Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in pediatric patients. Certain aspects of the invention relate to identifying one or more biomarkers associated with septic shock in pediatric patients in combination with one or more endothelial-derived biomarkers, receiving a dataset comprising biomarker concentrations, wherein the dataset is from a sample obtained from a pediatric patient having at least one indication of septic shock, then determining whether the biomarker concentrations of each of the at least one biomarkers are greater than one or more pre-determined cut-off biomarker concentration, wherein the level of said biomarker correlates with a predicted outcome.
G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
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
2.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF SEPSIS
Disclosed are methods of treating sepsis in an individual in need thereof via administration of a therapeutically effective amount of a humanin protein, or an analog thereof, to the individual. In one aspect, the methods may comprise administration of the humanin analog colivelin for reducing lung, liver and kidney injury and systemic inflammation after an infection.
A61K 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
Disclosed are vaccine compositions, in particular, polyvalent icosahedral compositions for presentation of an HA1 influenza antigen. The disclosed compositions may contain an S particle comprising a norovirus (NoV) S domain and an HA1 influenza antigen, which may be linked via a linker protein domain operatively connected to the norovirus S domain and an influenza antigen. Fusion proteins for producing the vaccine compositions, and methods of using the disclosed vaccine composition are also provided.
Disclosed herein are organoid models of the blood-brain barrier and methods for making the same from pluripotent stem cells. These organoids exhibit the complex organization of cells including neurons, endothelial cells, glial cells, and pericytes resembling the natural blood-brain barrier structure. These organoids may be used for studying the functions of the blood-brain barrier and cerebrovascular disorders.
Disclosed are methods for the enhancement of nucleic acid delivery systems. The methods may employ treatment with a compound and/or an RNAi molecule in combination with a nucleic acid to improve nucleic acid uptake into a cell. In particular, the disclosed methods may be useful for improved gene therapy techniques.
A61K 31/132 - Amines, e.g. amantadine having two or more amino groups, e.g. spermidine, putrescine
A61K 31/265 - Esters, e.g. nitroglycerine, selenocyanates of carbonic, thiocarbonic or thiocarboxylic acids, e.g. thioacetic acid, xanthogenic acid, trithiocarbonic acid
A61K 31/352 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. cannabinols, methantheline
A61K 31/395 - Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
A61K 31/4709 - Non-condensed quinolines containing further heterocyclic rings
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 31/565 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids not substituted in position 17 beta by a carbon atom, e.g. oestrane, oestradiol
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 31/7048 - Compounds having saccharide radicals and heterocyclic rings having oxygen as a ring hetero atom, e.g. leucoglucosan, hesperidin, erythromycin, nystatin
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
A61K 31/713 - Double-stranded nucleic acids or oligonucleotides
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 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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 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
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C12N 15/11 - DNA or RNA fragmentsModified forms thereof
C12N 15/85 - Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
Disclosed herein are improved pluripotent stem cell-derived biliary organoids, having extrahepatic cholangiocytes. In various embodiments, the biliary organoids can additionally include intrahepatic cholangiocytes. Also disclosed are methods of producing the biliary organoids having extrahepatic cholangiocytes. The disclosure also relates to methods of studying or treating a biliary-related disease or disorder using the described biliary organoids, including surgical methods such as in transplants, engraftments, ligations, and/or bililary bypass. The disclosure also includes surgical implants for biliary bypass. The disclosure additional describes methods of producing tubular organoids and cell culture devices for performing said methods.
The instant disclosure relates to methods which employ the detection of ADP-D-glycero-β-D-manno-heptose (ADP-heptose) in a biological sample obtained from an individual. In certain aspects, the methods comprise administering a treatment to an individual in which ADP-heptose is detected. The methods may further comprise determining whether the individual has clonal hematopoiesis of indeterminate potential (CHIP) and circulating ADP-heptose.
The present invention provides methods relating to the discovery of olfactomedin 4 (OLFM4) as a biomarker for acute kidney injury (AKI) and need for renal replacement therapy, and further as a biomarker for responsiveness to the furosemide stress test (FST). The methods described here are useful in clinical decision support and personalized therapy for AKI, as well as for clinical trial design.
Disclosed are in vitro transcribed (IVT) messenger RNAs (mRNAs) comprising a polynucleotide that encodes human TJP2 gene, a 5' terminal cap, and a poly-adenylation (poly- A) tail at the 3' terminal end. In aspects, the polynucleotide is substantially free of uridine bases and comprises pseudouridine bases. Methods, compositions and uses employing the IVT mRNA for treating or preventing progressive familial intrahepatic Cholestasis type IV (PFIC type 4) in a patient in need thereof is also disclosed.
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 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 31/7088 - Compounds having three or more nucleosides or nucleotides
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 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A61K 31/7115 - Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
A61K 31/7125 - Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
A61K 31/713 - Double-stranded nucleic acids or 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
C12N 15/113 - Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
C12N 15/67 - General methods for enhancing the expression
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
Disclosed herein are improved pluripotent stem cell-derived multi-zonal liver organoids, having two or more hepatocyte subpopulations, and methods of producing the same, by coculturing a first human liver organoid (HLO) with a bilirubin-treated second HLO, and contacting the co-cultured HLOs with bilirubin for a period of time sufficient for the co-cultured HLOs to self-assemble and fuse into interconnected dual organoids. The first HLO can be an ascorbateenriched HLO, such as a functional L-gulonolactone oxidase (GULO)-expressing HLO. In particular embodiments, the hepatocyte subpopulations can include zone 1 and zone 3 hepatocyte subpopulations, or zone 1, zone 2, and zone 3 hepatocyte subpopulations. Also disclosed are methods of studying or treating a liver-related disease or disorder using the described multi-zonal liver organoids.
Disclosed herein are organoid compositions and methods of use thereof for the treatment of intestinal injury and damage. The methods involve the intraluminal administration of cellular compositions derived from stem cell-derived organoids, which comprise both epithelial and mesenchymal components. The administered cells show robust engraftment into the appropriate regions of the recipient intestinal tissue. Integration of the multiple cell types present from the stem cell-derived organoids results in a more complete healing of the intestinal injury.
Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in pediatric patients. Certain aspects of the disclosure relate to identifying one or more biomarkers associated with septic shock in pediatric patients in combination with one or more endothelial-derived biomarkers, receiving a sample from a pediatric patient having at least one indication of septic shock, then quantifying from the sample an amount of said biomarkers, wherein the level of said biomarker correlates with a predicted outcome.
G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
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
G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients
14.
METHODS FOR EVALUATING A SUBJECT FOR FRAGILE X SYNDROME
Methods for determining methylation status and CGG repeat extent in a untranslated region (UTR) of Fragile X messenger riboprotein gene 1 (FMR1) in a subject comprise (a) isolating high molecular weight DNA from a whole blood sample; (b) enriching the isolated DNA for an UTR of FMR1 with Cas9-assisted gene-targeted cleavage of regions of interest in the FMR1 gene and ligation of sequencing adapters to the cleaved regions of interest; and (c) sequencing the regions of interest to determine a cumulative number of methylated CGG repeats in the regions of interest and a cumulative number of CGG repeats in the regions of interest. Methods for evaluating a subject for Fragile X Syndrome further comprise (d) relating the cumulative number of CGG repeats and the cumulative number of methylated CGG repeats to a clinical phenotype of Fragile X Syndrome.
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
C12Q 1/6874 - Methods for sequencing involving nucleic acid arrays, e.g. sequencing by hybridisation [SBH]
B.G. Negev Technologies and Applications Ltd., at Ben-Gurion University (Israel)
Mor Research Applications Ltd. (Israel)
Inventor
Gutmark-Little, Iris
Gutmark, Ephraim
Cavari, Yuval
Katoshevski, David
Abstract
The present disclosure relates to a device for treating obstructed airways. Specifically, the present disclosure relates to a device for treating the symptoms of diseases that cause persistent airflow limitation by applying air pressure oscillations and acoustic vibrations to the airways of a patient during treatment. The device may be used in a system that allows in-home treatment under remote supervision of a physician.
G16H 20/30 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to physical therapies or activities, e.g. physiotherapy, acupressure or exercising
G16H 40/67 - ICT specially adapted for the management or administration of healthcare resources or facilitiesICT specially adapted for the management or operation of medical equipment or devices for the operation of medical equipment or devices for remote operation
17.
METHODS AND COMPOSITIONS FOR TREATMENT OF HYDROCEPHALUS
Disclosed are compositions and methods for the treatment of hydrocephalus, for example pediatric hydrocephalus. The disclosed methods employ a non-surgical tool that may be used to control CSF production rate by inducing choroid plexus-specific apoptosis. In aspects, the non-surgical tools employ an AAV vector, a promoter, a suicide gene, and a suicide gene activator to achieve targeted choroid-plexus ablation and subsequent reduction of CSF volume.
A61K 31/436 - 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 oxygen as a ring hetero atom, e.g. rapamycin
A61P 25/00 - Drugs for disorders of the nervous system
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
Formulations for alleviating a sleep disorder or improving sleep in a subject comprise (a) an immediate-release component comprising at least one of melatonin and a melatonin receptor agonist in an amount effective to shift sleep timing in a subject to an earlier time than the subject would experience in the absence of the melatonin or melatonin receptor agonist, (b) a sleep-inducing hypnotic in an amount effective to induce sleep in the subject, and (c) a delayed-release component delaying release of the sleep-inducing hypnotic. The delayed-release component is operable to delay release of the hypnotic until the melatonin or melatonin receptor agonist has exhibited its effect to shift sleep timing in a subject. The formulations are adapted for oral administration and are useful in methods of alleviating a sleep disorder or improving sleep in a subject.
A61K 31/437 - 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 five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 9/24 - Layered or laminated unitary dosage forms
A61K 9/48 - Preparations in capsules, e.g. of gelatin, of chocolate
A61K 31/165 - Amides, e.g. hydroxamic acids having aromatic rings, e.g. colchicine, atenolol, progabide
A61K 31/195 - Carboxylic acids, e.g. valproic acid having an amino group
A61K 31/335 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
A61K 31/343 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide condensed with a carbocyclic ring, e.g. coumaran, bufuralol, befunolol, clobenfurol, amiodarone
A61K 31/4045 - Indole-alkylaminesAmides thereof, e.g. serotonin, melatonin
A61K 31/4168 - 1,3-Diazoles having a nitrogen atom attached in position 2, e.g. clonidine
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Health care services, namely, providing prenatal and postnatal health care services through community health workers, nurses, dietitians, and social workers; Charitable services, namely, providing health care services in the nature of prenatal and postnatal health care services to mothers and babies impacted by racial health disparities
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Conducting quality assurance surveys in hospitals to determine service quality Providing medical information to expecting mothers in the form of reports in the field of maternal equity efforts of birthing hospitals; Providing healthcare information; Providing medical information in the field of maternal equity efforts and assessments of birthing hospitals; Providing medical information via a website; Clinical medical practice consultation services; Clinical medical consultation services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Conducting quality assurance surveys in hospitals to determine service quality Providing medical information to expecting mothers in the form of reports in the field of maternal equity efforts of birthing hospitals; Providing healthcare information; Providing medical information in the field of maternal equity efforts and assessments of birthing hospitals; Providing medical information via a website; Clinical medical practice consultation services; Clinical medical consultation services
45 - Legal and security services; personal services for individuals.
Goods & Services
Charitable services, namely, providing emotional support services for Black women by means of arranging and hosting events, workshops, and educational programs; Organizing and conducting support groups in the field of maternal equity for Black mothers and babies
41 - Education, entertainment, sporting and cultural services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Promoting the interests of mothers and babies impacted by racial health disparities by means of public advocacy; Promoting public awareness of maternal and infant health care by means of public advocacy; Promoting public interest and awareness of maternal and infant health equity Education services, namely, providing workshops and training programs in the field of prenatal and postnatal health; Education services, namely, providing professional learning collaboratives in the field of improving prenatal care quality and outcomes; Providing training in the field of maternal well-being and health as part of a wellness program; Community outreach services provided to pregnant women, namely, providing information, news and commentary in the field of current events relating to pregnant women; Educational services, namely, conducting conferences and workshops in the field of maternal and infant health equity and distribution of educational materials in connection therewith Health care services, namely, providing prenatal and postnatal health care services through community health workers, nurses, dietitians, and social workers; Charitable services, namely, providing health care services in the nature of prenatal and postnatal health care services to mothers and babies impacted by racial health disparities; Advisory services relating to health care; Providing breastfeeding information; Breastfeeding counseling services
41 - Education, entertainment, sporting and cultural services
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Promoting the interests of mothers and babies impacted by racial health disparities by means of public advocacy; Promoting public awareness of maternal and infant health care by means of public advocacy; Promoting public interest and awareness of maternal and infant health equity Education services, namely, providing workshops and training programs in the field of prenatal and postnatal health; Education services, namely, providing professional learning collaboratives in the field of improving prenatal care quality and outcomes; Providing training in the field of maternal well-being and health as part of a wellness program; Community outreach services provided to pregnant women, namely, providing information, news and commentary in the field of current events relating to pregnant women; Educational services, namely, conducting conferences and workshops in the field of maternal and infant health equity and distribution of educational materials in connection therewith Health care services, namely, providing prenatal and postnatal health care services through community health workers, nurses, dietitians, and social workers; Charitable services, namely, providing health care services in the nature of prenatal and postnatal health care services to mothers and babies impacted by racial health disparities; Advisory services relating to health care; Providing breastfeeding information; Breastfeeding counseling services
45 - Legal and security services; personal services for individuals.
Goods & Services
Charitable services, namely, providing emotional support services for Black women by means of arranging and hosting events, workshops, and educational programs; Organizing and conducting support groups in the field of maternal equity for Black mothers and babies
26.
METHODS FOR PREDICTING AND TREATING CHRONIC LUNG ALLOGRAFT DYSFUNCTION
Disclosed are methods for treating a lung condition selected from one or more of lung allograft dysfunction, rejection, or failure, in an individual who has undergone a lung transplant, comprising a) detecting a biomarker; b) quantifying a biomarker level; and c) comparing the level of a biomarker to a control value; wherein a deviation in a level of biomarker indicates that said individual is likely to develop the lung condition.
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
27.
BIOMETRIC-BASED VIDEO GAME CONTROL SYSTEM AND METHODS THEREOF
Systems and methods for detecting biological characteristics of a wearer of a biometric sensor unit and converting the biological characteristics into simulated gaming control signals. These simulated control signals can correspond to any type of conventional gaming controller input. By providing video game control seeking to teach breathing control and heart rate lowering techniques, physiological stress indicators of the wearer can be reduced.
A63F 13/212 - Input arrangements for video game devices characterised by their sensors, purposes or types using sensors worn by the player, e.g. for measuring heart beat or leg activity
A63F 13/235 - Input arrangements for video game devices for interfacing with the game device, e.g. specific interfaces between game controller and console using a wireless connection, e.g. infrared or piconet
A63F 13/24 - Constructional details thereof, e.g. game controllers with detachable joystick handles
28.
MRNA-LIPID NANOPARTICLE IMMUNE MODULATORS AGAINST ALLERGIC AND INFLAMMATORY DISEASES
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA (USA)
CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
Weissman, Drew
Rothenberg, Marc
Rochman, Yrina
Abstract
Provided are compositions comprising mRNA molecules encoding antigens, such as allergens and autoantigens, and methods of use thereof, optionally in combination with an mTOR inhibitor, to prevent or reduce allergic responses or to promote tolerance to the encoded antigens.
Disclosed are methods for treating an individual at risk for developing bronchiolitis obliterans syndrome (BOS) after hematopoietic stem cell transplant (HSCT), comprising a) detecting a biomarker; b) quantifying a biomarker level; and c) comparing the level of a biomarker to a control value; wherein a deviation in a level of biomarker indicates that said individual is likely to develop the lung condition.
A system and method for identifying genomic regions with higher fragmentation rates than the local and global backgrounds as part of diagnosing early stage cancer is provided. The method includes steps of: de-novo characterizing genome-wide cell-free DNA fragmentation regions with higher fragmentation rates than the local and global backgrounds from whole-genome sequencing by weighing the fragment coverages in each region by a ratio of average fragment sizes in the region versus that in the whole chromosome to generate a score; and identifying DNA fragmentation regions of interest based upon comparing the score with a threshold. The system and method can utilize identified DNA fragmentation hotspots for the detection and localization of multiple early-stage cancers (or certain other non-malignant disease).
G16B 20/00 - ICT specially adapted for functional genomics or proteomics, e.g. genotype-phenotype associations
C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
G16B 30/00 - ICT specially adapted for sequence analysis involving nucleotides or amino acids
G16B 40/00 - ICT specially adapted for biostatisticsICT specially adapted for bioinformatics-related machine learning or data mining, e.g. knowledge discovery or pattern finding
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
31.
ENZYME-LINKED IMMUNOSORBENT ASSAY (ELISA) FOR ANTI-BSEP AUTOANTIBODY
Methods of detecting the presence and quantity of anti-BSEP antibodies in a biological sample from a subject include contacting the biological sample with one or more BSEP peptides to form an antibody-BSEP peptide complex, and detecting the amount of the antibody-BSEP peptide complex in the biological sample. The one or more BSEP peptides may include one or more of a full length BSEP peptide (SEQ ID NO:1), and/or one or more epitopes of BSEP.
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
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
G01N 33/543 - ImmunoassayBiospecific binding assayMaterials therefor with an insoluble carrier for immobilising immunochemicals
The present technology is directed to a method of treating a cancer, or multiple cancers, comprising administering to said individual one or more compounds selected from a VAV3-inhibitor such as IODVA1, followed by administration of a selective estrogen receptor modulator (SERM) and/or selective estrogen receptor degrader (SERD), to an individual in need thereof. In embodiments, the administration may be concurrently with or following IODVA1 administration.
A61K 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61K 31/138 - Aryloxyalkylamines, e.g. propranolol, tamoxifen, phenoxybenzamine
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 31/565 - Compounds containing cyclopenta[a]hydrophenanthrene ring systemsDerivatives thereof, e.g. steroids not substituted in position 17 beta by a carbon atom, e.g. oestrane, oestradiol
A61P 35/02 - Antineoplastic agents specific for leukemia
33.
INTEIN TECHNOLOGY TO DELIVER ABCA3 LIPID TRANSPORTER FOR ABCA3 DEFICIENCY
In order to develop a therapeutic strategy to rescue ABCA3 deficiency caused by a splicing mutation of ABCA3, we have designed an experimental scheme using CRISPR/Cas9 to delete a loxP site located at an intronic region of ABCA3 flox mice (Sftpc-Cre;Abca3flox/flox). The deletion of this loxP site extended survival of the mice by only a week, probably due to insufficient re-ligation efficiency after the CRISPR/Cas9 deletion. An alternative approach to rescuing ABCA3 deficiency caused by mutations in ABCA3, including coding or non-coding (e.g., intronic) mutations, is to deliver intact ABCA3 into lung alveolar type II cells using AAV (e.g., AAV6.2FF), a clinically approved delivery system. Due to the large size of ABCA3 (1704 amino acids), it is technically impossible to package full length ABCA3 cDNA into an AAV virus. However, a recent technological advance using intein makes it possible to split ABCA3 cDNA into two fragments for separate AAV delivery to the cells wherein the translated ABCA3 protein fragments would recombine into full length ABCA3 protein.
Disclosed are compositions, in particular, organoid compositions, derived from more than one donor cell. Further disclosed are methods of making compositions, for example, organoid compositions, that comprise a differentiated cell population derived from more than one donor cell. Donor cells may include, for example, a precursor cell such as an embryonic stem cell or other precursor cell. The disclosed methods use synchronization conditions to produce a synchronized pooled-precursor cell population, which may then be differentiated into an organoid composition. Methods of using the compositions are also disclosed.
The disclosure relates to methods for pain management in the perioperative context, particularly through the use of one or more biomarkers such as the DNA methylation status in genes of the PARK16 locus.
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
A61K 31/245 - Amino benzoic acid types, e.g. procaine, novocaine
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 31/7068 - 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 having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
36.
METHODS AND COMPOSITIONS FOR TREATMENT OF MEDULLOBLASTOMA
Disclosed are methods of treating a medulloblastoma (MB) tumor in an individual in need thereof, comprising administering to the individual a composition comprising one or more of a nucleic acid having specific binding to a SOX11 mRNA, a nucleic acid having specific binding to an HNRNPH1 mRNA and/or a nucleic acid having specific binding to a MYC enhancer region. In aspects, the nucleic acid has specific binding to a MYC enhancer region and impedes binding of one or both of a SOX11 protein and a HNRNPH1 protein to the MYC enhancer region. CRISPRi and/or CRISPR methods may be used for delivery of the nucleic acids contemplated herein.
C12Q 1/6886 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material for cancer
37.
METHOD OF MAKING IN VIVO HUMAN SMALL INTESTINE ORGANOIDS FROM PLURIPOTENT STEM CELLS
Disclosed are methods for making a vascularized hollow organ derived from human intestinal organoid (HIOs). The HIOs may be obtained from human embryonic stem cells (ESC's) and/or induced pluripotent stem cells (iPSCs), such that the HIO forms mature intestinal tissue. Also disclosed are methods for making a human intestinal tissue containing a functional enteric nervous system (ENS).
16 - Paper, cardboard and goods made from these materials
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Printed educational publications, namely, hand-outs and workbooks in the fields of emotional regulation Health care services, namely, group therapy program for children and teens experiencing emotion dysregulation; Mental health therapy services
The present invention provides compounds for use as inhibitors of BCL2A1/BFL1 proteins and related methods, including methods of therapy which may include use in treating a disease or disorder characterized by inappropriate BCL2A1/BFL1 expression and/or activity. In aspects, the disease or disorder is chorioamnionitis, an autoimmune disease or allergic disorder, or a cancer. Also provided are methods for attenuating a pathologic immune response.
A61K 31/437 - 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 five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/5365 - 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 oxygen as the ring hetero atoms, e.g. 1,2-oxazines ortho- or peri-condensed with heterocyclic ring systems
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES (USA)
KUROME THERAPEUTICS, INC. (USA)
Inventor
Hoyt, Scott, Bryan
Starczynowski, Daniel, T.
Thomas, Craig, Joseph
Rosenbaum, Jan, Susan
Abstract
Some embodiments of the disclosure include compounds of Formula (I) and Formula (la), and compositions thereof, e.g., pharmaceutical compositions, which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the compounds (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
Disclosed herein are methods of transferring exogenous material using platelets. The methods comprise administering the platelets with the exogenous material to a recipient. The platelets may be obtained by directly contacting the platelets, or contacting megakaryocytes that produce the platelets.
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
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
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
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C12N 5/078 - Cells from blood or from the immune system
42.
THROMBIN AND FIBRINOGEN AS TARGETS FOR ATOPIC DERMATITIS DIAGNOSIS AND TREATMENT
Methods for diagnosing atopic dermatitis (AD) by using thrombin and/or fibrinogen as biomarkers to determine the presence or severity of AD. Also provided are methods for treating AD by administering to a subject an effective amount of a thrombin inhibitor.
A61K 38/58 - Protease inhibitors from animalsProtease inhibitors from humans from leeches, e.g. hirudin, eglin
A61K 9/00 - Medicinal preparations characterised by special physical form
A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
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
THE USA, AS REPRESENTEDBY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES (USA)
KUROME THERAPEUTICS, Inc. (USA)
Inventor
Hoyt, Scott Bryan
Thomas, Craig Joseph
Starczynowski, Daniel T.
Rosenbaum, Jan Susan
Gracia Maldonado, Gabriel
Abstract
Some embodiments of the invention include inventive compounds (e.g., compounds of Formula (I), (II), or (III)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
Some embodiments of the invention include inventive compounds (e.g., compounds of Formula (I), (II), or (III)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
A61K 31/439 - 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 forming part of a bridged ring system, e.g. quinuclidine
A61K 31/4439 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with nitrogen as a ring hetero atom, e.g. omeprazole
A61K 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61K 31/451 - Non-condensed piperidines, e.g. piperocaine having a carbocyclic ring directly attached to the heterocyclic ring, e.g. glutethimide, meperidine, loperamide, phencyclidine, piminodine
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 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
A61K 31/519 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
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 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
A61K 31/7068 - 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 having oxo groups directly attached to the pyrimidine ring, e.g. cytidine, cytidylic acid
Disclosed are methods for identifying and/or treating an individual having, or likely to have, acute pancreatitis. In aspects, the methods comprise detecting Elastase 2A and/or 2B in a sample obtained from the individual, for example via detection of Elastase 2A and/or 2B protein and/or an mRNA encoding Elastase 2A and/or 2B. Further disclosed are kits for detecting Elastase 2A and/or 2B.
A securement device for an infant including a lower layer having a silicone adhesive and a center opening configured to receive an umbilical cord of the infant. An upwardly extending first securement tab and an upwardly extending second securement tab are positioned on opposing sides of the center opening. The first securement tab and the second securement tab define a bridge capturing an umbilical line. The lower layer includes a first antimicrobial, and the first securement tab includes a second antimicrobial.
39 - Transport, packaging, storage and travel services
Goods & Services
Retail pharmacy services; medical practice management for others; medical practice business administration; business management consulting Medical transport service
41 - Education, entertainment, sporting and cultural services
42 - Scientific, technological and industrial services, research and design
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Education services, namely, conducting fellowship training, pediatric residency training, research training including extensive doctoral and post-doctoral programs, international educational programs, child abuse prevention programs, patient services programs, pediatric dentistry fellowship, emergency medical services programs and hosting national educational conferences in the field of pediatric and adolescent medicine Medical and scientific research services; providing medical and scientific research information in the field of pediatric medicine and clinical trials; medical diagnostic laboratory services associated with direct patient care Pediatric and adolescent inpatient and outpatient hospital services
48.
METHODS FOR PREDICTING AUTOIMMUNITY POST GENE THERAPY
A method for detecting the presence of an allo-antibody against bile salt export pump (BSEP) protein in a subject, comprising: (a) providing a cell culture vessel comprising (i) an upper chamber and a lower chamber, wherein the chambers comprise a culture medium for culturing hepatocytes, (ii) a permeable membrane separating the chambers, and (iii) a layer of hepatocyte-like cells grown on the permeable membrane that are differentiated from a population of pluripotent stem cells having a modified ABCB11 gene; (b) adding taurocholic acid (TCA) to the lower chamber; (c) adding a serum sample from the subject to the upper chamber; (d) collecting culture medium from each chamber; and (e) determining the concentration of TCA in the culture medium from each chamber; wherein the lack of TCA in the culture medium of the upper chamber indicates that the sample contains allo-antibody against BSEP.
Disclosed herein are methods and systems of studying and modeling Neurotrophic Receptor Tyrosine Kinase 2 (NTRK2) signaling-mediated alveolar capillary injury and repair. Embodiments of the disclosure include generating a NTRK2vessel organoid having hyperoxic injury (NTRK2 HIVO) comprising increased NTRK2-truncated isoform 1 (NTRK2-T1) expression compared to a control NTRK2 vessel organoid (NTRK2 VO) cultured in a normoxic culture condition. The disclosure also includes methods of treating alveolar capillary injury by transfected NTRK2 full length (FL) isoforms, for example, via lipid nanoparticle delivery.
The invention provides methods for increasing SPINK7 activity in a target tissue of a subject by inhibiting urokinase plasminogen activator (uPA) proteolytic activity in the target tissue, and related methods and compositions.
A61K 31/352 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only ring hetero atom condensed with carbocyclic rings, e.g. cannabinols, methantheline
A61K 31/7088 - Compounds having three or more nucleosides or nucleotides
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 31/711 - Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
A framework is provided to assess and mitigate bias in clinical artificial intelligence and machine learning. The framework includes features that demonstrate usefulness in indicating the potential causes of bias and ways to mitigate them. The framework measures classification parity, as well as the effect of biased words on predictions. Techniques are provided that reduce bias in training data (derived from electronic health records) by filtering the salient clinical information and neutralizing biased words. In an embodiment, training data for a machine learning engine is rewritten to mitigate bias in the training dataset, where the re-writing step includes a step of information density filtering on electronic health record training data and/or replacing biased words in electronic health record training data with relevant neutral versions of the biased words.
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
G16H 50/70 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for mining of medical data, e.g. analysing previous cases of other patients
G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients
G16H 20/70 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to mental therapies, e.g. psychological therapy or autogenous training
Disclosed are methods for non-invasively classifying an individual diagnosed with lupus nephritis (LN) as a responder, a partial responder or a non-responder to lupus nephritis induction therapy. The method generally comprises (a) comparing a first Renal Activity Index for Lupus (RAIL) score and a second RAIL score, based on the detection of neutrophil gelatinase-associated lipocalin (NGAL), kidney injury molcculc-1 (KIM-1), monocyte chemotactic protein 1 (MCP-1), adiponectin, hemopexin and ceruloplasmin in a urine sample from the individual. Based on the RAIL score, the individual can be classified as a responder, a partial responder or a complete responder, and therapy can be appropriately adjusted.
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
G01N 33/564 - ImmunoassayBiospecific binding assayMaterials therefor for pre-existing immune complex or autoimmune disease
G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
Disclosed are methods for treating active eosinophilic colitis (EoC), or in certain aspects, inflammatory bowel disease (IBD), in an individual in need thereof. In one aspect, the methods may comprise a) assaying a tissue sample obtained from a colon of an individual who may be in need of such treatment, wherein the assaying comprises detecting expression of one or more gene of a transcriptome gene set; b) calculating a score based on the expression of one or more gene of a transcriptome gene set; and c) selecting a tissue sample that exhibits a score indicative of EoC or IBD. The methods may further comprise administering an EoC or IBD therapy to the individual whose tissue sample exhibited a score indicative of having EoC or IBD.
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
Disclosed are vaccine compositions, in particular, polyvalent icosahedral compositions for presentation of an mpox antigen. The disclosed compositions may contain an S particle comprising a norovirus (NoV) S domain protein and an mpox antigen, which may be linked via a linker protein domain operatively connected to the norovirus S domain protein and an mpox antigen. Fusion proteins for producing the vaccine compositions, and methods of using the disclosed vaccine composition are also provided.
Immunogenic targets associated with one or more types of cancer, wherein the immunogenic target includes a shared splicing amino acid-based neoantigen present in any of SEQ ID NOs: 1-2545, or a nucleotide coding therefor, are described, as well as uses thereof, and methods of identifying such immunogenic targets. Particular embodiments relate to immunotherapies for treating a type of cancer, and methods of treating a type of cancer by administering such immunotherapies, wherein the immunotherapy includes an immunogenic target including a shared splicing amino acid-based neoantigen present in any of SEQ ID NOs: 1-2545, or a nucleotide coding therefor.
The instant disclosure relates to methods for the treatment of an individual having TA-TMA, In one aspect, the methods encompass administration of a C5 inhibitor, more particularly eculizumab, or an antigen-binding fragment thereof. The disclosed methods, in one aspect, may be used for the treatment of individuals having TA-TMA and clinically significant bleeding.
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
G16H 20/17 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients delivered via infusion or injection
44 - Medical, veterinary, hygienic and cosmetic services; agriculture, horticulture and forestry services
Goods & Services
Medical pediatric and adolescent surgical services, namely, cardiothoracic surgery; medical services in the field of cardiothoracic diseases; medical diagnosis and treatment of cardiothoracic ailments; medical treatment in the field of cardiothoracic surgery; medical procedures for treatment of cardiothoracic diseases
58.
MULTI-CYCLIC IRAK1 AND IRAK4 INHIBITING COMPOUNDS AND USES THEREOF
THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES (USA)
KUROME THERAPEUTICS, INC. (USA)
Inventor
Hoyt, Scott, Bryan
Starczynowski, Daniel, T.
Thomas, Craig, Joseph
Rosenbaum, Jan, Susan
Bennett, Joshua
Hayes, Gregory, M.
Abstract
The present disclosure provides a method of treating an inflammatory disease or disorder, acute myeloid leukemia (AML), or myelodysplastic syndrome (MDS) in a subject, the method comprising administering to the subject a compound that inhibits IRAK1 and IRAK4. The present disclosure further provides a method of determining a subject with a disease/disorder that can be treated by the administration of a compound which inhibits IRAK1 and IRAK4.
Methods and compositions disclosed herein generally relates to methods of determining minimum hematopoietic stem cell (HSC) chimerism and gene dosage for correction of a hematopoietic disease; in particular, in in vivo models. The invention also relates to modified lentiviral expression vectors for increase a viral titer and various methods for increasing such titers as well as expression vectors capable of enhancing such titers. The invention also relates to CHS4 chromatin insulator-derived functional insulator sequences. The invention further relates to methods for genetic correction of diseases or reducing symptoms thereof, such as sickle cell anemia, a lysosomal storage disease. The invention further relates to a method of improving and/or correcting one or more central nervous system (CNS) abnormalities caused by one or more lysosomal storage disease. The invention further relates to methods of improving titer in transfection-based bioreactor culture production or transfection-based production systems using eukaryotic cells.
A method (100) of fabricating a heart valve replacement, the method (100) comprising: obtaining a representation (200) of a native heart valve (202) of a subject; obtaining one or more profiles of the native heart valve (202) from the representation (200) of the native heart valve (202), the one or more profiles defining one or more geometries of the native heart valve (202); deriving one or more mathematical functions from the one or more profiles; generating a model (400) of the heart valve replacement using the one or more mathematical functions; and fabricating the heart valve replacement based on the model (400) of the heart valve replacement.
The present invention provides a system and methods for detecting long-range cis-regulatory element (CRE) activities in a nucleic acid molecule obtained from a cell sample, the system comprising: one or more components for measuring multi-omics from a single nucleic acid molecule obtained from the cell sample, wherein the multi-omics measurements comprise one or more of: three-dimensional chromosomal conformation, CpG methylation, GpC accessibility, single nucleotide polymorphisms (SNPs); or one or more combinations thereof, and one or more components for measuring a transcriptome of the cell sample; wherein the system is capable of profiling a plurality of long-range CREs within the nucleic acid molecule obtained from the cell sample.
G16B 20/20 - Allele or variant detection, e.g. single nucleotide polymorphism [SNP] detection
C12Q 1/48 - Measuring or testing processes involving enzymes, nucleic acids or microorganismsCompositions thereforProcesses of preparing such compositions involving transferase
C12Q 1/6806 - Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
C12Q 1/6827 - Hybridisation assays for detection of mutation or polymorphism
Disclosed herein are organoid models of the blood-brain barrier, optionally having a disease associated feature, such as a cerebral cavernous malformation (CCM)-like feature, compositions comprising the same, methods for making the same from pluripotent stem cells, and methods of using the same. These organoids exhibit the complex organization of cells including neurons, endothelial cells, glial cells, and pericytes resembling the natural blood-brain barrier structure. The organoids may also exhibit disease like characteristics, such as having a cerebral cavernous malformation (CCM)-like features. These organoids may be used for studying the functions of the blood-brain barrier and cerebrovascular disorders.
Disclosed herein are methods of utilizing and forming suspension cultures of pluripotent stem cells (PSC), and differentiated cells, spheroids and organoids derived from PSCs (e.g., at industrial efficiency and/or scalability), and compositions comprising the same. These methods may be performed in suspension culture, without the use of basement membrane matrices, during PSC maintenance and expansion, as well as during differentiation of PSCs into differentiated cells and organoids, for example definitive endoderm (DE), hindgut spheroids (HGS), and intestinal organoids (IO). In some aspects, the methods may be xeno-free, and may be performed as per Good Manufacturing Practices (GMP). Also disclosed herein are methods controlling the polarity of epithelial cells in IOs, wherein the apical layer is oriented to the outside of the organoid, or alternatively to the inside of the organoid. Also disclosed are uses of the methods and compositions for transplantation and treatment.
Disclosed herein are methods for forecasting response to lupus nephritis (LN) therapy in individual diagnosed with childhood-onset SLE (cSLE). The methods may include the step of detecting each protein in a protein set in a sample obtained from an individual in need thereof. The protein set may include ceruloplasmin, kidney injury molecule 1 (KIM-1), monocyte chemotactic protein 1 (MCP-1), adiponectin, hemopexin, and NGAL.
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
Rhodothermus marinusRhodothermus marinus intein-N cDNA (CFTR-N-inteinN) and a second construct comprising a second portion of a human CFTR gene fused to Rhodothermus marinus intein-C cDNA (inteinC-CFTR-C). Further disclosed are methods of treating an individual having cystic fibrosis (CF) comprising administration of the paired nucleic acid constructs.
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 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
Disclosed herein arc vaccine compositions, in particular, polyvalent icosahedral compositions for presentation of a SARS-CoV-2 antigen. The disclosed compositions may contain an S particle comprising a norovirus (NoV) S domain protein and a SARS-CoV-2 antigen, which may be linked via a linker protein domain operatively connected to the norovirus S domain protein and the SARS- CoV-2 antigen. Methods of using the disclosed vaccine composition are also provided.
Disclosed are methods for determining risk of transplant graft rejection of an individual, and treatment thereof. The method may comprise a) identifying a risk level of the individual as either high-risk or moderate-risk, the identification comprising determining the presence of a risk factor selected from mismatched or haploidentical donor, ex vivo T-cell depleted graft, and history of prior graft failure; and b) categorizing an individual with two identified risk factors as high-risk and an individual with one identified risk factor as moderate-risk. The method may further comprise administering an IFNy neutralizing agent to the individual identified as high-risk.
A61K 39/395 - AntibodiesImmunoglobulinsImmune serum, e.g. antilymphocytic serum
A61P 37/06 - Immunosuppressants, e.g. drugs for graft rejection
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
A61K 39/00 - Medicinal preparations containing antigens or antibodies
68.
POLYPEPTIDES, NUCLEIC ACID MOLECULES, COMPOSITIONS, AND RELATED METHODS
THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SECRETARY, DEPT. OF HEALTH AND HUMAN SERVICES (USA)
Inventor
Millay, Douglas
Gamage, Dilani
Chernomordik, Leonid
Leikina, Evgenia
Abstract
Some embodiments of the invention include polypeptides comprising a myomerger polypeptide or an extracellular myomerger polypeptide. Other embodiments of the invention include nucleic acid molecules encoding polypeptides comprising a myomerger polypeptide or an extracellular myomerger polypeptide. Other embodiments of the invention include vectors comprising the nucleic acid molecule. Yet other embodiments of the invention include methods of using a myomerger poly peptide or an extracellular myomerger polypeptide. Additional embodiments of the invention are also discussed herein.
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 38/17 - Peptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from animalsPeptides having more than 20 amino acidsGastrinsSomatostatinsMelanotropinsDerivatives thereof from humans
Disclosed herein are systems and methods for assessing a learning network (LN). Aspects may include collecting data related to an organization to be assessed, analyzing the data with respect to a set of indicators associated with an organizational ontology of an actor-oriented architecture (AOA), determining a strength of LN capabilities for each indicator in the set of indicators, generating a dashboard to graphically communicate at least one summary-statistic indicative of a strength of at least one LN capability.
Disclosed herein are methods of producing liver organoids with intrahepatic sympathetic neurons, or sympathetic nerves, from pluripotent stem cells. These intrahepatic sympathetic neurons, or sympathetic nerves, are involved in fatty liver disease pathogenesis, and the liver organoids produced from the methods provided herein may be used as a model system for fatty liver disease and pharmaceutical screening.
Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in pediatric patients. Certain aspects of the invention relate to identifying one or more biomarkers associated with septic shock in pediatric patients in combination with one or more endothelial-derived biomarkers, receiving a dataset comprising biomarker concentrations, wherein the dataset is from a sample obtained from a pediatric patient having at least one indication of septic shock, then determining whether the biomarker concentrations of each of the at least one biomarkers are greater than one or more pre-determined cut-off biomarker concentration, wherein the level of said biomarker correlates with a predicted outcome.
G01N 33/68 - Chemical analysis of biological material, e.g. blood, urineTesting involving biospecific ligand binding methodsImmunological testing involving proteins, peptides or amino acids
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
72.
LIVER ORGANOID MODEL FOR HYPERBILIRUBINEMIA AND METHODS OF MAKING AND USING SAME
Disclosed herein are improved methods of maturing pluripotent stem cell-derived liver organoids with the use of hemoglobin metabolites such as bilirubin. Furthermore, by modulating the amounts of bilirubin used, liver organoids can be used as a model for hyperbilirubinemia. The scalability and tractability of these liver organoids made them excellent targets for drug screening against diseases such as hyperbilirubinemia. Also shown herein is the use of exogenous L-gulonolactone oxidase for improving viability of bilirubin-treated liver organoids.
Disclosed are methods for determining an initial or subsequent dose of an anti-tumor necrosis factor α (anti-TNFα) biologic for administering to an individual having an inflammatory condition, based on, in part, determining in the individual a level of one or more time-varying covariates selected from weight, albumin, erythrocyte sedimentation rate (ESR), neutrophil CD64 (nCD64) expression, and combinations thereof, and applying a correction factor to account for the changes in covariates over time. The inflammatory condition may be one selected from an IBD, such as Crohn's Disease (CD) or Ulcerative Colitis (UC), or inflammatory conditions such as uveitis or rheumatoid arthritis.
G16H 20/17 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients delivered via infusion or injection
A61K 39/00 - Medicinal preparations containing antigens or antibodies
A61P 1/00 - Drugs for disorders of the alimentary tract or the digestive system
A61P 29/00 - Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agentsNon-steroidal antiinflammatory drugs [NSAID]
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
74.
COMPOSITIONS FOR INTERFERON BLOCKADE AND METHODS OF USING SAME
Disclosed are compositions that may include one or more inhibitors of interferon activity for the treatment of a disease state, for example, a disorder associated with increased interferon levels such as thrombotic microangiopathy (“TMA”). Also disclosed are methods of treating an individual having a disease state such as thrombotic microangiopathy. Further disclosed are methods of diagnosing an individual with TMA.
C07K 16/24 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
A61K 39/00 - Medicinal preparations containing antigens or antibodies
C07K 16/18 - Immunoglobulins, e.g. monoclonal or polyclonal antibodies against material from animals or humans
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
75.
METHODS AND SYSTEMS FOR TRAINING AND PREPARING MACHINE LEARNING MODEL(S) FOR USE IN IDENTIFYING A SPECIFIC PATIENT'S FUTURE RISK OF DEVELOPING A PSYCHIATRIC CONDITION
Early identification of psychiatric conditions such as anxiety and depression in children, adolescents, and young adults coupled with evidenced based treatment can significantly reduce acute and chronic morbidity and complications that occur later in life. An individual with a high risk of developing these serious psychiatric conditions can be identified with data and machine-learning methods before the conditions develop. A method for training and preparing a machine learning model for use in identifying a specific patient's future risk of developing a psychiatric condition (e.g. diagnosis, emergency room visit, hospitalization, suicide attempt) includes the following steps: collecting first electronic health records from previous patients who have been diagnosed with the psychiatric condition; processing the collected electronic health records into a training dataset and training a machine learning model with the training dataset to compute the specific patient's future risks for developing the psychiatric condition (e.g. diagnosis, emergency room visit, hospitalization, suicide attempt).
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
G16H 50/70 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for mining of medical data, e.g. analysing previous cases of other patients
A distraction device for subcutaneous attachment to vertebrae for correcting curvature of a spine is provided. The distraction device includes a housing that includes a generally hollow interior, a drive system including a drive rod operatively supported in the hollow interior of the housing and a drive element coupled to the drive rod. The drive rod is rotatable within the housing about an axis of rotation and includes a first member a second member. The distraction device includes a first extension arm operatively coupled to the first end of the housing and a second extension arm operatively coupled to the second end of the housing. Rotation of the drive rod, induced by the drive element, causes the first extension arm to move into or out from the opening at the first end of the housing in a first movement direction and the second extension arm to move into or out from the opening at the second end of the housing in a second movement direction to vary a length of the distraction device.
Disclosed herein are compositions and methods for treating an individual having a leukemia. In certain aspects, the methods may include administering an I0DVA1 compound and ponatinib to an individual in need thereof for treatment of a leukemia, which may include, for example, TKI-resistant leukemia, TKI-resistant Ph+ B-ALL, Chronic Myelogenous Leukemia (CML), Ph-Positive Acute Lymphoblastic Leukemia (ALL), Ph-like ALL, Resistant Chronic Phase Chronic Myeloid Leukemia (CP-CML), MLL-rearranged B-ALL, and VAV3 positive leukemia.
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 31/341 - Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having five-membered rings with one oxygen as the only ring hetero atom, e.g. isosorbide not condensed with another ring, e.g. ranitidine, furosemide, bufetolol, muscarine
A61K 31/4184 - 1,3-Diazoles condensed with carbocyclic rings, e.g. benzimidazoles
A61K 31/444 - Non-condensed pyridinesHydrogenated derivatives thereof containing further heterocyclic ring systems containing a six-membered ring with nitrogen as a ring hetero atom, e.g. amrinone
A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
A61P 35/02 - Antineoplastic agents specific for leukemia
Disclosed herein are improved methods of expanding cell populations and methods of producing organoids from pluripotent stem cells, as well as compositions and uses thereof, compositions including an amino acid supplemented liquid component which can be used for performing the methods, and kits including components for performing the methods and/or using the products of the methods. These methods may be performed without the need for xenogeneic and undefined basement membrane matrices for cell culture, which enables good manufacturing practice (GMP) compliant approaches for producing organoids for various uses such as clinical screening and therapeutics, including methods for treating a liver-related disease or disorder and screening candidate compounds or compositions for treating the same.
Disclosed herein are compositions of gastrointestinal organoids comprising cells originating from all three primary germ layers and methods of making and use thereof. These gastrointestinal organoids exhibit complex cellular organization and functions resembling naturally occurring organ tissue, and serve as excellent three dimensional models for studying gastrointestinal physiology.
Disclosed herein are compositions of organoids comprising a mesodermal vascular network and definitive endoderm derivatives. Also disclosed herein are methods making use of human pluripotent stem cells to differentiate cells of multiple germ layer lineages with proper organization. These organoids exhibit vasculature and can be used as models for studying endoderm-derived organogenesis and vascular interactions.
Disclosed herein are vaccine compositions, in particular, polyvalent icosahedral compositions for antigen presentation. The disclosed compositions may contain an S particle made up of recombinant fusion proteins. The recombinant fusion proteins may include a norovirus (NoV) S domain protein, a linker protein domain operatively connected to the norovirus S domain protein, and an antigen protein domain operatively connected to said linker.
Disclosed herein are systems and methods for respiration-controlled virtual experiences. In an embodiment, a controller presents a virtual experience via a user interface that is perceptible to a user. The controller receives a respiration-data stream from a respiration device with which the user is operably engaged. The respiration-data stream includes one or more respiration-parameter values of one or more respiration parameters associated with ongoing respiration of the user. The controller updates the virtual experience based at least in part on the one or more respiration-parameter values in the respiration-data stream. The virtual experience includes a plurality of sequential phases that have an associated phase sequence. Among the sequential phases is an active-induction phase that corresponds in time to the user receiving anesthetic induction via the respiration device, as well as one or more phases that each precede the active-induction phase.
A61B 5/00 - Measuring for diagnostic purposes Identification of persons
A61B 5/08 - Measuring devices for evaluating the respiratory organs
A61B 5/097 - Devices for facilitating collection of breath or for directing breath into or through measuring devices
A61B 5/16 - Devices for psychotechnicsTesting reaction times
A61M 16/01 - Devices for influencing the respiratory system of patients by gas treatment, e.g. ventilators Tracheal tubes specially adapted for anaesthetising
A63F 13/21 - Input arrangements for video game devices characterised by their sensors, purposes or types
A63F 13/212 - Input arrangements for video game devices characterised by their sensors, purposes or types using sensors worn by the player, e.g. for measuring heart beat or leg activity
G06F 3/01 - Input arrangements or combined input and output arrangements for interaction between user and computer
A61M 21/00 - Other devices or methods to cause a change in the state of consciousnessDevices for producing or ending sleep by mechanical, optical, or acoustical means, e.g. for hypnosis
83.
MODULATION OF ANTIBODY FUNCTION VIA SIALIC ACID MODIFICATION
Disclosed are methods of modulating antibody function, in particular, via modifying one or more terminal sialic acid residues of an antibody. In certain aspects, the antibody may be an IgG antibody, wherein one or more terminal sialic acid residues is modified to modulate the antibody function. Further disclosed are methods of using such modified antibodies, and compositions comprising modified antibodies.
THE UNITED STATES OF AMERICA,ASREPRESENTED BY THE SECRETARY,DEPT. OF HEALTH AND HUMAN SERVICES (USA)
Inventor
Hoyt, Scott Bryan
Thomas, Craig Joseph
Starczynowski, Daniel T.
Rosenbaum, Jan
Gracia Maldonado, Gavriel
Abstract
Some embodiments of the disclosure include inventive compounds (e.g., compounds of Formula (I)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.), Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
A61K 31/496 - Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/541 - Non-condensed thiazines containing further heterocyclic rings
A61K 31/635 - Compounds containing para-N-benzene- sulfonyl-N-groups, e.g. sulfanilamide, p-nitrobenzenesulfonohydrazide having a heterocyclic ring, e.g. sulfadiazine
A61P 35/02 - Antineoplastic agents specific for leukemia
C07D 519/00 - Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups or
85.
DIFFERENTIAL CXCR4 EXPRESSION ON HEMATOPOIETIC PROGENITOR CELLS VERSUS STEM CELLS DIRECTS HOMING AND LONG-TERM ENGRAFTMENT
Compositions and methods for delivering a protein of interest such as a cell homing molecule (e.g., CXCR4) into host cells (e.g., to hematopoietic cells). The compositions and methods provided herein may be used to enhance homing and long-term engraftment of hematopoietic cells post transplantation.
Disclosed herein are methods for enhancing an immune response in an individual in need thereof. The methods, in certain aspects, may comprise administering bacterial aldehyde dehydrogenase, a bacteria that produces aldehyde dehydrogenase, or combinations thereof to an individual. Further disclosed are compositions, such as nutritional compositions, which may comprise bacterial aldehyde dehydrogenase, a bacteria that produces aldehyde dehydrogenase, or combinations thereof.
The invention provides methods for treating a disease or disorder characterized by epithelial barrier dysfunction by modulating CARD14:MYC interaction, and related methods and compositions for modulating MYC activity in target epithelial cells, the methods comprising contacting the epithelial cells with an inhibitor or an agonist of CARD14 binding to MYC in the target epithelial cells.
THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES (USA)
KUROME THERAPEUTICS, INC. (USA)
Inventor
Hoyt, Scott Bryan
Starczynowski, Daniel T.
Thomas, Craig Joseph
Rosenbaum, Jan Susan
Abstract
Some embodiments of the invention include inventive compounds (e.g., compounds of Formula (I), (II), or (III)) and compositions (e.g., pharmaceutical compositions) which inhibit IRAK and/or FLT3 and which can be used for treating, for example, certain diseases. Some embodiments include methods of using the inventive compound (e.g., in compositions or in pharmaceutical compositions) for administering and treating (e.g., diseases such as hematopoietic cancers, myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), etc.). Additional embodiments provide disease treatment using combinations of the inventive IRAK and/or FLT3 inhibiting compounds with other therapies, such as cancer therapies.
A61K 31/437 - 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 five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
A61K 31/445 - Non-condensed piperidines, e.g. piperocaine
A61K 31/497 - Non-condensed pyrazines containing further heterocyclic rings
A61K 31/4985 - Pyrazines or piperazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/5025 - PyridazinesHydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
A61K 31/506 - PyrimidinesHydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic rings
89.
MODEL-INFORMED PRECISION DOSING SYSTEMS FOR TREATMENT OF INFLAMMATORY BOWEL DISEASE
Disclosed are methods for preparing a patient-specific vedolizumab dosing regimen for an individual in need thereof, comprising accessing, using a processor, a memory; selecting a first model stored in the memory; forecasting, based on the selected first model, a patient-specific predicted concentration time profile of the vedolizumab, selecting a dosing regimen, based on said forecasting, wherein said dosing regimen achieves a treatment objective, said treatment objective being a therapeutic level of vedolizumab; and outputting said dosing regimen. Further disclosed are methods of treating an individual having an inflammatory bowel disease.
G16H 20/10 - ICT specially adapted for therapies or health-improving plans, e.g. for handling prescriptions, for steering therapy or for monitoring patient compliance relating to drugs or medications, e.g. for ensuring correct administration to patients
90.
COLONIC ORGANOIDS AND METHODS OF MAKING AND USING SAME
Disclosed herein are methods for the in vitro differentiation of a precursor cell into definitive endoderm, which may further be differentiated into a human colonic organoid (HCO), via modulation of signaling pathways. Further disclosed are HCOs and methods of using HCOs, which may be used, for example, for the HCOs may be used to determine the efficacy and/or toxicity of a potential therapeutic agent for a disease selected from colitis, colon cancer, polyposis syndromes, and/or irritable bowel syndrome.
The instant disclosure relates to pseudovirus nanoparticles (PVNPs) and compositions comprising PVNPs. The disclosed PVNPs may be comprised of fusion proteins that form an icosahedral structure and a nanoparticle shell. The disclosed fusion proteins may comprise a modified norovirus (NoV) S domain protein; a hemagglutinin I (HA1) antigen of the influenza hemagglutinin I (HA1) of influenza vims; and a peptide linker connecting the C-terminus of the NoV S domain to the HA1 antigen. The modified NoV S domain proteins form the interior nanoparticle shell of said PVNP composition and display the 60 HA1 antigens on the surface of the nanoparticle shell. Methods of making and using the PVNPs and compositions containing PVNPs are also disclosed.
Some embodiments of the invention include chimeric polypeptides. Other embodiments of the invention include chimeric nucleic acid molecules encoding a chimeric polypeptide. Other embodiments of the invention include vectors comprising a chimeric nucleic acid molecule. Still other embodiments of the invention include cells comprising a chimeric nucleic acid molecule, a chimeric polypeptide, or both. Yet other embodiments of the invention include methods of making cells. Some embodiments include methods of treating disease. Additional embodiments of the invention are also discussed herein.
A device using resorbable materials to actuate, such as a device (10) for repairing pectus excavatum in a patient through activation of a bioresorbable material (40, 52).
Methods and compositions disclosed herein generally relate to methods of identifying, validating, and measuring clinically relevant, quantifiable biomarkers of diagnostic and therapeutic responses for blood, vascular, cardiac, and respiratory tract dysfunction, particularly as those responses relate to septic shock in patients, such as pediatric. In particular, the invention relates to analyzing biomarkers associated with septic shock in pediatric patients, obtaining a sample from a patient having at least one indication of septic shock, then determining the gene expression mosaic for the patient, wherein the gene expression mosaic can correlate with a predicted outcome and can inform treatment strategy.
C12Q 1/6883 - Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
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
G16H 50/30 - ICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for calculating health indicesICT specially adapted for medical diagnosis, medical simulation or medical data miningICT specially adapted for detecting, monitoring or modelling epidemics or pandemics for individual health risk assessment
Disclosed herein are methods of treating a human patient having TA-TMA, for example high- risk TA-TMA with MODS, comprising administration of a C5 inhibitor, for example eculizumab or an antigen binding fragment thereof. The disclosed dosing regimens may comprise one or more of a loading phase, induction phase, and maintenance phase. The disclosed methods are particularly advantageous for individuals that do not have clinically relevant bleeding.
Disclosed are methods for making a vascularized hollow organ derived from human intestinal organoid (HIOs). The HIOs may be obtained from human embryonic stem cells (ESC's) and/or induced pluripotent stem cells (iPSCs), such that the HIO forms mature intestinal tissue. Also disclosed are methods for making a human intestinal tissue containing a functional enteric nervous system (ENS).
The Board of Regents of The University of Texas System (USA)
Children's Hospital Medical Center (USA)
Inventor
Shay, Jerry
Drissi, Rachid
Abstract
Brain tumors remain the leading cause of cancer-related deaths in children and often are associated with long-term sequelae among survivors of current therapies. Telomerase and telomeres play important roles in cancer, representing attractive therapeutic targets to treat children with poor-prognosis brain tumors such as diffuse intrinsic pontine glioma (DIPG), high-grade glioma (HGG) and high-risk medulloblastoma (MB). It has shown that DIPG, HGG and MB frequently express telomerase activity. It is now shown that the telomerase-dependent incorporation of 6-thio-2′deoxyguanosine (6-thio-dG), a telomerase substrate precursor analog, into telomeres leads to telomere dysfunction-induced foci (TIFs) along with extensive genomic DNA damage, cell growth inhibition and cell death of primary stem-like cells derived from patients with DIPG, HGG and MB. Importantly, the effect of 6-thio-dG is persistent even after drug withdrawal. Treatment with 6-thio-dG elicits a sequential activation of ATR and ATM pathways and induces G2/M arrest. In vivo, treatment of mice bearing MB xenografts with 6-thio-dG delays tumor growth, increases in-tumor TIFs and apoptosis. Furthermore, 6-thio-dG crosses the blood-brain barrier and specifically targets tumor cells in an orthotopic mouse model of DIPG. Together, these findings suggest that 6-thio-dG is a promising approach to treat therapy-resistant telomerase-positive pediatric brain tumors.
A61K 31/708 - 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 having oxo groups directly attached to the purine ring system, e.g. guanosine, guanylic acid
A61K 45/06 - Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
B. G. NEGEV TECHNOLOGIES AND APPLICATIONS LTD., AT BEN-GURION UNIVERSITY (Israel)
CHILDREN'S HOSPITAL MEDICAL CENTER (USA)
Inventor
Mangano, Francesco
Zarrouk, David
Daniel, Atai
Peer, Segev
Grinshpan, Ben
Coronel, Matan
Abstract
Embodiments pertain to an access port assembly configured to assist in creating access for and accommodating a medical device extending from outside a patient body and terminating in a cavity internal to the patient body. The assembly may comprise a guide element comprising a guide channel having a longitudinal guide axis extending from an inlet to an outlet of the guide element for guiding the medical device through the guide channel. The guide element may be movably coupled with the fixation base by an articulating linkage to allow selecting an orientation of the guide channel relative to the site of interest of the patient body. The articulating linkage may be implemented as a ball-and-socket linkage. Embodiments also pertain to a system for controlling delivery of fluid into the patient body and/or removal of fluid from the patient body, via one or more access ports.
A61B 90/11 - Instruments, implements or accessories specially adapted for surgery or diagnosis and not covered by any of the groups , e.g. for luxation treatment or for protecting wound edges for stereotaxic surgery, e.g. frame-based stereotaxis with guides for needles or instruments, e.g. arcuate slides or ball joints
F16M 11/14 - Means for attachment of apparatusMeans allowing adjustment of the apparatus relatively to the stand allowing pivoting in more than one direction with ball-joint
99.
Non-invasive methods for skin sample collection and analysis
Kits for assessing a skin condition of a subject comprise (a) a first set of adhesive tapes adapted to adhere a first biological material from stratum corneum of a target skin site of the subject, (b) a second set of adhesive tapes adapted to adhere a second biological material from an epidermis layer below stratum corneum of a target skin site of the subject, wherein each of the adhesive tapes in the first set of adhesive tapes and the second set of adhesive tapes comprises a water-soluble adhesive layer attached to a substrate, (c) one or more reagents for extracting biological material adhered to the water-soluble adhesive layers of the adhesive tapes, and (d) one or more detection agents for determining a presence of microbial biological materials and/or host biological materials from water-soluble adhesive tape extract.
A frequency encoded source imaging system includes an EEG or MEG sensor array and a processing system for analyzing the signals from the sensor array in at least two different frequency bands, where the analysis is localized with respect to a three-dimensional grid corresponding to the portion of the human body. Alternately, a frequency encoded source imaging system includes an EEG or MEG sensor array and a processing system for analyzing the signals from the sensor array in a high-definition frequency band comprising frequencies greater than 70 Hz, where the analysis is localized with respect to a three-dimensional grid corresponding to the portion of the human body
A61B 5/374 - Detecting the frequency distribution of signals, e.g. detecting delta, theta, alpha, beta or gamma waves
A61B 5/00 - Measuring for diagnostic purposes Identification of persons
A61B 5/055 - Detecting, measuring or recording for diagnosis by means of electric currents or magnetic fieldsMeasuring using microwaves or radio waves involving electronic [EMR] or nuclear [NMR] magnetic resonance, e.g. magnetic resonance imaging
A61B 5/245 - Detecting biomagnetic fields, e.g. magnetic fields produced by bioelectric currents specially adapted for magnetoencephalographic [MEG] signals
A61B 6/46 - Arrangements for interfacing with the operator or the patient
