Publication Date 2025-08-07
Owner
  • The Children's Medical Center Corporation (USA)
  • University of Helsinki (Finland)
Inventor
  • Kirchhausen, Tomas
  • Kreutzberger, Alex
  • Balistreri, Giuseppe

Abstract

The present disclosure provides compositions comprising a phosphatidylinositol-3-phosphate 5-kinase (PIKfyve kinase) inhibitor, or a pharmaceutically acceptable salt, solvate, hydrate, stereoisomer, polymorph, tautomer, isotopically enriched form, or prodrug thereof; a serine protease inhibitor, or a pharmaceutically acceptable salt, solvate, hydrate, stereoisomer, polymorph, tautomer, isotopically enriched form, or prodrug thereof; and water. Also provided in the present disclosure are methods, uses, and kits involving the compositions for treating and/or preventing a viral infection (e.g., infection by a coronavirus, an ebolavirus, a Lassa virus) in a subject. Also provided are methods, uses, and kits involving the compositions for treating and/or preventing damage to respiratory tissue (e.g. upper respiratory tissue, for example, nasal tissue, nasopharyngeal tissue, and/or lower respiratory tissue, for example, lung tissue) in a subject.

IPC Classes  ?

  • A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 9/08 - Solutions
  • A61K 31/245 - Amino benzoic acid types, e.g. procaine, novocaine
  • A61K 47/10 - AlcoholsPhenolsSalts thereof, e.g. glycerolPolyethylene glycols [PEG]PoloxamersPEG/POE alkyl ethers
  • A61K 47/20 - Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing sulfur, e.g. dimethyl sulfoxide [DMSO], docusate, sodium lauryl sulfate or aminosulfonic acids
  • A61K 47/26 - Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharidesDerivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
  • A61P 31/14 - Antivirals for RNA viruses

36.

COMPOSITIONS AND METHODS FOR TREATING USHER SYNDROME

      
Application Number US2025014149
Publication Number 2025/166253
Status In Force
Filing Date 2025-01-31
Publication Date 2025-08-07
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Geleoc, Gwenaelle, G.
  • Mauriac, Stephanie, A.
  • Yu, Timothy, W.
  • Huang, Yu-Han
  • Koehler, Karl

Abstract

USH2AUSH2A resulting in exon skipping and correcting splicing are used to treat individuals suffering from Usher Syndrome, type 2A and/or its symptoms. Compositions and kits containing antisense oligonucleotides of the disclosure are also provided.

IPC Classes  ?

  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
  • A61K 31/712 - Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
  • A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
  • A61P 27/02 - Ophthalmic agents

37.

ANTI-NET COMPOUNDS FOR TREATING AND PREVENTING FIBROSIS AND FOR FACILITATING WOUND HEALING

      
Application Number 19091081
Status Pending
Filing Date 2025-03-26
First Publication Date 2025-07-31
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Wagner, Denisa D.
  • Wong, Siu Ling
  • Martinod, Kimberly Lindsay
  • Erpenbeck, Luise
  • Witsch, Jöm Thilo
  • Savchenko, Alexander

Abstract

Embodiments of the technology described herein are based, in part, upon the discovery that NETosis, the formation of neutrophil extracellular traps (NETs) is increased in wounds, in organ fibrosis and in subjects with diabetes. Accordingly, methods for treating wounds, fibrosis and NET associated complications in diabetes are provided. The methods comprise administrating a therapeutically effective amount of at least one anti-NET compound to a subject in need of treatment, e.g. a PAD 4 inhibitor, a DNase, a histone-degrading enzyme; an inhibitor of chromatin decondensation; an antibody against a component of a NET; an inhibitor of NET release, a protease inhibitor, or an elastase inhibitor.

IPC Classes  ?

  • A61K 38/46 - Hydrolases (3)
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca

38.

CALMODULIN INHIBITORS FOR THE TREATMENT OF RIBOSOMAL DISORDERS AND RIBOSOMOPATHIES

      
Application Number 19085621
Status Pending
Filing Date 2025-03-20
First Publication Date 2025-07-31
Owner Children's Medical Center Corporation (USA)
Inventor
  • Zon, Leonard I.
  • Taylor, Alison M.

Abstract

The present invention relates generally to methods, compositions and kits for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA). In some embodiments, the invention relates to methods for the use of calmodulin inhibitors and calcium channel blockers for treatment of ribosomal disorders and ribosomopathy, e.g. Diamond Blackfan anemia (DBA).

IPC Classes  ?

  • A61K 31/553 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole having at least one nitrogen and at least one oxygen as ring hetero atoms, e.g. loxapine, staurosporine
  • A61K 31/145 - Amines, e.g. amantadine having sulfur atoms, e.g. thiurams (N—C(S)—S—C(S)—N or N—C(S)—S—S—C(S)—N)Sulfinylamines (—N=SO)Sulfonylamines (—N=SO2)
  • A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
  • A61K 31/4375 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having nitrogen as a ring hetero atom, e.g. quinolizines, naphthyridines, berberine, vincamine
  • A61K 31/4418 - Non-condensed pyridinesHydrogenated derivatives thereof having a carbocyclic ring directly attached to the heterocyclic ring, e.g. cyproheptadine
  • A61K 31/495 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two nitrogen atoms as the only ring hetero atoms, e.g. piperazine
  • A61K 31/4965 - Non-condensed pyrazines
  • A61K 31/54 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one sulfur as the ring hetero atoms, e.g. sulthiame
  • A61K 31/5415 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and at least one sulfur as the ring hetero atoms, e.g. sulthiame ortho- or peri-condensed with carbocyclic ring systems, e.g. phenothiazine, chlorpromazine, piroxicam

39.

MANIPULATION OF PURINE NUCLEOTIDE METABOLISM AS THERAPIES

      
Application Number US2025012868
Publication Number 2025/160341
Status In Force
Filing Date 2025-01-24
Publication Date 2025-07-31
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Agarwal, Suneet
  • Mannherz, William

Abstract

This document provides methods and materials for manipulating telomere length. For example, this document provides methods and materials for using one or more agents that can manipulate (e.g., increase or decrease) telomere length. In some cases, one or more agents that can increase telomere length can be contacted with a cell to increase telomere length in that cell. For example, one or more agents that can increase telomere length can be administered to a mammal having one or more TBDs to increase telomere length in cells within the mammal (e.g., to treat the mammal). In some cases, one or more agents that can decrease telomere length can be contacted with a cell to decrease telomere length in that cell. For example, one or more agents that can decrease telomere length can be administered to a mammal having a disease or disorder associated with increased telomere synthesis and/or increased telomerase activity to decrease telomere length in cells within the mammal (e.g., to treat the mammal).

IPC Classes  ?

  • A61K 45/00 - Medicinal preparations containing active ingredients not provided for in groups
  • H01L 31/18 - Processes or apparatus specially adapted for the manufacture or treatment of these devices or of parts thereof

40.

VECTORS COMBINING ANTI-SICKLING BETA-AS3-GLOBIN WITH ANTI BCEL11A SHRNAMIR TO TREAT BETA-HEMOGLOBINOPATHIES

      
Application Number 18845136
Status Pending
Filing Date 2023-03-10
First Publication Date 2025-07-24
Owner
  • The Regents of the University of California (USA)
  • The Children's Medical Center Corporation (USA)
  • Dana-Farber Cancer Institute, Inc. (USA)
Inventor
  • Kohn, Donald B.
  • Hollis, Roger
  • Hart, Kevyn
  • Williams, David
  • Brendel, Christian
  • Liu, Boya

Abstract

In certain embodiments, a lentiviral vector for the treatment of sickle cell disease (SCD) is provided. In certain embodiments, the vector comprises an expression cassette that encodes that an anti-sickling β-globin gene and an shRNA that inhibits expression of a BCL11A gene (BCL11A shRNA) wherein said expression cassette is in reverse orientation in the vector; a β-globin locus control region (LCR) comprising a reduced length hypersensitive site 1 (HS1) sequence, a reduced length hypersensitive site 2 (HS2) sequence, a reduced length hypersensitive site 3 (HS3) sequence, and a reduced length hypersensitive site 4 (HS4) sequence, where said anti-sickling β-globin gene is operably linked to the human β-globin locus control region.

IPC Classes  ?

  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid
  • C07K 14/805 - HaemoglobinsMyoglobins
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • C12N 5/0775 - Mesenchymal stem cellsAdipose-tissue derived stem cells
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C12N 15/86 - Viral vectors

41.

SYSTEMS AND METHODS FOR GENERATING NORMALIZED IMAGES OF BIOLOGICAL TISSUE SECTIONS

      
Application Number US2025012052
Publication Number 2025/155834
Status In Force
Filing Date 2025-01-17
Publication Date 2025-07-24
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • La, Jeffrey
  • Raghunathan, Krishnan
  • Silvester, Jocelyn
  • Thiagarajah, Jay

Abstract

The present disclosure provides methods, systems, and computer-readable media for stain normalization image processing of digitized biological tissue images. A method includes identifying a plurality of stain channels from an input image of a biological tissue section, generating a plurality of images for each stain channel based on relative stain concentration, and outputting stain-normalized images based on the generated images. A method for constructing scale invariant images of biological tissue sections includes identifying nuclei within an image, setting a length scale based on nuclei size, and scaling parameters for other morphological structures using the length scale. A method for identifying morphological structures includes generating a stain channel from an input image, determining a lower bound, creating a segmentation map, and identifying a morphological structure from the segmentation map. Systems and computer-readable media for performing these methods are also provided.

IPC Classes  ?

  • G01N 1/30 - StainingImpregnating
  • G01N 15/1433 - Signal processing using image recognition
  • G02B 21/36 - Microscopes arranged for photographic purposes or projection purposes
  • G06T 5/92 - Dynamic range modification of images or parts thereof based on global image properties
  • G06T 7/00 - Image analysis
  • G06V 10/56 - Extraction of image or video features relating to colour
  • G16H 10/40 - ICT specially adapted for the handling or processing of patient-related medical or healthcare data for data related to laboratory analysis, e.g. patient specimen analysis

42.

COMBINED VACCINE AGAINST MYCOBACTERIUM TUBERCULOSIS

      
Application Number 18787546
Status Pending
Filing Date 2024-07-29
First Publication Date 2025-07-17
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Zhang, Fan
  • Malley, Richard
  • Lu, Yingjie

Abstract

The present embodiments provide for a Mycobacterium tuberculosis (M. tuberculosis) Multiple Antigen Presenting System (MAPS) immunogenic composition comprising an immunogenic polysaccharide which induces an immune response, where at least one M. tuberculosis peptide or polypeptide antigen is associated to the immunogenic polysaccharide by complementary affinity molecules. In some embodiments, the immunogenic polysaccharide can be an antigenic capsular polysaccharide of a Mycobacterium tuberculosis, Type 5 (CP5) or Type 8 (CP8), or a combination of Type 5 or Type 8 capsular polysaccharide from Staphylococcus aureus, or alternatively, a different immunogenic capsular or noncapsular polysaccharide, and where the protein or peptide M. tuberculosis antigens are indirectly linked via an affinity binding pair. The present M. tuberculosis-MAPS immunogenic compositions can elicit both humoral and cellular immune responses to the immunogenic polysaccharide and one or multiple M. tuberculosis antigens at the same time.

IPC Classes  ?

  • A61K 39/04 - Mycobacterium, e.g. Mycobacterium tuberculosis
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61P 31/06 - Antibacterial agents for tuberculosis
  • C07K 14/35 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria from Mycobacteriaceae (F)
  • C07K 14/465 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from birds

43.

FUNCTIONALIZED IMIDAZOPYRIMIDINE COMPOUNDS AND USES THEREOF

      
Application Number US2025010919
Publication Number 2025/151620
Status In Force
Filing Date 2025-01-09
Publication Date 2025-07-17
Owner
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
  • INIMMUNE CORP. (USA)
Inventor
  • Levy, Ofer
  • Dowling, David J.
  • Soni, Dheeraj
  • Bazin-Lee, Helene
  • Bess, Laura
  • Hoody, John
  • Khalaf, Juhienah
  • Ryter, Kendal

Abstract

The present disclosure provides compounds of Formula (I), and pharmaceutically acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, isotopically labeled derivatives, and compositions thereof. The compound of Formula (I) is used as an enhancer and/or modifier of an immune response (e.g., innate and/or adaptive immune response), and is useful in treating and/or preventing a disease, as an adjuvant in a vaccine for a disease, (e.g., a proliferative disease, an inflammatory disease, an autoimmune disease, an infectious disease, an allergy, a fibrotic disease, a cardiovascular disease, a graft rejection, graft-versus-host disease, chronic disease, addiction, or risk of drug overdose), or as stand alone anti-infective or immune response modifying agents. Also provided in the present disclosure are vaccines, pharmaceutical compositions, kits, methods, and uses including or using compounds of Formula (I).

IPC Classes  ?

  • A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
  • A61K 31/166 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the carbon atom of a carboxamide group directly attached to the aromatic ring, e.g. procainamide, procarbazine, metoclopramide, labetalol
  • A61K 31/4188 - 1,3-Diazoles condensed with heterocyclic ring systems, e.g. biotin, sorbinil
  • A61K 31/33 - Heterocyclic compounds

44.

METHODS AND COMPOSITIONS FOR TREATING DISEASES ASSOCIATED WITH INFLAMMATION, PAIN AND/OR LESIONS

      
Application Number US2025010932
Publication Number 2025/151629
Status In Force
Filing Date 2025-01-09
Publication Date 2025-07-17
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Rogers, Michael S.
  • Heintz, Olivia

Abstract

The technology described herein is directed to methods of administering an IL-8 receptor inhibitor to prevent, reduce, or treat inflammation, pain, and/or lesions. Compositions comprising a IL-8 receptor inhibitor are also described herein.

IPC Classes  ?

  • A61K 31/145 - Amines, e.g. amantadine having sulfur atoms, e.g. thiurams (N—C(S)—S—C(S)—N or N—C(S)—S—S—C(S)—N)Sulfinylamines (—N=SO)Sulfonylamines (—N=SO2)
  • A61K 38/20 - Interleukins
  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61P 15/00 - Drugs for genital or sexual disordersContraceptives
  • A61P 29/02 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID] without antiinflammatory effect
  • A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons

45.

ASYMMETRIC EXPANDER

      
Application Number US2025011426
Publication Number 2025/151879
Status In Force
Filing Date 2025-01-13
Publication Date 2025-07-17
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor Maclaine, James

Abstract

An expansion device disclosed herein may include a lever arm attachable to a first side of a jaw of a patient; a body portion attachable to a second side of the jaw of the patient; wherein the lever arm is configured to pivot about a fixed pivot point on the body portion, the lever arm configured to pivot first side of the jaw away from the second side of the jaw of the patient while substantially maintaining a relative orientation of the second side of the jaw.

IPC Classes  ?

  • A61C 7/10 - Devices having means to apply outwardly directed force, e.g. expanders
  • A61C 8/00 - Means to be fixed to the jaw-bone for consolidating natural teeth or for fixing dental prostheses thereonDental implantsImplanting tools
  • A61B 17/64 - Devices extending alongside the bones to be positioned
  • A61B 17/66 - Compression or distraction mechanisms
  • A61C 7/36 - Devices acting between upper and lower teeth

46.

PREVENTION AND TREATMENT OF DIABETIC NEPHROPATHY

      
Application Number 18810089
Status Pending
Filing Date 2024-08-20
First Publication Date 2025-07-17
Owner
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
  • DOMPE FARMACEUTICI S.P.A. (Italy)
Inventor
  • Fiorina, Paolo
  • Bassi, Roberto
  • Vergani, Andrea
  • Allegretti, Marcello

Abstract

Embodiments herein disclose methods relating to diabetic nephropathy (DN); methods for preventing the onset and also for preventing the progressing of DN, as well as the treatment of DN in diabetic subjects comprising administering reparixin and/or ladarixin which are inhibitors of CXCL8 receptor CXCR1 and CXCR2 activation.

IPC Classes  ?

  • A61K 31/18 - Sulfonamides
  • A61K 31/426 - 1,3-Thiazoles
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 3/10 - Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
  • A61P 13/12 - Drugs for disorders of the urinary system of the kidneys
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

47.

SYSTEMS AND METHODS FOR TRANSCATHETER SURGERY

      
Application Number 18703198
Status Pending
Filing Date 2022-10-19
First Publication Date 2025-07-17
Owner
  • Children's Medical Center Corporation (USA)
  • The General Hospital Corporation (USA)
Inventor
  • Dupont, Pierre E.
  • Elmariah, Sammy

Abstract

A system for lacerating a tissue includes a catheter including one or more lacerators, where the one or more lacerators are configured to lacerate the tissue at a first exposure window and a second exposure window. The system also includes one or more aligners, where the one or more aligners are deployable and configured to, when deployed, promote contact between the one or more lacerators and the tissue at the first exposure window and/or the second exposure window.

IPC Classes  ?

  • A61B 18/24 - Surgical instruments, devices or methods for transferring non-mechanical forms of energy to or from the body by applying electromagnetic radiation, e.g. microwaves using laser the beam being directed along or through a flexible conduit, e.g. an optical fibreHand-pieces therefor with a catheter
  • A61B 18/00 - Surgical instruments, devices or methods for transferring non-mechanical forms of energy to or from the body
  • A61B 18/14 - Probes or electrodes therefor
  • A61B 18/20 - Surgical instruments, devices or methods for transferring non-mechanical forms of energy to or from the body by applying electromagnetic radiation, e.g. microwaves using laser
  • A61B 18/22 - Surgical instruments, devices or methods for transferring non-mechanical forms of energy to or from the body by applying electromagnetic radiation, e.g. microwaves using laser the beam being directed along or through a flexible conduit, e.g. an optical fibreHand-pieces therefor

48.

SUBCUTANEOUS ABRASION TREATMENT FOR STRETCH MARKS

      
Application Number 18703100
Status Pending
Filing Date 2022-10-19
First Publication Date 2025-07-10
Owner The Children's Medical Center Corporation (USA)
Inventor Greene, Arin K.

Abstract

Devices and methods for subcutaneously abrading an inner surface of a dermis of a subject are disclosed herein. A subcutaneous abrasion device may include a handle. an elongated shaft extending distally from the handle, and an abrasive surface disposed on a distal end portion of the elongated shaft. The abrasive surface may be configured to subcutaneously abrade an inner surface of a dermis of a subject without substantially cutting or puncturing the dermis of the subject.

IPC Classes  ?

49.

MODIFIED ALGINATES FOR ANTI-FIBROTIC MATERIALS AND APPLICATIONS

      
Application Number 18799747
Status Pending
Filing Date 2024-08-09
First Publication Date 2025-07-10
Owner
  • Massachusetts Institute of Technology (USA)
  • The Children's Medical Center Corporation (USA)
Inventor
  • Vegas, Arturo J.
  • Doloff, Joshua C.
  • Veiseh, Omid
  • Ma, Minglin
  • Langer, Robert S.
  • Anderson, Daniel G.

Abstract

Covalently modified alginate polymers, possessing enhanced biocompatibility and tailored physiochemical properties, as well as methods of making and use thereof, are disclosed herein. The covalently modified alginates are useful as a matrix for coating of any material where reduced fibrosis is desired, such as encapsulated cells for transplantation and medical devices implanted or used in the body.

IPC Classes  ?

  • A61L 29/08 - Materials for coatings
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 9/48 - Preparations in capsules, e.g. of gelatin, of chocolate
  • A61K 9/50 - Microcapsules
  • A61K 35/39 - PancreasIslets of Langerhans
  • A61K 47/36 - PolysaccharidesDerivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
  • A61L 31/10 - Macromolecular materials
  • A61L 33/08 - Polysaccharides
  • C07D 487/04 - Ortho-condensed systems
  • C08B 37/00 - Preparation of polysaccharides not provided for in groups Derivatives thereof
  • C12N 5/00 - Undifferentiated human, animal or plant cells, e.g. cell linesTissuesCultivation or maintenance thereofCulture media therefor
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues

50.

SELECTIVE HYPOTHALAMUS PERMEABLE HDAC6 INHIBITORS FOR TREATMENT OF LEPTIN-RESISTANT OBESITY

      
Application Number 19005377
Status Pending
Filing Date 2024-12-30
First Publication Date 2025-07-03
Owner
  • The Children’s Medical Center Corporation (USA)
  • The General Hospital Corporation (USA)
Inventor
  • Ozcan, Umut
  • Mazitschek, Ralph
  • Guan, Dongxian

Abstract

Formulations of HDAC6 inhibitors passing through the blood brain barrier in hypothalamus and inhibiting HDAC6 in the arctuate AgRP neurons in the hypothalamus, are effective to cause weight loss in obese individuals. These inhibitors also restore leptin sensitivity in leptin-resistant individuals.

IPC Classes  ?

  • A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
  • A61K 31/195 - Carboxylic acids, e.g. valproic acid having an amino group
  • A61K 31/505 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim
  • A61P 3/04 - AnorexiantsAntiobesity agents

51.

BIODEGRADABLE LIPIDS AND FORMULATIONS FOR DELIVERY OF MRNA

      
Application Number US2024061952
Publication Number 2025/144929
Status In Force
Filing Date 2024-12-26
Publication Date 2025-07-03
Owner
  • MASSACHUSETTS INSTITUTE OF TECHNOLOGY (USA)
  • CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Anderson, Daniel, Griffith
  • Rudra, Arnab
  • Gupta, Akash
  • Reed, Kaelan

Abstract

Provided herein are compounds, such as compounds of Formulae (I), (I'), (XI), (XII), and (XIII), and pharmaceutically acceptable salts, solvates, tautomers, stereoisomers, and isotopically labeled derivatives thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of agents, including polynucleotides such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, spleen diseases, lung diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, metabolic disorders, inflammatory diseases, and autoimmune diseases). Also provided herein are methods of synthesis of compounds of Formulae (I'), (XI), (XII), (XIII), and (VIII).

IPC Classes  ?

  • C07D 259/00 - Heterocyclic compounds containing rings having more than four nitrogen atoms as the only ring hetero atoms
  • C07D 471/10 - Spiro-condensed systems
  • C07D 243/08 - Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms having the nitrogen atoms in positions 1 and 4 not condensed with other rings
  • C07D 255/00 - Heterocyclic compounds containing rings having three nitrogen atoms as the only ring hetero atoms, not provided for by groups
  • A61P 1/16 - Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
  • A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
  • A61K 31/438 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom the ring being spiro-condensed with carbocyclic or heterocyclic ring systems

52.

ENTEROCOCCUS PORE-FORMING TOXINS AND METHODS OF USE THEREOF

      
Application Number 18843656
Status Pending
Filing Date 2023-03-03
First Publication Date 2025-07-03
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Dong, Min
  • Xiong, Xiaozhe
  • Tian, Songhai

Abstract

The present application describes in part isolated and modified Enterococci toxin (Epx) polypeptides, immunogenic compositions comprising Exp polypeptides, nanopores formed by Epx polypeptides, apparatus comprising Epx nanopores, and methods of use thereof.

IPC Classes  ?

  • C07K 14/315 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
  • A61K 38/00 - Medicinal preparations containing peptides

53.

COMBINATION THERAPIES COMPRISING ANTIBODY MOLECULES TO TIM-3

      
Application Number 18980647
Status Pending
Filing Date 2024-12-13
First Publication Date 2025-07-03
Owner
  • Novartis AG (Switzerland)
  • Dana-Farber Cancer Institute, Inc. (USA)
  • Children's Medical Center Corporation (USA)
Inventor
  • Dranoff, Glenn
  • Sabatos-Peyton, Catherine Anne
  • Brannetti, Barbara
  • Harris, Alan S.
  • Huber, Thomas
  • Pietzonka, Thomas
  • Mataraza, Jennifer Marrie
  • Blattler, Walter A.
  • Hicklin, Daniel J.
  • Vasquez, Maximiliano
  • Dekruyff, Rosemarie H.
  • Umetsu, Dale T.
  • Freeman, Gordon James
  • Hu, Tiancen
  • Taraszka, John A.
  • Xu, Fangmin

Abstract

Combination therapies comprising antibody molecules that specifically bind to TIM-3 are disclosed. The combination therapies can be used to treat or prevent cancerous or infectious conditions and disorders.

IPC Classes  ?

  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 35/00 - Antineoplastic agents
  • C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants

54.

BIODEGRADABLE LIPIDS AND FORMULATIONS FOR DELIVERY OF MRNA

      
Application Number 19002347
Status Pending
Filing Date 2024-12-26
First Publication Date 2025-06-26
Owner
  • Massachusetts Institute of Technology (USA)
  • Children's Medical Center Corporation (USA)
Inventor
  • Anderson, Daniel Griffith
  • Rudra, Arnab
  • Gupta, Akash
  • Reed, Kaelan

Abstract

Provided herein are compounds, such as compounds of Formulae (I), (I′), (XI), (XII), and (XIII), and pharmaceutically acceptable salts, solvates, tautomers, stereoisomers, and isotopically labeled derivatives thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of agents, including polynucleotides such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, spleen diseases, lung diseases, painful conditions, psychiatric disorders, musculoskeletal diseases, metabolic disorders, inflammatory diseases, and autoimmune diseases). Also provided herein are methods of synthesis of compounds of Formulae (I′), (XI), (XII), (XIII), and (VIII).

IPC Classes  ?

  • A61K 9/1272 - Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
  • A61K 9/51 - Nanocapsules
  • C07D 243/08 - Heterocyclic compounds containing seven-membered rings having two nitrogen atoms as the only ring hetero atoms having the nitrogen atoms in positions 1 and 4 not condensed with other rings
  • C07D 255/02 - Heterocyclic compounds containing rings having three nitrogen atoms as the only ring hetero atoms, not provided for by groups not condensed with other rings
  • C07D 259/00 - Heterocyclic compounds containing rings having more than four nitrogen atoms as the only ring hetero atoms
  • C07D 471/10 - Spiro-condensed systems

55.

MODIFIED INTEGRIN POLYPEPTIDES, MODIFIED INTEGRIN POLYPEPTIDE DIMERS, AND USES THEREOF

      
Application Number 19041606
Status Pending
Filing Date 2025-01-30
First Publication Date 2025-06-26
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Springer, Timothy Alan
  • Dong, Xianchi
  • Lu, Chafen

Abstract

Described herein are modified integrin α and/or β headpiece polypeptides, and crystallizable integrin polypeptide dimers comprising a modified integrin α and/or β headpiece polypeptide and a disulfide bond linking the two integrin headpiece polypeptide subunits. Methods for using the modified integrin α and/or β headpiece polypeptides and the integrin polypeptide dimers are also provided herein. For example, methods for characterizing integrin-ligand interaction and identifying integrin ligands are also provided herein. In some embodiments, the identified integrin ligands can be used as inhibitors of integrins.

IPC Classes  ?

  • C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
  • G16C 10/00 - Computational theoretical chemistry, i.e. ICT specially adapted for theoretical aspects of quantum chemistry, molecular mechanics, molecular dynamics or the like

56.

CATHETER MANEUVERING DEVICE AND USES THEREOF

      
Application Number US2024061067
Publication Number 2025/137306
Status In Force
Filing Date 2024-12-19
Publication Date 2025-06-26
Owner
  • TRUSTEES OF BOSTON UNIVERSITY (USA)
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Ranzani, Tommaso
  • Rogatinsky, Jacob
  • Kang, Yuchen
  • Feichtmeier, Joseph
  • Recco, Dominic
  • Hoganson, David
  • Zamora, Leonardo
  • Matthews, Grace
  • Mah, Douglas Yeu
  • O'Leary, Edward

Abstract

Devices and accompanying methods and systems for performing a stabilized endoluminal procedure are disclosed. A device includes a catheter defining a working channel and configured to carry multiple fluidic lines. The device includes a stabilization mechanism configured to expand in a deployed state to stabilize the catheter against tissue. A soft manipulator disposed at a distal end of the device includes multiple actuators configured to expand, to collapse, or to bend. The multiple actuators define a guided channel in communication with the working channel. A hub at a proximal end of the catheter is in coupled configuration with a stabilization actuator mechanically coupled to the stabilization mechanism. The hub defines a port in communication with the working channel and enables the passage of the fluidic lines. The device enables interventional therapies in motile environments, for example a beating-heart procedure.

IPC Classes  ?

  • A61F 2/848 - Devices providing patency to, or preventing collapsing of, tubular structures of the body, e.g. stents having means for fixation to the vessel wall, e.g. barbs
  • A61F 2/82 - Devices providing patency to, or preventing collapsing of, tubular structures of the body, e.g. stents
  • A61F 2/86 - Stents in a form characterised by wire-like elementsStents in a form characterised by a net-like or mesh-like structure
  • B25J 13/08 - Controls for manipulators by means of sensing devices, e.g. viewing or touching devices

57.

NON-CLEAVING GASDERMIN D AGONISTS

      
Application Number US2024061219
Publication Number 2025/137411
Status In Force
Filing Date 2024-12-20
Publication Date 2025-06-26
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Wu, Hao
  • Lieberman, Judy
  • Fontana, Pietro
  • Zhang, Haiwei
  • Vora, Setu M.
  • Hu, Jun

Abstract

in vitro in vivo.in vivo. In mouse tumor models, pulsed and low level pyroptosis induction by DMB suppressed tumor growth without harming GSDMD-expressing immune cells, in an immune-mediated mechanism. Vaccination with DMB-treated cancer cells protected mice from secondary tumor challenge, indicating immunogenic cell death is induced. DMB treatment also synergized with anti-PD-1. Accordingly, the disclosed GSDMD compounds form the basis for a new strategy for tumor immunotherapy.

IPC Classes  ?

  • A61P 35/00 - Antineoplastic agents
  • C07D 241/44 - Benzopyrazines with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to carbon atoms of the hetero ring

58.

HYDROGELS COMPRISING PROTEIN-STABILIZED NANOPARTICLES FOR EXTENDED RELEASE

      
Application Number US2024059479
Publication Number 2025/128639
Status In Force
Filing Date 2024-12-11
Publication Date 2025-06-19
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Kohane, Daniel S.
  • Choi, Wonmin

Abstract

The present disclosure provides compositions comprising a hydrogel and a protein-stabilized nanoparticle. The hydrogel comprises a crosslinked protein matrix, the protein-stabilized nanoparticle comprises a therapeutic agent or a diagnostic agent, and the protein-stabilized nanoparticle is embedded in the crosslinked protein matrix. The compositions thus provide a means of controlled and prolonged delivery of therapeutic and diagnostic agents. The present disclosure further provides kits comprising the compositions, methods of treating or preventing a disease or disorder, and methods of preparing the compositions.

IPC Classes  ?

  • A61K 9/14 - Particulate form, e.g. powders
  • A61L 27/52 - Hydrogels or hydrocolloids
  • C07K 17/02 - Peptides being immobilised on, or in, an organic carrier
  • C07K 17/08 - Peptides being immobilised on, or in, an organic carrier the carrier being a synthetic polymer

59.

COVALENT ANESTHETIC-POLYMER CONJUGATES FOR PROLONGED LOCAL ANESTHESIA

      
Application Number 18817160
Status Pending
Filing Date 2024-08-27
First Publication Date 2025-06-19
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Kohane, Daniel S.
  • Zhao, Chao

Abstract

Anesthetics covalently conjugated onto biodegradable and biocompatible hydrophilic polymers via hydrolysable linkages provide controlled release of local anesthetics in vivo in an effective amount for nerve blockade with reduced toxicity relative to the unconjugated anesthetic agent. The rate of anesthetic release can be tuned by changing the hydrophilicity of the polymer. Exemplary formulations of Poly (glycerol sebacate) (PGS), optionally including Poly ethylene glycol (PEG) polymers conjugated to Tetrodotoxin (TTX) (PGS-PEG-TTX and PGS-TTX), and methods of use thereof are provided Nerve blockade from PGS-PEG-TTX and PGS-TTX was associated with minimal systemic and local toxicity to the muscle and the peripheral nerves. TDP-TTX conjugates homogeneously dispersed into PEG200 are also described. PEG200 not only worked as a medium, but also worked as a chemical permeation enhancer (CPE) to enhance the effectiveness of TTX.

IPC Classes  ?

  • A61K 47/60 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings

60.

GTP CYCLOHYDROLASE-CLEAVING PROTEASES

      
Application Number 19064858
Status Pending
Filing Date 2025-02-27
First Publication Date 2025-06-19
Owner
  • The Broad Institute, Inc. (USA)
  • Children's Medical Center Corporation (USA)
Inventor
  • Liu, David R.
  • Blum, Travis R.
  • Dong, Min
  • Manion, John
  • Hemez, Colin
  • Mccreary, Julia

Abstract

Aspects of the disclosure relate to Botulinum toxin X (BoNT X) protein variants. The variants provided herein have been evolved to cleave GTP cyclohydrolase 1 (GCH1). Some of the variants provided herein were evolved from a procaspase-1 cleaving polypeptide. Further aspects of the disclosure relate to nucleic acids encoding the GCH1 cleaving polypeptides described herein and expression vectors comprising the nucleic acids, as well as host cells and fusion proteins comprising the GCH1 cleaving polypeptides described herein, and kits comprising the GCH1 polypeptides, fusion proteins, nucleic acids, expression vectors, or host cells described herein. Further aspects of the disclosure relate to methods of producing BoNT X variants and methods of using the BoNT X protein variants, for example, to reduce pain.

IPC Classes  ?

61.

METHOD FOR TREATING CLONAL HEMATOPOIETIC BLOOD DISORDERS

      
Application Number 18843935
Status Pending
Filing Date 2023-03-31
First Publication Date 2025-06-12
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Zon, Leonard I.
  • Henninger, Jonathan E.
  • Avagyan, Serine

Abstract

The disclosure provides method for treating clonal hematopoiesis disorders such as clonal hematopoiesis, myelodysplasia, and leukemia.

IPC Classes  ?

  • C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
  • A61K 31/404 - Indoles, e.g. pindolol
  • A61K 31/415 - 1,2-Diazoles
  • A61K 31/4745 - QuinolinesIsoquinolines ortho- or peri-condensed with heterocyclic ring systems condensed with ring systems having nitrogen as a ring hetero atom, e.g. phenanthrolines
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

62.

A MULTIPLE ANTIGEN PRESENTING SYSTEM (MAPS)-BASED STAPHYLOCOCCUS AUREUS VACCINE COMPRISING B- AND T-CELL ANTIGENS, IMMUNOGENIC COMPOSITION, AND USES THEREOF

      
Application Number 18846176
Status Pending
Filing Date 2023-03-10
First Publication Date 2025-06-12
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Malley, Richard
  • Lu, Yingjie
  • Zhang, Fan
  • Moffitt, Kristin

Abstract

The present embodiments provide for an S. aureus (SA). Multiple Antigen Presenting System (MAPS) immunogenic composition comprising an immunogenic polysaccharide which induces a B-cell and T-cell immune response, where at least one S. aureus (SA) peptide or polypeptide antigen is associated to the immunogenic polysaccharide by complementary affinity molecules. The present SA-MAPS immunogenic composition herein generates an immune response in a subject, preferably an antibody response and a B-cell and/or T-cell response, and can comprise at least one B-cell SA antigen and at least one T-cell SA antigen, and can elicit both humoral and cellular immune responses to the immunogenic polysaccharide and one, or multiple SA antigens at the same time.

IPC Classes  ?

  • C07K 14/31 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria from Micrococcaceae (F) from Staphylococcus (G)
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies

63.

PYK2 INHIBITION MODULATES IMMUNE CELL FUNCTION

      
Application Number 18847215
Status Pending
Filing Date 2023-03-16
First Publication Date 2025-06-12
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Kong, Sek Won
  • Lee, Jiwon

Abstract

Provided herein are novel therapeutic applications of Pyk2 (PTK2B) inhibitors to treat low bone mineral density and/or osteoporosis and to treat, prevent, or delay the progression of neurodegenerative disorders such as Alzheimer's disease.

IPC Classes  ?

  • A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
  • A61K 31/5377 - 1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
  • A61P 19/10 - Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease for osteoporosis
  • A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
  • C07K 16/40 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against enzymes
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides

64.

CARDIOMYOCYTES AND COMPOSITIONS AND METHODS FOR PRODUCING THE SAME

      
Application Number 18286405
Status Pending
Filing Date 2022-03-29
First Publication Date 2025-06-12
Owner
  • President and Fellows of Harvard College (USA)
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Lee, Richard T.
  • Garbern, Jessica

Abstract

Disclosed herein are methods for generating mature cardiomyocytes and compositions including mature cardiomyocytes. Also disclosed herein are methods for enhancing maturation of quiescent cardiomyocytes and compositions including mature quiescent cardiomyocytes.

IPC Classes  ?

  • C12N 5/077 - Mesenchymal cells, e.g. bone cells, cartilage cells, marrow stromal cells, fat cells or muscle cells
  • A61K 35/34 - MusclesSmooth muscle cellsHeartCardiac stem cellsMyoblastsMyocytesCardiomyocytes

65.

COMPOSITIONS AND METHODS FOR TARGETING A VIRAL INFECTION

      
Application Number 18763369
Status Pending
Filing Date 2024-07-03
First Publication Date 2025-06-05
Owner THE CHILDEREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Chen, Bing
  • Frey, Gary
  • Xiao, Tianshu

Abstract

Described herein are compounds that inhibits the membrane proximal external region (MPER) of a viral envelope (Env), as well as and compositions thereof. Further provided herein are methods for treating or preventing a viral infection comprises administering to a subject in need thereof an agent or compound that inhibits the MPER of a viral Env. In certain embodiments, the viral infection is an HIV infection.

IPC Classes  ?

  • A61K 31/4709 - Non-condensed quinolines containing further heterocyclic rings

66.

Automated Isolation of Viable Mitochondria

      
Application Number 18800924
Status Pending
Filing Date 2024-08-12
First Publication Date 2025-06-05
Owner Children`s Medical Center Corporation (USA)
Inventor
  • Mccully, James D.
  • Cowan, Douglas B.
  • Del Nido, Pedro J.
  • Duignan, Thomas

Abstract

An automated isolation device for isolation of mitochondria includes an incubation station including a holder for a viable mitochondria solution; and a cooling system, controlled by a processor of the device, for cooling the holder. The device includes a processor-controlled transfer system for transferring solution from the holder to a filtration station; and the filtration station, including a series of filters. The device includes a processor-controlled spectrometry station including a spectrometer positioned to illuminate a cuvette fluidically coupled to an output of the filtration station; and a detector coupled to the processor and positioned on a side of the cuvette opposite the spectrometer. The device includes a processor-controlled transfer system for transferring solution from the spectrometry station to a centrifuge. The centrifuge is processor-controlled and configured to centrifuge the filtrate to separate viable mitochondria from a supernatant.

IPC Classes  ?

  • C12M 1/36 - Apparatus for enzymology or microbiology including condition or time responsive control, e.g. automatically controlled fermentors
  • C12M 1/00 - Apparatus for enzymology or microbiology
  • C12M 1/26 - Inoculator or sampler
  • G01J 3/44 - Raman spectrometryScattering spectrometry
  • G01N 15/04 - Investigating sedimentation of particle suspensions
  • G01N 33/50 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing
  • G01N 35/00 - Automatic analysis not limited to methods or materials provided for in any single one of groups Handling materials therefor

67.

REGULATORY T CELLS WITH CHIMERIC ANTIGEN RECEPTOR TARGETING CO-STIMULATORY MOLECULES TO PREVENT AND/OR TREAT INFLAMMATORY CONDITIONS

      
Application Number 18842076
Status Pending
Filing Date 2023-02-28
First Publication Date 2025-06-05
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Kean, Leslie S.
  • Rui, Xianliang
  • Tkachev, Victor
  • Gerdemann, Ulrike

Abstract

Described herein are methods and compositions for regulatory T cells (Tregs) that are modified to express a chimeric antigen receptor (CAR) that targets OX40L. Aspects of the invention relate to administering these modified Tregs to a subject having an inflammatory or autoimmune condition.

IPC Classes  ?

  • A61K 40/31 - Chimeric antigen receptors [CAR]
  • A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
  • A61K 40/11 - T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cellsLymphokine-activated killer [LAK] cells
  • A61K 40/42 - Cancer antigens
  • C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
  • C07K 14/725 - T-cell receptors
  • C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
  • C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells

68.

METHODS AND COMPOSITIONS FOR THE TREATMENT OF BLOOD CANCER

      
Application Number US2024057259
Publication Number 2025/117429
Status In Force
Filing Date 2024-11-25
Publication Date 2025-06-05
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Italiano, Joseph E.
  • Becker, Isabelle C.

Abstract

Described herein are methods relating to the treatment of a blood cancer by administration of a RhoA/ROCK inhibitor, ROCK1 inhibitor, and/or JAK2 inhibitor.

IPC Classes  ?

  • A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
  • A61K 31/47 - QuinolinesIsoquinolines
  • A61K 31/18 - Sulfonamides
  • A61K 31/33 - Heterocyclic compounds

69.

METHODS AND COMPOSITIONS RELATING TO IMMUNIZATION OF IMMUNE DISTINCT PATIENTS

      
Application Number 18727421
Status Pending
Filing Date 2023-01-10
First Publication Date 2025-06-05
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Dowling, David J.
  • Brook, Byron Nf

Abstract

Described herein are methods and compositions relating to vaccinating, immunizing, or inducing an immune response in an immune distinct subject. In some embodiments, the methods and compositions relate to a first cytokine mRNA construct comprising a first open reading frame (ORF), wherein the first ORF encodes a proinflammatory cytokine; and optionally, one or more of: a first antigen mRNA construct comprising a second open reading frame (ORF), wherein the second ORF encodes an antigen; and an antigen polypeptide, antigen molecule, or killed or attenuated pathogenic agent. The methods and compositions described herein provide adjuvantation that overcomes the resistance of immune distinct patients to vaccination, permitting more effective vaccination, as well as the ability to reduce dosages, reduce the need for boosters, and permit antigen stacking to immunize more comprehensively.

IPC Classes  ?

  • A61K 39/39 - Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61K 39/015 - Hemosporidia antigens, e.g. Plasmodium antigens
  • A61K 39/02 - Bacterial antigens
  • A61K 39/04 - Mycobacterium, e.g. Mycobacterium tuberculosis
  • A61K 39/085 - Staphylococcus
  • A61K 39/09 - Streptococcus
  • A61K 39/12 - Viral antigens
  • A61K 39/145 - Orthomyxoviridae, e.g. influenza virus
  • A61K 39/21 - Retroviridae, e.g. equine infectious anemia virus
  • A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
  • A61K 39/25 - Varicella-zoster virus
  • A61P 37/04 - Immunostimulants

70.

METHODS AND COMPOSITIONS FOR EDITING SBDS GENE

      
Application Number 18947543
Status Pending
Filing Date 2024-11-14
First Publication Date 2025-06-05
Owner The Children’s Medical Center Corporation (USA)
Inventor Brendel, Christian

Abstract

Provided herein is a base editor system for editing the SBDS gene comprising a base editor protein and a guide polynucleotide, wherein the base editor protein comprises a polynucleotide programmable DNA binding domain and a cytosine deaminase domain. Methods, edited cells, and compositions thereof are also described herein.

IPC Classes  ?

  • C12N 9/80 - Hydrolases (3.) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides
  • A61P 19/08 - Drugs for skeletal disorders for bone diseases, e.g. rachitism, Paget's disease
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12N 15/90 - Stable introduction of foreign DNA into chromosome

71.

METHODS AND COMPOSITIONS RELATING TO HEMATOPOIETIC STEM CELL EXPANSION

      
Application Number 18987345
Status Pending
Filing Date 2024-12-19
First Publication Date 2025-06-05
Owner CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Rossi, Derrick J.
  • Ebina, Wataru

Abstract

Described herein are compositions and kits comprising at least a compound of structure (1).

IPC Classes  ?

  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells

72.

ANTI-VIRULENCE AGENTS AND METHODS FOR THE TREATMENT OF PATHOGENIC BACTERIUM

      
Application Number US2024057701
Publication Number 2025/117707
Status In Force
Filing Date 2024-11-27
Publication Date 2025-06-05
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Schaefers, Matthew M.
  • Priebe, Gregory P.

Abstract

This disclosure relates to agents and methods useful for inhibiting and/or reducing the virulence of pathogenic bacterial cells, and for treating infections associated with such bacterial cells.

IPC Classes  ?

73.

METHODS FOR GENERATING FUNCTIONAL HEMATOPOIETIC STEM CELLS

      
Application Number 18968775
Status Pending
Filing Date 2024-12-04
First Publication Date 2025-05-29
Owner
  • The Brigham and Women`s Hospital, Inc. (USA)
  • Children`s Medical Center Corporation (USA)
Inventor
  • Shah, Dhvanit I.
  • Daley, George Q.

Abstract

Described in the present application are methods for preparing populations of hematopoietic stem cells (HSCs), e.g., autologous and/or allogenic HSCs, using mechanical stretching or Trpv4 agonists, and methods of use of the HSCs in transplantation. In some embodiments, the methods include providing a population comprising hemogenic endothelial (HE) cells, and (i) contacting the HE cells with an amount of an agonist of transient receptor potential cation channel-subfamily vanilloid member 4 (Trpv4); and/or (ii) subjecting the cells to cyclic 2-dimensional stretching, for a time and under conditions sufficient to stimulating endothelial-to-HSC transition. Also provided herein are methods for treating subjects who have, bone marrow, metabolic, and immune diseases; the methods include administering to the subject a therapeutically effective amount of hematopoietic stem cells (HSCs) obtained by a method described herein.

IPC Classes  ?

  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • A01K 67/0271 - Chimeric vertebrates, e.g. comprising exogenous cells
  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells

74.

EPITOPE ENGINEERING OF CELL-SURFACE RECEPTORS

      
Application Number 18838922
Status Pending
Filing Date 2023-02-16
First Publication Date 2025-05-29
Owner
  • Dana-Farber Cancer Institute, Inc. (USA)
  • The Children's Medical Center Corporation (USA)
Inventor
  • Genovese, Pietro
  • Casirati, Gabriele

Abstract

Genetically engineered hematopoietic cells such as hematopoietic stem cells having one or more genetically edited genes of cell-surface proteins and therapeutic uses thereof, either alone or in combination with immune therapy that targets the cell-surface protein(s).

IPC Classes  ?

  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
  • A61K 40/11 - T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cellsLymphokine-activated killer [LAK] cells
  • A61K 40/31 - Chimeric antigen receptors [CAR]
  • A61K 40/42 - Cancer antigens
  • A61P 35/02 - Antineoplastic agents specific for leukemia
  • C07K 14/715 - ReceptorsCell surface antigensCell surface determinants for cytokinesReceptorsCell surface antigensCell surface determinants for lymphokinesReceptorsCell surface antigensCell surface determinants for interferons
  • C07K 14/725 - T-cell receptors
  • C07K 16/28 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
  • C07K 16/40 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against enzymes
  • C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • C12N 9/12 - Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof

75.

SELECTIVE OXIDATION OF 5-METHYLCYTOSINE BY TET-FAMILY PROTEINS

      
Application Number 19028618
Status Pending
Filing Date 2025-01-17
First Publication Date 2025-05-29
Owner
  • The Children's Medical Center Corporation (USA)
  • The United States of America, As Represented by the Secretary, Department of Health & Human Services (USA)
Inventor
  • Rao, Anjana
  • Tahiliani, Mamta
  • Koh, Kian Peng
  • Agarwal, Suneet
  • Iyer, Aravind

Abstract

The present invention provides for novel methods for regulating and detecting the cytosine methylation status of DNA. The invention is based upon identification of a novel and surprising catalytic activity for the family of TET proteins, namely TET1, TET2, TET3, and CXXC4. The novel activity is related to the enzymes being capable of converting the cytosine nucleotide 5-methylcytosine into 5-hydroxymethylcytosine by hydroxylation.

IPC Classes  ?

  • C12Q 1/6827 - Hybridisation assays for detection of mutation or polymorphism
  • C12N 9/10 - Transferases (2.)
  • C12Q 1/26 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving oxidoreductase
  • C12Q 1/6806 - Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
  • C12Q 1/6869 - Methods for sequencing
  • G01N 33/53 - ImmunoassayBiospecific binding assayMaterials therefor

76.

COMPOUNDS FOR THE TREATMENT OF OBESITY AND METHODS OF USE THEREOF

      
Application Number 18767788
Status Pending
Filing Date 2024-07-09
First Publication Date 2025-05-22
Owner
  • The Children’s Medical Center Corporation (USA)
  • The General Hospital Corporation (USA)
Inventor
  • Ozcan, Umut
  • Majzoub, Joseph
  • Mazitschek, Ralph

Abstract

Pentacyclic triterpene weight loss agents are provided herein. Also provided are pharmaceutical formulations containing a therapeutically effective amount of one or more of the weight loss agents, or pharmaceutically acceptable salts or prodrugs thereof, in combination with one or more pharmaceutically acceptable excipients. The pharmaceutical formulations can be administered to a pre-obese, obese, or morbidly obese patient to induce weight loss, reduce body fat, reduce food intake, improve glucose homeostasis, prevent obesity, or a combination thereof. The weight loss agents can also be co-administered with leptin or a leptin analog.

IPC Classes  ?

  • A61K 31/198 - Alpha-amino acids, e.g. alanine or edetic acid [EDTA]
  • A61K 31/19 - Carboxylic acids, e.g. valproic acid
  • A61K 31/192 - Carboxylic acids, e.g. valproic acid having aromatic groups, e.g. sulindac, 2-aryl-propionic acids, ethacrynic acid
  • A61K 31/194 - Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
  • A61K 31/195 - Carboxylic acids, e.g. valproic acid having an amino group
  • A61K 38/22 - Hormones
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 3/04 - AnorexiantsAntiobesity agents

77.

METHODS OF VASCULAR CELL THERAPY AND ENDOTHELIAL CELL ENGRAFTMENT VIA MITOPHAGY INDUCERS

      
Application Number US2024055896
Publication Number 2025/106654
Status In Force
Filing Date 2024-11-14
Publication Date 2025-05-22
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Melero-Martin, Juan M.
  • Lin, Ruei-Zeng

Abstract

inter aliainter aliainter alia, are methods vascular cell therapy, promoting blood vessel formation, and increasing vascular development, angiogenesis, and cell junction via administration of a population of ECs comprising a mitophagy-inducing agent (MIA-ECs).

IPC Classes  ?

  • A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
  • A61K 9/08 - Solutions
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • A61P 9/14 - VasoprotectivesAntihaemorrhoidalsDrugs for varicose therapyCapillary stabilisers

78.

COMBINATION BCL11A ENHANCER EDITING

      
Application Number 18717370
Status Pending
Filing Date 2022-12-09
First Publication Date 2025-05-15
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Bauer, Daniel E.
  • Zeng, Jing

Abstract

Provided herein are vectors, and compositions thereof, comprising at least two guide RNAs (gRNAs) that targets and hybridizes to a target sequence on a DNA molecule, wherein each of the at least two gRNAs hybridizes at least two genomic DNA locations selected from human chromosome 2 at location 60725424 to 60725688 according to GRCh37/hg19 human genome reference build (+55 functional region); human chromosome 2 at location 60722238 to 60722466 GRCh37/hg19 human genome reference build (+58 functional region); or human chromosome 2 at location 60718042 to 60718186 according to GRCh37/hg19 human genome reference build (+62 functional region). Methods for the use of such vectors are further provided.

IPC Classes  ?

  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • C12N 5/074 - Adult stem cells
  • C12N 9/22 - Ribonucleases
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells

79.

METHODS AND COMPOSITIONS FOR THE TREATMENT OF VASCULAR ANOMALIES

      
Application Number US2024054845
Publication Number 2025/101692
Status In Force
Filing Date 2024-11-07
Publication Date 2025-05-15
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Bischoff, Joyce E.
  • Holm, Annegret

Abstract

The technology described herein is directed to methods and compositions relating to the treatment of vascaular anomalies, e.g. hemangiomas, including the discovery that famesyltransferase and/or geranylgeranyltransferas inhibitors target pathways controlling the differentiation of hemangioma-derived stem cells.

IPC Classes  ?

  • A61K 31/4709 - Non-condensed quinolines containing further heterocyclic rings
  • A61K 31/138 - Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
  • A61K 31/366 - Lactones having six-membered rings, e.g. delta-lactones
  • A61K 31/435 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
  • A61K 31/573 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
  • A61P 9/00 - Drugs for disorders of the cardiovascular system

80.

ENDOTHELIAL CELL FACTORS AND METHODS THEREOF

      
Application Number US2024053916
Publication Number 2025/096798
Status In Force
Filing Date 2024-10-31
Publication Date 2025-05-08
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Zon, Leonard I.
  • Han, Tianxiao

Abstract

The technology described herein relates to compositions and methods of generating endothelial niche cells. Embodiments of the technology described herein comprise compositions, kits, vectors, and methods related to generating or engineering endothelial niche cells.

IPC Classes  ?

  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
  • A61K 35/44 - VesselsVascular smooth muscle cellsEndothelial cellsEndothelial progenitor cells

81.

METHODS AND COMPOSITIONS FOR IMMUNOSUPPRESSANT RESISTANT CELLULAR THERAPIES

      
Application Number US2024054075
Publication Number 2025/096907
Status In Force
Filing Date 2024-11-01
Publication Date 2025-05-08
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Wobma, Holly M.
  • Kean, Leslie S.

Abstract

PPIA PPIA gene resulting in a modified version of the gene product, cyclophilin A, in order to confer immunosuppressant drug resistance. Compositions comprising the engineered immune cells, and methods for treatment comprising administering the same are described herein.

IPC Classes  ?

  • C12N 15/10 - Processes for the isolation, preparation or purification of DNA or RNA
  • A61K 35/17 - LymphocytesB-cellsT-cellsNatural killer cellsInterferon-activated or cytokine-activated lymphocytes
  • C12N 5/078 - Cells from blood or from the immune system
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • A61P 35/00 - Antineoplastic agents
  • A61P 37/00 - Drugs for immunological or allergic disorders

82.

NANOBODY-DRUG ADDUCTS AND USES THEREOF

      
Application Number 18730142
Status Pending
Filing Date 2023-01-19
First Publication Date 2025-05-08
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Ploegh, Hidde L.
  • Liu, Xin
  • Pishesha, Novalia
  • Verhaar, Elisha

Abstract

Provided herein are conjugate molecules comprising an antibody or antibody fragment capable of binding polyclonal immnunoglobulins within a subject. Such conjugates are useful for recruiting immune cells of the subject to one or more cell types that are targeted by the conjugate. Also provided herein are compositions comprising the conjugates, including pharmaceutical compositions that may be administered to a subject, for such a purpose as to treat or prevent a disease.

IPC Classes  ?

  • A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
  • A61P 31/14 - Antivirals for RNA viruses
  • A61P 31/16 - Antivirals for RNA viruses for influenza or rhinoviruses
  • A61P 33/06 - Antimalarials
  • A61P 35/00 - Antineoplastic agents
  • C07K 16/10 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from viruses from RNA viruses
  • C07K 16/20 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans from protozoa
  • C07K 16/30 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants from tumour cells

83.

ENGINEERED SEMAPHORINS AND USES THEREOF

      
Application Number 18788401
Status Pending
Filing Date 2024-07-30
First Publication Date 2025-05-08
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Briscoe, David M.
  • Bielenberg, Diane
  • Wedel, Johannes

Abstract

Provided herein are methods and compositions comprising or using mutant semaphorin polypeptides.

IPC Classes  ?

  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • A61K 38/00 - Medicinal preparations containing peptides
  • A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection

84.

MEMBRANE FUSION AND IMMUNE EVASION BY THE SPIKE PROTEIN OF SARS-COV-2 DELTA VARIANT

      
Application Number 18684129
Status Pending
Filing Date 2022-08-16
First Publication Date 2025-05-01
Owner Children's Medical Center Corporation (USA)
Inventor Chen, Bing

Abstract

Provided herein, in some aspects, are methods for using the receptor-binidng domain (RBD) of the SARS-COV-2 S protein, including the the RBD-1, RBD-2, and/or RBD-3 regions, to identify therapeutics for the treatment of SARS-COV-2, developing a vaccine for the treatment or prevention of SARS-COV-2, and identifying a patient as being in need for a treatment for SARS-COV-2.

IPC Classes  ?

  • A61K 39/215 - Coronaviridae, e.g. avian infectious bronchitis virus
  • C07K 14/005 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from viruses

85.

DYNAMIC DISTORTION CORRECTION FOR MRI USING FID NAVIGATORS

      
Application Number 18687865
Status Pending
Filing Date 2022-08-19
First Publication Date 2025-05-01
Owner Children's Medical Center Corporation (USA)
Inventor
  • Wallace, Tess E.
  • Warfield, Simon K.
  • Afacan, Onur

Abstract

Described herein are various embodiments of techniques for controlling creation of a magnetic resonance (MR) image from a magnetic resonance imaging (MRI) scan of a subject using an MRI scanner, through real-time, dynamic control of the MRI scanner during the MRI scan and/or of processing during the MRI scan of acquired MR signals as part of generating MR data for creation of the MR image. More particularly, some embodiments described herein relate to identifying changes that occur in a magnetic field within an MRI scanner during a course of an MRI scan. In response to changes in the magnetic field, an MRI scanner may be adjusted to change a way in which additional MR data is captured and/or a processing of captured MR signals (that are to be used in generating an MR image) may be adjusted.

IPC Classes  ?

  • G01R 33/54 - Signal processing systems, e.g. using pulse sequences
  • G01R 33/56 - Image enhancement or correction, e.g. subtraction or averaging techniques

86.

PROMININ-1 PEPTIDE FOR TREATING LUNG INJURY

      
Application Number 18741486
Status Pending
Filing Date 2024-06-12
First Publication Date 2025-05-01
Owner Children's Medical Center Corporation (USA)
Inventor
  • Adini, Avner
  • Matthews, Benjamin

Abstract

Described herein are compositions and methods for treating a lung disorder associated with dysregulated VEGF signaling. The PR1P peptide (DRVQRQTTTVVA, SEQ ID NO: 1) and variants thereof are able to enhance VEGF signaling in the lungs and reduce lung cell apoptosis (e.g., induced by toxicity or injury), thus treating the disorder.

IPC Classes  ?

  • A61K 38/10 - Peptides having 12 to 20 amino acids
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 47/14 - Esters of carboxylic acids, e.g. fatty acid monoglycerides, medium-chain triglycerides, parabens or PEG fatty acid esters
  • A61P 11/00 - Drugs for disorders of the respiratory system
  • C07K 14/475 - Growth factorsGrowth regulators

87.

ABCB5-TARGETED STEM CELL REPROGRAMMING AND ANTI-AGING STRATEGIES

      
Application Number US2024052497
Publication Number 2025/090565
Status In Force
Filing Date 2024-10-23
Publication Date 2025-05-01
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Frank, Markus H.
  • Wilson, Brian
  • Frank, Alexander

Abstract

Methods and compositions for rejuvenating and reprogramming stem cells are disclosed. The methods involve administering to a subject an ABCB5 targeted composition comprising an anti-ABCB5 antibody conjugated to a therapeutic payload comprised of an epigenetic reprogramming factor or a nucleic acid encoding an epigenetic reprogramming factor in an effective amount to reprogram and rejuvenate ABCB5+ stem cells in the subject. The compositions include anti-ABCB5 antibody conjugated to a therapeutic payload comprising an epigenetic reprogramming factor or a nucleic acid encoding an epigenetic reprogramming factor.

IPC Classes  ?

  • A61K 47/68 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
  • C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
  • C12N 15/864 - Parvoviral vectors
  • A61K 47/54 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
  • A61K 9/127 - Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
  • A61K 9/51 - Nanocapsules
  • A61P 25/28 - Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
  • A61P 27/02 - Ophthalmic agents
  • C07K 16/22 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against growth factors
  • C12N 15/11 - DNA or RNA fragmentsModified forms thereof
  • A61K 47/69 - Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additivesTargeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit

88.

COMPOSITIONS AND METHODS RELATING TO ATP/P2X7R SIGNALING INHIBITION

      
Application Number 18835427
Status Pending
Filing Date 2023-02-13
First Publication Date 2025-04-24
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Fiorina, Paolo
  • Nasr, Moufida Ben

Abstract

Described herein are compositions, combinations, and methods relating to treating or preventing immune-related diseases, type 1 diabetes, transplant rejection, or lung fibrosis, or slowing or delaying aging processes or extending lifespan in a subject, including subjects with rs3751143.

IPC Classes  ?

  • C07D 498/04 - Ortho-condensed systems
  • A61K 31/138 - Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
  • A61K 31/166 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide having the carbon atom of a carboxamide group directly attached to the aromatic ring, e.g. procainamide, procarbazine, metoclopramide, labetalol
  • A61K 31/4015 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil having oxo groups directly attached to the heterocyclic ring, e.g. piracetam, ethosuximide
  • A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
  • A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
  • A61K 31/505 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim
  • A61K 31/53 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with three nitrogens as the only ring hetero atoms, e.g. chlorazanil, melamine
  • A61K 31/7076 - Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
  • C07C 217/64 - Compounds containing amino and etherified hydroxy groups bound to the same carbon skeleton having etherified hydroxy groups bound to carbon atoms of at least one six-membered aromatic ring and amino groups bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings of the same carbon skeleton with amino groups linked to the six-membered aromatic ring, or to the condensed ring system containing that ring, by carbon chains further substituted by singly-bound oxygen atoms
  • C07D 235/18 - BenzimidazolesHydrogenated benzimidazoles with aryl radicals directly attached in position 2
  • C07D 239/48 - Two nitrogen atoms

89.

COMPOSITIONS AND METHODS FOR PREVENTION AND TREATMENT OF GENETIC DISEASE

      
Application Number US2024051733
Publication Number 2025/085607
Status In Force
Filing Date 2024-10-17
Publication Date 2025-04-24
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor Fauza, Dario O.

Abstract

Methods and compositions for the transamniotic delivery of mRNA encoding alpha- 1 antitrypsin protein to a fetus for the prenatal treatment of alpha- 1 antitrypsin deficiency.

IPC Classes  ?

  • A61K 31/7105 - Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
  • A61K 38/57 - Protease inhibitors from animalsProtease inhibitors from humans
  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61P 11/00 - Drugs for disorders of the respiratory system
  • C07K 14/81 - Protease inhibitors

90.

METHODS OF MAKING AND USING IPSC-DERIVED MURAL PROGENITOR CELLS VIA ACTIVATION OF NKX3.1

      
Application Number US2024050882
Publication Number 2025/080915
Status In Force
Filing Date 2024-10-11
Publication Date 2025-04-17
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Melero-Martin, Juan M.
  • Lee, Umji
  • Wang, Kai

Abstract

inter aliainter aliainter alia, are methods maturing the iMPCs into functional mural cell subtypes, including smooth muscle cells, pericytes, and fibroblasts, as well as methods of increasing vascular development, angiogenesis, and cell junction via the iMPCs. The iMPCs mediate the formation of functional vessels when implanted with endothelial cells (ECs); thus, methods of administering iMPCs and ECs and methods of modeling vascular diseases (e.g., a 3D vascular organoid (VO)) and therapeutic vascularization comprising administering iMPCs and EC are also described herein.

IPC Classes  ?

  • C07K 14/47 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans from vertebrates from mammals
  • C12N 5/074 - Adult stem cells
  • C12N 5/071 - Vertebrate cells or tissues, e.g. human cells or tissues
  • C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells

91.

METHODS AND COMPOSITIONS FOR THE TREATMENT OF THROMBOCYTOPENIA

      
Application Number 18911620
Status Pending
Filing Date 2024-10-10
First Publication Date 2025-04-17
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Bekendam, Roelof H.
  • Stone, Andrew P.
  • Machlus, Kellie Rae
  • Italiano, Jr., Joseph Edward

Abstract

This disclosure relates to methods and compositions for the treatment of thrombocytopenia.

IPC Classes  ?

  • A61K 31/454 - Non-condensed piperidines, e.g. piperocaine containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. pimozide, domperidone
  • A61K 31/501 - PyridazinesHydrogenated pyridazines not condensed and containing further heterocyclic rings
  • A61K 31/55 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
  • A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
  • A61P 7/00 - Drugs for disorders of the blood or the extracellular fluid

92.

VACCINE COMPOSITIONS COMPRISING SIALIC ACID BINDING DOMAIN (SBD) PROTEINS AND USE THEREOF TO ENHANCE IMMUNE RESPONSE

      
Application Number US2024050680
Publication Number 2025/080776
Status In Force
Filing Date 2024-10-10
Publication Date 2025-04-17
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Malley, Richard
  • Zhang, Fan
  • Lu, Yingjie

Abstract

Aspects of the invention described herein relate to a vaccine composition comprising at an antigenic polysaccharide and an immunomodulatory amount of a sialic acid binding moiety, wherein the sialic acid binding moiety comprises sialic acid binding domain (SBD) which is not fused to an antigenic polypeptide.

IPC Classes  ?

  • C07K 14/315 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci
  • A61K 39/39 - Medicinal preparations containing antigens or antibodies characterised by the immunostimulating additives, e.g. chemical adjuvants
  • A61P 37/04 - Immunostimulants
  • A61K 47/64 - Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent

93.

PROTEIN ANTIGENS THAT PROVIDE PROTECTION AGAINST PNEUMOCOCCAL COLONIZATION AND/OR DISEASE

      
Application Number 18799550
Status Pending
Filing Date 2024-08-09
First Publication Date 2025-04-10
Owner CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Malley, Richard
  • Lu, Yingjie
  • Zhang, Fan

Abstract

The present application is generally directed to novel pneumococcal polypeptide antigens and nucleic acids encoding such antigens, and immunogenic compositions comprising such antigens for treating and preventing pneumococcal infection. The present invention further provides method of using the antigens to elicits an immune response (e.g., IL-17A response, a T cell-mediated and/or B-cell-mediated immune responses). The present invention also provides methods of prophylaxis and/or treatment of pneumococcal-mediated diseases, such as sepsis, comprising administering an immunogenic composition including one or more of a combination of pneumococcal antigens or functional fragments thereof as disclosed herein. In some embodiments, one or more pneumococcal antigens can be present in a polysaccharide conjugate. The compositions induce an anti-pneumococcus immune response when administered to a mammal. The compositions can be used prophylactically to vaccinate an individual and/or therapeutically to induce a therapeutic immune response to an infected individual.

IPC Classes  ?

  • A61K 39/09 - Streptococcus
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • C07K 14/315 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from bacteria from Streptococcus (G), e.g. Enterococci

94.

METHODS AND COMPOSITIONS RELATING TO CHONDRISOMES FROM BLOOD PRODUCTS

      
Application Number 18404389
Status Pending
Filing Date 2024-01-04
First Publication Date 2025-04-10
Owner The Children's Medical Center Corporation (USA)
Inventor
  • Von Maltzahn, Geoffrey A.
  • Milwid, John Miles
  • Mee, Michael Travis
  • Rubens, Jacob Rosenblum
  • Chess, David
  • Trudeau, Kyle Marvin
  • Mahdaviani, Kiana
  • Feala, Jacob
  • Mccully, James D.
  • Cowan, Douglas B.

Abstract

Therapeutic chondrisome and mitoplast compositions and related methods are described.

IPC Classes  ?

95.

METHYL-BRANCHED LIPOSOMES FOR SUSTAINED DRUG DELIVERY

      
Application Number US2024049440
Publication Number 2025/075976
Status In Force
Filing Date 2024-10-01
Publication Date 2025-04-10
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Kohane, Daniel, S.
  • Li, Yang

Abstract

The present disclosure provides compositions comprising a liposome encapsulating a therapeutic agent or a diagnostic agent. The liposome comprises a lipid having a branched molecular structure (i.e., a lipid of Formula (I)). The compositions thus provide a means of controlled and prolonged delivery of therapeutic and diagnostic agents. The present disclosure further provides kits comprising the compositions, methods of treating or preventing a disease or disorder, and methods of preparing the compositions.

IPC Classes  ?

  • A61K 31/6615 - Compounds having two or more esterified phosphorus acid groups, e.g. inositol triphosphate, phytic acid
  • A61K 31/14 - Quaternary ammonium compounds, e.g. edrophonium, choline
  • C07F 9/09 - Esters of phosphoric acids

96.

COMPOSITIONS AND METHODS FOR SELECTING A SUBJECT FOR INFLAMMATORY BOWEL DISEASE TREATMENT

      
Application Number US2024050050
Publication Number 2025/076414
Status In Force
Filing Date 2024-10-04
Publication Date 2025-04-10
Owner
  • THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
  • ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI (USA)
Inventor
  • Snapper, Scott
  • Collen, Lauren
  • Schadt, Eric
  • Argmann, Carmen
  • Beckmann, Noam

Abstract

The disclosure provides methods for predicting response in a subject having IBD to anti-IL12 and/or anti-IL23-based therapies, and selecting an effective therapy.

IPC Classes  ?

  • A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
  • A61P 1/04 - Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
  • A61P 37/00 - Drugs for immunological or allergic disorders
  • G01N 33/543 - ImmunoassayBiospecific binding assayMaterials therefor with an insoluble carrier for immobilising immunochemicals
  • G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids

97.

CELLS WITH INCREASED IMMUNO-REGULATORY PROPERTIES AND METHODS FOR THEIR USE AND MANUFACTURE

      
Application Number 18632967
Status Pending
Filing Date 2024-04-11
First Publication Date 2025-03-27
Owner The Children's Medical Center Corporation (USA)
Inventor Fiorina, Paolo

Abstract

The present invention is directed to compositions and methods to increase the expression of PD-L1 and/or IDO-1 in a population of cells, the modulated cells expressing increased PD-L1 and/or IDO-1, and methods related to the immunosuppressive effects obtained by cells expressing increased PD-L1 and/or IDO-1.

IPC Classes  ?

  • C12N 5/0789 - Stem cellsMultipotent progenitor cells
  • A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61K 38/44 - Oxidoreductases (1)
  • A61K 39/00 - Medicinal preparations containing antigens or antibodies
  • A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
  • A61P 37/00 - Drugs for immunological or allergic disorders
  • C07K 14/555 - Interferons [IFN]
  • C07K 14/705 - ReceptorsCell surface antigensCell surface determinants
  • C12N 5/0783 - T cellsNK cellsProgenitors of T or NK cells
  • C12N 9/02 - Oxidoreductases (1.), e.g. luciferase

98.

METHODS OF VASCULAR CELL THERAPY AND ENDOTHELIAL CELL ENGRAFTMENT USING MITOCHONDRIA

      
Application Number US2024047754
Publication Number 2025/064863
Status In Force
Filing Date 2024-09-20
Publication Date 2025-03-27
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor
  • Melero-Martin, Juan M.
  • Lin, Ruei-Zeng

Abstract

inter aliainter aliainter alia, are methods vascular cell therapy, promoting blood vessel formation, and increasing vascular development, angiogenesis, and cell junction via administration of a population of mitoAT-ECs.

IPC Classes  ?

  • A61K 35/28 - Bone marrowHaematopoietic stem cellsMesenchymal stem cells of any origin, e.g. adipose-derived stem cells
  • C12N 5/077 - Mesenchymal cells, e.g. bone cells, cartilage cells, marrow stromal cells, fat cells or muscle cells
  • A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
  • C12N 5/07 - Animal cells or tissues
  • A61P 9/00 - Drugs for disorders of the cardiovascular system

99.

MATERIALS WITH IMPROVED PROPERTIES

      
Application Number 18952686
Status Pending
Filing Date 2024-11-19
First Publication Date 2025-03-20
Owner
  • Massachusetts Institute of Technology (USA)
  • The Children’s Medical Center Corporation (USA)
Inventor
  • Vegas, Arturo J.
  • Doloff, Joshua C.
  • Veiseh, Omid
  • Langer, Robert S.
  • Anderson, Daniel G.

Abstract

Products, such as devices, prostheses, and materials, whose surfaces have been modified in order to impart beneficial properties to these products are disclosed. The surface-modified products have improved biocompatibility compared to a corresponding product that lacks the modification. Following implantation in a subject, the surface-modified products induce a lower foreign-body response, compared to a corresponding unmodified product.

IPC Classes  ?

  • A61L 27/38 - Animal cells
  • A61K 9/00 - Medicinal preparations characterised by special physical form
  • A61K 35/12 - Materials from mammalsCompositions comprising non-specified tissues or cellsCompositions comprising non-embryonic stem cellsGenetically modified cells
  • A61K 35/36 - SkinHairNailsSebaceous glandsCerumenEpidermisEpithelial cellsKeratinocytesLangerhans cellsEctodermal cells
  • A61K 35/39 - PancreasIslets of Langerhans
  • A61K 38/02 - Peptides of undefined number of amino acidsDerivatives thereof
  • A61L 27/04 - Metals or alloys
  • A61L 27/10 - Ceramics or glasses
  • A61L 27/16 - Macromolecular materials obtained by reactions only involving carbon-to-carbon unsaturated bonds
  • A61L 27/18 - Macromolecular materials obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds
  • A61L 27/20 - Polysaccharides
  • A61L 27/34 - Macromolecular materials
  • A61L 27/54 - Biologically active materials, e.g. therapeutic substances
  • A61L 27/56 - Porous or cellular materials
  • A61L 29/14 - Materials characterised by their function or physical properties
  • C08J 7/12 - Chemical modification

100.

COMPOSITIONS AND METHODS FOR PREVENTION AND TREATMENT OF GENETIC DISEASE

      
Application Number 18890368
Status Pending
Filing Date 2024-09-19
First Publication Date 2025-03-13
Owner THE CHILDREN'S MEDICAL CENTER CORPORATION (USA)
Inventor Fauza, Dario O.

Abstract

Methods and compositions for the transamniotic delivery of mRNA encoding cystic fibrosis transmembrane conductance regulator protein or surfactant protein B to a fetus for the prenatal treatment of cystic fibrosis or a surfactant protein B deficiency.

IPC Classes  ?

  • A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
  • A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
  • A61P 11/00 - Drugs for disorders of the respiratory system
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