Produits et services

Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders. Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders. Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical consulting services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders from an online database; providing medical information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal disease and disorder information based on collected patient data and information from an online database all for treatment and diagnostic purposes.

Abrégé

This application relates to adeno-associated viral vectors encoding a truncated yet functional ATP7B for use in gene therapy for treating Wilson disease (WD). The truncated ATP7B described herein has several advantages over the wild-type ATP7B such as higher efficacy and improved manufacturing yield.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes

Abrégé

Provided are systems and methods for separating adeno-associated viral (rAAV) particles from host cell derived impurities and other byproducts in a preparation produced from a host cell culture.

Classes IPC  ?

• B01D 15/36 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction ionique, p. ex. échange d'ions, paire d'ions, suppression d'ions ou exclusion d'ions
• C12N 15/86 - Vecteurs viraux

Abrégé

Disclosed herein are methods for treating Angelman syndrome (AS) in a human subject in need thereof by administering at least one loading dose and one maintenance dose of an antisense compound (e.g., antisense compound comprising the nucleic acid sequence of SEQ ID NO: 1 or modified nucleic acid sequence of SEQ ID NO: 2). The disclosed methods achieve a concentration of the antisense compound in the brain that 1) is effective for treating or reducing symptoms of Angelman syndrome and 2) minimizes the risk of unwanted side effects (e.g., radiculopathy).

Classes IPC  ?

• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• A61K 31/115 - Formaldéhyde
• A61K 31/712 - Acides nucléiques ou oligonucléotides ayant des sucres modifiés, c.-à-d. autres que le ribose ou le 2'-désoxyribose
• A61K 31/7125 - Acides nucléiques ou oligonucléotides ayant des liaisons internucléosides modifiées, c.-à-d. autres que des liaisons 3'-5' phosphodiester
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 25/00 - Médicaments pour le traitement des troubles du système nerveux
• A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides

Abrégé

This application relates to double-stranded small interfering RNAs that modulate DUX4 gene expression and describes methods of inhibiting DUX4 gene expression by contacting a cell with said double-stranded small interfering RNAs. The application further provides compositions comprising said double-stranded small interfering RNAs and their use in methods of preventing or treating a disease or disorder associated with aberrant expression of DUX4, such as facioscapulohumeral muscular dystrophy (FSHD) or cancer, in a subject.

Classes IPC  ?

• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
• A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p. ex. un fragment Fc

Produits et services

Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders. Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific and medical research, namely, providing patient data for research purposes in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing online non-downloadable software enabling input and collection of data and information on bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders for screening, diagnosis and treatment purposes. Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical consulting services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders via an online database; medical information services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes provided via a database.

Produits et services

Pharmaceutical preparations for the treatment of genetic, central nervous system, and lysosomal storage diseases and disorders; gene therapy products for the treatment of genetic, central nervous system, and lysosomal storage diseases and disorders, namely, virus vector-based gene therapy preparations for intravenous delivery. Pharmaceutical, scientific, and medical research and development in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; providing medical and scientific research information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; providing medical research and scientific research information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; pharmaceutical, scientific, and medical research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of gene therapy, genetic, central nervous system, and lysosomal storage diseases and disorders; biomedical research in the field of gene therapy design and delivery; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; scientific research in the nature of conducting clinical trials for others in the field of gene therapy design and delivery. Providing medical services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; medical consulting services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders, featuring inputting and collection of data and information on genetic, central nervous system, and lysosomal storage diseases and disorders for screening, diagnosis and treatment purposes (Term considered too vague by the International Bureau pursuant to Rule 13 (2) (b) of the Regulations); medical information services in the nature of providing a database in the field of genetic, central nervous system, and lysosomal storage disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes (Term considered too vague by the International Bureau pursuant to Rule 13 (2) (b) of the Regulations); providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders.

Abrégé

The invention is directed to the field of therapeutic formulations, in particular to lyophilization of a therapeutic cargo molecule, such as RNA. The invention provides a method for lyophilization of a molecule. The present disclosure further describes a lyophilized composition obtainable by the inventive method, a pharmaceutical composition, a vaccine, a therapeutic and a kit or kit of parts. Moreover, the disclosure herein provides a novel lyophilization excipient that protects the composition from degrading when, for example, lyophilizing RNA. The use of the inventive method further includes the manufacture of a composition that can be used after lyophilization with equivalent therapeutic effect and composition integrity.

Classes IPC  ?

• A61K 9/51 - Nanocapsules
• A61K 9/19 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres lyophilisées
• A61K 31/7105 - Acides ribonucléiques naturels, c.-à-d. contenant uniquement des riboses liés à l'adénine, la guanine, la cytosine ou l'uracile et ayant des liaisons 3'-5' phosphodiester
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• A61K 38/45 - Transférases (2)
• A61K 38/47 - Hydrolases (3) agissant sur des composés glycosyliques (3.2), p. ex. cellulases, lactases

Abrégé

This application provides modified batch systems and methods using perfusion (e.g., intensified perfusion) for increasing productivity and/or cell density in viral particle production to achieve high viral particle, e.g., AAV yield.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux

Abrégé

UBE3AUBE3AUBE3A mRNA and/or UBE3A polypeptide levels in a subject.

Classes IPC  ?

• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides

Produits et services

(1) Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders. (1) Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific and medical research, namely, providing patient data for research purposes in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing online non-downloadable software enabling input and collection of data and information on bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders for screening, diagnosis and treatment purposes. (2) Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical consulting services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders via an online database; medical information services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes provided via a database.

Abrégé

The present invention includes solid compositions of triglycerides with one or more fatty acids, such as triheptanoin and glycerol phenylbutyrate, and therapeutic use thereof. The solid compositions can be prepared by spray-drying or other processes.

Classes IPC  ?

• A61K 31/23 - Esters, p. ex. nitroglycérine, sélénocyanates d'acides carboxyliques d'acides acycliques, p. ex. pravastatine d'acides ayant un groupe carboxyle lié à une chaîne d'au moins sept atomes de carbone
• A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
• A61K 9/16 - AgglomérésGranulésMicrobilles
• A61K 31/20 - Acides carboxyliques, p. ex. acide valproïque ayant un groupe carboxyle lié à une chaîne acyclique d'au moins sept atomes de carbone, p. ex. acides stéarique, palmitique ou arachidique

Abrégé

The present invention includes solid compositions of triglycerides with one or more fatty acids, such as triheptanoin and glycerol phenylbutyrate, and therapeutic use thereof. The solid compositions can be prepared by spray-drying or other processes.

Classes IPC  ?

• A61K 31/23 - Esters, p. ex. nitroglycérine, sélénocyanates d'acides carboxyliques d'acides acycliques, p. ex. pravastatine d'acides ayant un groupe carboxyle lié à une chaîne d'au moins sept atomes de carbone
• A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
• A61K 9/16 - AgglomérésGranulésMicrobilles
• A61K 31/20 - Acides carboxyliques, p. ex. acide valproïque ayant un groupe carboxyle lié à une chaîne acyclique d'au moins sept atomes de carbone, p. ex. acides stéarique, palmitique ou arachidique

Abrégé

The present invention includes solid compositions of triglycerides with one or more fatty acids, such as triheptanoin and glycerol phenylbutyrate, and therapeutic use thereof. The solid compositions can be prepared by spray-drying or other processes.

Classes IPC  ?

• A61K 31/23 - Esters, p. ex. nitroglycérine, sélénocyanates d'acides carboxyliques d'acides acycliques, p. ex. pravastatine d'acides ayant un groupe carboxyle lié à une chaîne d'au moins sept atomes de carbone
• A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
• A61K 9/16 - AgglomérésGranulésMicrobilles
• A61K 31/20 - Acides carboxyliques, p. ex. acide valproïque ayant un groupe carboxyle lié à une chaîne acyclique d'au moins sept atomes de carbone, p. ex. acides stéarique, palmitique ou arachidique

Abrégé

This application provides antisense nucleic acid compounds that diminish COMP mRNA levels and/or COMP polypeptide levels in chondrocytes, thereby reducing accumulation of mutant COMP and restoring chondrocyte health. The application further provides compositions comprising antisense nucleic acid compounds and their use in methods of preventing or treating conditions associated with COMP accumulation in chondrocytes of a subject.

Classes IPC  ?

• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens

Produits et services

(1) Pharmaceutical preparations for the treatment of genetic, central nervous system, and lysosomal storage diseases and disorders; gene therapy products for the treatment of genetic, central nervous system, and lysosomal storage diseases and disorders, namely, virus vector-based gene therapy preparations for intravenous delivery. (1) Pharmaceutical, scientific, and medical research and development in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; providing medical and scientific research information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; providing medical research and scientific research information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; pharmaceutical, scientific, and medical research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of gene therapy, genetic, central nervous system, and lysosomal storage diseases and disorders; biomedical research in the field of gene therapy design and delivery; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; scientific research in the nature of conducting clinical trials for others in the field of gene therapy design and delivery. (2) Providing medical services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; medical consulting services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders, featuring inputting and collection of data and information on genetic, central nervous system, and lysosomal storage diseases and disorders for screening, diagnosis and treatment purposes (Term considered too vague by the International Bureau pursuant to Rule 13 (2) (b) of the Regulations); medical information services in the nature of providing a database in the field of genetic, central nervous system, and lysosomal storage disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes (Term considered too vague by the International Bureau pursuant to Rule 13 (2) (b) of the Regulations); providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders.

Abrégé

The present disclosure provides novel methods of producing CDKL5 proteins, novel CDKL5 antibodies, and uses of the same including for in vitro detection assays.

Classes IPC  ?

• A61K 38/00 - Préparations médicinales contenant des peptides
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C12N 9/12 - Transférases (2.) transférant des groupes contenant du phosphore, p. ex. kinases (2.7)
• C12N 15/62 - Séquences d'ADN codant pour des protéines de fusion

Abrégé

This application provides polynucleotides comprising a coding sequence for a functionally active bile salt export pump (BSEP) protein or a functionally active fragment thereof. The application further provides compositions comprising said polynucleotides and their use in methods of preventing or treating conditions associated with BSEP deficiency in a subject, such as progressive familial intrahepatic cholestasis type 2 (PFIC2).

Classes IPC  ?

• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• A61K 31/7115 - Acides nucléiques ou oligonucléotides ayant des bases modifiées, c.-à-d. autres que l'adénine, la guanine, la cytosine, l'uracile ou la thymine
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 1/16 - Médicaments pour le traitement des troubles du tractus alimentaire ou de l'appareil digestif des troubles de la vésicule biliaire ou du foie, p. ex. protecteurs hépatiques, cholagogues, cholélitholytiques
• C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales

Abrégé

The present disclosure provides novel synthetic nucleic acids and recombinant adeno-associated virus (rAAV) comprising the same, as well as methods of their use in the treatment of muscular dystrophies associated with a dystrophin mutation. Also provided are pharmaceutical compositions comprising a novel synthetic nucleic acid or rAAV of the invention, and a pharmaceutically acceptable carrier or excipient. Pharmaceutical compositions comprising an rAAV of the invention may be useful in gene therapy for the treatment of dystrophin-associated muscular dystrophies, such as Duchenne muscular dystrophy (DVD). Becker muscular dystrophy (BMD), and X-linked cardiomyopathy.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux

Abrégé

This application relates to recombinant adeno-associated virus (rAAV) packaging and/or producer cell lines which have been engineered to reduce expression and/or activity of one or more genes and/or proteins to increase rAAV titers. The methods of generating the engineered cell lines have also been described herein.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 5/00 - Cellules non différenciées humaines, animales ou végétales, p. ex. lignées cellulairesTissusLeur culture ou conservationMilieux de culture à cet effet
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens

Abrégé

Disclosed are methods and dosing regimens for treating a patient suffering from osteogenesis imperfecta, comprising administering to the patient a therapeutically effective amount of an anti-sclerostin antibody. The invention also provides an anti-sclerostin antibody for use in the treatment of osteogenesis imperfecta, comprising administering a therapeutically effective amount of the anti-sclerostin antibody each month according to certain dosing regimens.

Classes IPC  ?

• C07K 16/22 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des facteurs de croissance
• A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
• A61P 19/08 - Médicaments pour le traitement des troubles du squelette des maladies osseuses, p. ex. rachitisme, maladie de Paget

Abrégé

This present disclosure provides pharmaceutical compositions for delivering recombinant adeno-associated virus (rAAV) particles, generally comprising a buffer, a monovalent salt, a poly hydric alcohol, and a triblock copolymer surfactant.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 47/26 - Hydrates de carbone, p. ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharidesLeurs dérivés, p. ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
• A61K 47/34 - Composés macromoléculaires obtenus par des réactions autres que celles faisant intervenir uniquement des liaisons non saturées carbone-carbone, p. ex. polyesters, acides polyaminés, polysiloxanes, polyphosphazines, copolymères de polyalkylène glycol ou de poloxamères

Produits et services

Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical consulting services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders, and collecting data and information on bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders for screening, diagnosis and treatment purposes; medical information services in the nature of providing a database in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes

Abrégé

Provided are methods and compositions for removal of host cell protein contaminants from recombinant adeno-associated viral vector preparations.

Classes IPC  ?

• C12N 7/02 - Isolement ou purification

Produits et services

Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical consulting services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders, featuring inputting and collection of data and information on bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders for screening, diagnosis and treatment purposes; medical information services in the nature of providing a database in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes

Abrégé

e.g.e.g., rare genetic CNS disorders such as CDKL5 deficiency disorder (CDD).

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux

Produits et services

Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical consulting services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders, featuring inputting and collection of data and information on bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders for screening, diagnosis and treatment purposes; medical information services in the nature of providing a database in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes

Produits et services

Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Pharmaceutical preparations for the treatment of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Pharmaceutical, scientific, and medical research and development in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; providing medical research and scientific research information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders

Produits et services

Providing medical services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; medical consulting services in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders, featuring inputting and collection of data and information on bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal diseases and disorders for screening, diagnosis and treatment purposes; medical information services in the nature of providing a database in the field of bone, genetic, joint, metabolic, musculoskeletal, osteopathic, orthopedic, and skeletal disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes

Abrégé

This application relates to recombinant adeno-associated virus (rAAV) packaging and/or producer cell lines which have been engineered to reduce expression and/or activity of one or more genes and/or proteins to increase rAAV titers. The methods of generating the engineered cell lines have also been described herein.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C12N 15/86 - Vecteurs viraux
• C12Q 1/6869 - Méthodes de séquençage

Abrégé

Provided are methods of separating full capsid particles and empty capsid particles in a viral capsid preparation using an anion exchange medium. Methods may involve using a wash solution comprising a quaternary ammonium salt. Methods include applying a viral capsid preparation to an anion exchange medium, combined with multiple quaternary ammonium salt wash solutions.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• B01D 15/36 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction ionique, p. ex. échange d'ions, paire d'ions, suppression d'ions ou exclusion d'ions
• B01D 15/42 - Adsorption sélective, p. ex. chromatographie caractérisée par le mode de développement, p. ex. par déplacement ou par élution

Produits et services

Pharmaceutical preparations for the treatment of genetic, central nervous system, and lysosomal storage diseases and disorders; gene therapy products for the treatment of genetic, central nervous system, and lysosomal storage diseases and disorders, namely, virus vector-based gene therapy preparations for intravenous delivery

Produits et services

Providing medical services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; medical consulting services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders, featuring inputting and collection of data and information on genetic, central nervous system, and lysosomal storage diseases and disorders for screening, diagnosis and treatment purposes; medical information services in the nature of providing a database in the field of genetic, central nervous system, and lysosomal storage disease and disorder information based on collected patient data and information all for treatment and diagnostic purposes; providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of genetic, central nervous system, and lysosomal storage diseases and disorders

Produits et services

Pharmaceutical, scientific, and medical research and development in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; scientific research in the nature of conducting clinical trials for others in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; providing medical and scientific research information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; providing medical research and scientific research information in the field of genetic, central nervous system, and lysosomal storage diseases and disorders; pharmaceutical, scientific, and medical research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of gene therapy, genetic, central nervous system, and lysosomal storage diseases and disorders; biomedical research in the field of gene therapy design and delivery; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; scientific research in the nature of conducting clinical trials for others in the field of gene therapy design and delivery

Abrégé

The disclosure provides improved human FGF23-binding antibodies with increased affinity for human FGF23 as compared to burosumab, enabling reduced dosing frequency, improving convenience of therapy, and achieving higher efficacy compared to burosumab, without compromising safety.

Classes IPC  ?

• C07K 16/22 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des facteurs de croissance
• A61K 38/18 - Facteurs de croissanceRégulateurs de croissance
• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61P 19/08 - Médicaments pour le traitement des troubles du squelette des maladies osseuses, p. ex. rachitisme, maladie de Paget
• A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
• C07K 14/50 - Facteur de croissance des fibroblastes [FGF]

Abrégé

The present disclosure relates to solid forms of (1R,4R,5R,6R,7S)-6-acetamido-4-((R)-1,2-dihydroxyethyl)-7-hydroxy-2-oxo-3,9-dioxabicyclo[3.3.1]nonan-1-yl palmitate (Compound I) or a pharmaceutically acceptable salt and/or solvate thereof, pharmaceutical compositions comprising the solid forms, and therapeutic uses thereof. In particular, the present disclosure relates to solid forms of Compound I or a pharmaceutically acceptable salt and/or solvate thereof, which are useful for treating sialic acid deficiency.

Classes IPC  ?

• C12P 19/26 - Préparation d'hydrates de carbone contenant de l'azote
• C12P 19/44 - Préparation d'O-glucosides, p. ex. glucosides
• C07H 1/06 - SéparationPurification

Abrégé

The present disclosure generally relates to methods of protecting the genomic integrity and/or biological activity of AAV viral particles in a sample containing both AAV particles and helper virus particles during heat inactivation. The methods include heating, to a temperature greater than or equal to 45° C., a sample containing helper virus particles, AAV particles, and a buffer. The buffer includes a concentration of 10 mM or greater kosmotropic salts and/or a concentration of 10 mM or greater of divalent or trivalent cations.

Classes IPC  ?

• A61K 35/761 - Adénovirus
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
• C12N 15/86 - Vecteurs viraux

Abrégé

The present application relates to sialylated glycoprotein compositions and methods of their use in treating various conditions and disorders.

Classes IPC  ?

• A61K 38/47 - Hydrolases (3) agissant sur des composés glycosyliques (3.2), p. ex. cellulases, lactases
• A61K 9/00 - Préparations médicinales caractérisées par un aspect particulier
• A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
• C12N 9/24 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2)
• C12N 9/38 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons bêta-galactose-glycoside, p. ex. bêta-galactosidase

Abrégé

The present disclosure provides novel methods of producing soluble human microdystrophin proteins and uses of the same including for in vitro detection assays.

Classes IPC  ?

• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C12Q 1/00 - Procédés de mesure ou de test faisant intervenir des enzymes, des acides nucléiques ou des micro-organismesCompositions à cet effetProcédés pour préparer ces compositions

Abrégé

The present invention provides compositions and methods for treating a hypophosphatemic disorder, such as X-linked hypophosphatemia (XLH). The method entails administering to a subject a pharmaceutical composition containing an anti-FGF23 ligand, wherein the dosing regimen of the pharmaceutical is designed to reach effective and efficient control of FGF23 activity.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
• C07K 16/22 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des facteurs de croissance

Abrégé

e.ge.g., a sample solution that comprises dsRNA and messenger RNA (mRNA). Also provided are methods of correlating the presence of dsRNA with potential immunogenicity in a host.

Classes IPC  ?

• C12Q 1/6806 - Préparation d’acides nucléiques pour analyse, p. ex. pour test de réaction en chaîne par polymérase [PCR]
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
• A61P 35/00 - Agents anticancéreux
• A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes

Abrégé

The present disclosure provides creatine prodrug analogs and their compositions useful for the treatment of creatine deficiencies.

Classes IPC  ?

• C07C 279/22 - Y étant un atome d'hydrogène ou de carbone, p. ex. benzoylguanidines
• C07B 59/00 - Introduction d'isotopes d'éléments dans les composés organiques
• C07D 213/82 - AmidesImides en position 3
• C07D 273/02 - Composés hétérocycliques contenant des cycles comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle, non prévus par les groupes comportant deux atomes d'azote et un seul atome d'oxygène
• C07D 498/10 - Systèmes condensés en spiro

Abrégé

Provided are methods of separating full capsid particles and empty capsid particles in a viral capsid preparation by overloading viral capsid preparations onto an anion exchange medium integrated with weak partitioning chromatography and multiple column chromatography (AEX-WPC-MCC). Also provided are viral capsid loading ranges for overloading AEX medium facilitating separating full and empty capsid particles.

Classes IPC  ?

• B01D 15/16 - Adsorption sélective, p. ex. chromatographie caractérisée par des caractéristiques de structure ou de fonctionnement relatives au conditionnement du fluide vecteur
• B01D 15/18 - Adsorption sélective, p. ex. chromatographie caractérisée par des caractéristiques de structure ou de fonctionnement relatives aux différents types d'écoulement
• B01D 15/20 - Adsorption sélective, p. ex. chromatographie caractérisée par des caractéristiques de structure ou de fonctionnement relatives au conditionnement de la matière adsorbante ou absorbante
• B01D 15/36 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction ionique, p. ex. échange d'ions, paire d'ions, suppression d'ions ou exclusion d'ions
• B01D 15/42 - Adsorption sélective, p. ex. chromatographie caractérisée par le mode de développement, p. ex. par déplacement ou par élution
• B01J 41/00 - Échange d'anionsUtilisation d'une substance comme échangeur d'anionsTraitement d'une substance en vue d'améliorer ses propriétés d'échange d'anions
• C12N 7/02 - Isolement ou purification
• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN

Abrégé

This application relates to double-stranded small interfering RNAs that modulate DUX4 gene expression and describes methods of inhibiting DUX4 gene expression by contacting a cell with said double-stranded small interfering RNAs. The application further provides compositions comprising said double-stranded small interfering RNAs and their use in methods of preventing or treating a disease or disorder associated with aberrant expression of DUX4, such as facioscapulohumeral dystrophy (FSHD) or cancer, in a subject.

Classes IPC  ?

• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• A61K 47/54 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un composé organique
• A61K 47/68 - Préparations médicinales caractérisées par les ingrédients non actifs utilisés, p. ex. les supports ou les additifs inertesAgents de ciblage ou de modification chimiquement liés à l’ingrédient actif l’ingrédient non actif étant chimiquement lié à l’ingrédient actif, p. ex. conjugués polymère-médicament l’ingrédient non actif étant un agent de modification l’agent de modification étant un anticorps, une immunoglobuline ou son fragment, p. ex. un fragment Fc
• A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire

Produits et services

Pharmaceutical preparations and substances for the treatment of metabolic and rare diseases and disorders; gene therapy products for the treatment of metabolic and rare diseases and disorders. Research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of gene therapy, metabolic, and rare diseases and disorders; biomedical research in the field of gene therapy design and delivery; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; conducting clinical trials for others in the field of gene therapy design and delivery; compiling and sharing of patient data for research purposes in the field of metabolic and rare diseases and disorders; medical and scientific research information in the field of metabolic and rare diseases and disorders. Providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of metabolic and rare diseases and disorders.

Abrégé

e.g.e.g., intensified perfusion) for increasing productivity and/or cell density in viral particle production to achieve high viral particle, e.g., AAV yield.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/86 - Vecteurs viraux
• C12N 5/00 - Cellules non différenciées humaines, animales ou végétales, p. ex. lignées cellulairesTissusLeur culture ou conservationMilieux de culture à cet effet

Abrégé

This application relates to double-stranded small interfering RNAs that modulate DUX4 gene expression and describes methods of inhibiting DUX4 gene expression by contacting a cell with said double-stranded small interfering RNAs. The application further provides compositions comprising said double-stranded small interfering RNAs and their use in methods of preventing or treating a disease or disorder associated with aberrant expression of DUX4, such as facioscapulohumeral muscular dystrophy (FSHD) or cancer, in a subject.

Classes IPC  ?

• A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide
• A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire
• A61P 19/00 - Médicaments pour le traitement des troubles du squelette
• C07K 1/00 - Procédés généraux de préparation de peptides

Produits et services

(1) Pharmaceutical preparations and substances for the treatment of metabolic and rare diseases and disorders; gene therapy products for the treatment of metabolic and rare diseases and disorders. (1) Research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of gene therapy, metabolic, and rare diseases and disorders; biomedical research in the field of gene therapy design and delivery; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; conducting clinical trials for others in the field of gene therapy design and delivery; compiling and sharing of patient data for research purposes in the field of metabolic and rare diseases and disorders; medical and scientific research information in the field of metabolic and rare diseases and disorders. (2) Providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of metabolic and rare diseases and disorders.

Abrégé

The present disclosure provides compositions including recombinant nucleic acid constructs, vectors, and host cells, and methods of their use for reducing reverse packaging of cap and/or rep DMA sequences in the production of recombinant adeno-associated vims (rAAV). Also provided are pharmaceutical compositions comprising an rAAV produced from a composition or method of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the prevention or treatment of a disease, condition, or disorder in a subject.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux

Abrégé

The present invention includes solid compositions of triglycerides with one or more fatty acids, such as triheptanoin and glycerol phenylbutyrate, and therapeutic use thereof. The solid compositions can be prepared by spray-drying or other processes.

Classes IPC  ?

• A61K 31/23 - Esters, p. ex. nitroglycérine, sélénocyanates d'acides carboxyliques d'acides acycliques, p. ex. pravastatine d'acides ayant un groupe carboxyle lié à une chaîne d'au moins sept atomes de carbone
• A61K 31/20 - Acides carboxyliques, p. ex. acide valproïque ayant un groupe carboxyle lié à une chaîne acyclique d'au moins sept atomes de carbone, p. ex. acides stéarique, palmitique ou arachidique
• A61K 9/14 - Préparations médicinales caractérisées par un aspect particulier à l'état particulaire, p. ex. poudres
• A61K 9/16 - AgglomérésGranulésMicrobilles

Abrégé

This application relates to recombinant adeno-associated virus (rAAV) packaging and/or producer cell lines which have been engineered to reduce expression and/or activity of one or more genes and/or proteins to increase rAAV titers. The methods of generating the engineered cell lines have also been described herein.

Classes IPC  ?

• C12N 7/02 - Isolement ou purification
• C12N 15/64 - Méthodes générales pour la préparation du vecteur, pour son introduction dans la cellule ou pour la sélection de l'hôte contenant le vecteur
• C12N 15/86 - Vecteurs viraux

Abrégé

The invention provides methods for the production of recombinant adeno-associated virus vectors (rAAV), comprising contacting a host cell with a solution comprising at least one compound of formula (I), (I-A), (I-B), (II), (III), or (IV), or a salt thereof, or a vitamin B, or any combination(s) thereof. Also provided are methods for increasing the production of rAAV by a host cell, comprising contacting a host cell with a solution comprising at least one compound of formula (I), (I-A), (I-B), (II), (III), or (IV), or a salt thereof, or a vitamin B, or any combination(s) thereof.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux

Abrégé

This present disclosure provides recombinant adeno-associated virus (rAAV) and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a rAAV of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of PA caused by a mutation in propionyl-CoA carboxylase α-subunit (PCCA) or a mutation in propionyl-CoA carboxylase β-subunit (PCCB).

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
• C12N 9/00 - Enzymes, p. ex. ligases (6.)ProenzymesCompositions les contenantProcédés pour préparer, activer, inhiber, séparer ou purifier des enzymes

Abrégé

Methods and compositions for the treatment or prevention of amyloidosis are provided. In some embodiments, the methods comprise administering to the subject a therapeutically effective amount of at least one catabolic enzyme or a biologically active fragment thereof. Such methods and compositions may be employed to reduce, prevent, degrade and/or eliminate amyloid formation in the lysosome and/or extracellularly.

Classes IPC  ?

• A61K 38/48 - Hydrolases (3) agissant sur des liaisons peptidiques (3.4)
• A61K 38/05 - Dipeptides
• A61K 38/54 - Mélanges d'enzymes ou de proenzymes couverts par plus d'un seul des groupes ou
• A61K 31/198 - Alpha-amino-acides, p. ex. alanine ou acide édétique [EDTA]
• A61K 31/454 - Pipéridines non condensées, p. ex. pipérocaïne contenant d'autres systèmes hétérocycliques contenant un cycle à cinq chaînons avec l'azote comme hétéro-atome du cycle, p. ex. pimozide, dompéridone
• A61K 31/475 - QuinoléinesIsoquinoléines ayant un cycle indole, p. ex. yohimbine, réserpine, strychnine, vinblastine
• A61K 31/573 - Composés contenant des systèmes cycliques du cyclopenta[a]hydrophénanthrèneLeurs dérivés, p. ex. stéroïdes substitués en position 17 bêta par une chaîne à deux atomes de carbone, p. ex. prégnane ou progestérone substitués en position 21, p. ex. cortisone, dexaméthasone, prednisone ou aldostérone
• A61K 31/675 - Composés du phosphore ayant l'azote comme hétéro-atome d'un cycle, p. ex. phosphate de pyridoxal
• A61K 31/69 - Composés du bore
• A61K 31/704 - Composés ayant des radicaux saccharide liés à des composés non-saccharide par des liaisons glycosidiques liés à un composé carbocyclique, p. ex. phloridzine liés à un système carbocyclique condensé, p. ex. sennosides, thiocolchicosides, escine, daunorubicine, digitoxine
• A61K 35/12 - Substances provenant de mammifèresCompositions comprenant des tissus ou des cellules non spécifiésCompositions comprenant des cellules souches non embryonnairesCellules génétiquement modifiées
• A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
• A61K 45/06 - Mélanges d'ingrédients actifs sans caractérisation chimique, p. ex. composés antiphlogistiques et pour le cœur
• C12N 9/48 - Hydrolases (3.) agissant sur les liaisons peptidiques, p. ex. thromboplastine, aminopeptidase de la leucine (3.4)
• C12N 9/64 - Protéinases provenant de tissu animal, p. ex. rennine

Abrégé

Disclosed are methods and dosing regimens for treating a patient suffering from osteogenesis imperfecta, comprising administering to the patient a therapeutically effective amount of an anti-sclerostin antibody. The invention also provides an anti-sclerostin antibody for use in the treatment of osteogenesis imperfecta, comprising administering a therapeutically effective amount of the anti-sclerostin antibody each month according to certain dosing regimens.

Classes IPC  ?

• C07K 16/22 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des facteurs de croissance
• A61P 19/08 - Médicaments pour le traitement des troubles du squelette des maladies osseuses, p. ex. rachitisme, maladie de Paget
• A61P 19/10 - Médicaments pour le traitement des troubles du squelette des maladies osseuses, p. ex. rachitisme, maladie de Paget de l'ostéoporose

Produits et services

Printed publications, namely, books, brochures, white papers, informational pamphlets, informational cards, and advertisements being signs and boards of paper in the field of rare diseases and disorders

Produits et services

Compiling of data for research purposes in the field of rare diseases and disorders; medical and scientific research information in the field of rare diseases and disorders

Produits et services

Organizing business networking events in the field of rare diseases and disorders; organizing business networking events in the field of pharmaceutical, drug, and therapy research and development; organizing business networking events in the field of clinical trial design; organizing business networking events in the field of intellectual property and licensing

Produits et services

Education and training services, namely, seminars, classes, workshops, and providing non-downloadable webinars, for healthcare professionals, caregivers, patients, advocates, and providers in the field of rare diseases and disorders; education and training services, namely, seminars, classes, workshops, and providing non-downloadable webinars, for healthcare professionals, caregivers, patients, advocates, and providers in the field of pharmaceutical, drug, and therapy research and development; education and training services, namely, seminars, classes, workshops, and providing non-downloadable webinars, for healthcare professionals, caregivers, patients, advocates, and providers in the field of clinical trial design; education and training services, namely, seminars, classes, workshops, and providing non-downloadable webinars, for healthcare professionals, caregivers, patients, advocates, and providers in the field of intellectual property and licensing; online journals, namely, blogs featuring information on rare diseases and disorders; Educational services, namely, developing curriculum for others in the field of rare diseases and disorders

Abrégé

The present disclosure provides methods of reducing, minimizing, or inhibiting the formation of double-stranded ribonucleic acid (dsRNA) during the preparation of ribonucleic acid (RNA), such as messenger ribonucleic acid (mRNA), by adding at least one chaotropic agent to a starting reaction mixture. The present disclosure provides methods of reducing, minimizing, or inhibiting intramolecular base-pairing within a ribonucleic acid (RNA) transcript and/or intermolecular base-pairing between an RNA transcript and deoxyribonucleic acid (DNA) or another RNA during the preparation of ribonucleic acid (RNA), by adding at least one chaotropic agent to a starting reaction mixture.

Classes IPC  ?

• C07H 21/02 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le ribosyle comme radical saccharide
• C07H 1/00 - Procédés de préparation des dérivés du sucre

Produits et services

Pharmaceutical preparations and substances for the treatment of metabolic and rare metabolic and genetic diseases and disorders; gene therapy products for the treatment of metabolic and rare diseases and disorders, namely, virus vector-based gene therapies for intravenous delivery Pharmaceutical, scientific, and medical research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of gene therapy, and metabolic and rare metabolic and genetic diseases and disorders; biomedical research in the field of gene therapy design and delivery; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; scientific research in the nature of conducting clinical trials for others in the field of gene therapy design and delivery; pharmaceutical, scientific, and medical research, namely, compiling and sharing of patient data for research purposes in the field of metabolic and rare metabolic and genetic diseases and disorders; providing medical and scientific research information in the field of metabolic and rare metabolic and genetic diseases and disorders Providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of metabolic and rare metabolic and genetic diseases and disorders

Abrégé

This present disclosure provides pharmaceutical compositions for delivering recombinant adeno-associated virus (rAAV) particles, generally comprising a buffer, a monovalent salt, a polyhydric alcohol, and a triblock copolymer surfactant.

Classes IPC  ?

• A61K 35/76 - VirusParticules sous-viralesBactériophages
• A61K 35/761 - Adénovirus
• A61K 47/26 - Hydrates de carbone, p. ex. polyols ou sucres alcoolisés, sucres aminés, acides nucléiques, mono-, di- ou oligosaccharidesLeurs dérivés, p. ex. polysorbates, esters d’acide gras de sorbitan ou glycyrrhizine
• A61K 47/30 - Composés macromoléculaires organiques ou inorganiques, p. ex. polyphosphates inorganiques

Abrégé

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated vims (rAAV), and methods of their use in gene therapy for treating CDKL5 deficiency disorder (CDD). Also provided are pharmaceutical compositions comprising an rAAV of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of CDD caused by mutations in CDKL.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 25/28 - Médicaments pour le traitement des troubles du système nerveux des troubles dégénératifs du système nerveux central, p. ex. agents nootropes, activateurs de la cognition, médicaments pour traiter la maladie d'Alzheimer ou d'autres formes de démence
• A61K 35/761 - Adénovirus
• A61K 31/573 - Composés contenant des systèmes cycliques du cyclopenta[a]hydrophénanthrèneLeurs dérivés, p. ex. stéroïdes substitués en position 17 bêta par une chaîne à deux atomes de carbone, p. ex. prégnane ou progestérone substitués en position 21, p. ex. cortisone, dexaméthasone, prednisone ou aldostérone

Abrégé

Provided are methods of separating full capsid particles and empty capsid particles in a viral capsid preparation using an anion exchange medium. Methods may involve using a wash solution comprising a quaternary ammonium salt. Methods include applying a viral capsid preparation to an anion exchange medium, combined with multiple quaternary ammonium salt wash solutions.

Classes IPC  ?

• B01D 15/16 - Adsorption sélective, p. ex. chromatographie caractérisée par des caractéristiques de structure ou de fonctionnement relatives au conditionnement du fluide vecteur
• B01D 15/36 - Adsorption sélective, p. ex. chromatographie caractérisée par le mécanisme de séparation impliquant une interaction ionique, p. ex. échange d'ions, paire d'ions, suppression d'ions ou exclusion d'ions
• B01D 15/42 - Adsorption sélective, p. ex. chromatographie caractérisée par le mode de développement, p. ex. par déplacement ou par élution
• C12N 7/02 - Isolement ou purification

Abrégé

The present disclosure provides novel synthetic nucleic acids and recombinant adeno-associated virus (rAAV) comprising the same, as well as methods of their use in the treatment of muscular dystrophies associated with a dystrophin mutation. Also provided are pharmaceutical compositions comprising a novel synthetic nucleic acid or rAAV of the invention, and a pharmaceutically acceptable carrier or excipient. Pharmaceutical compositions comprising an rAAV of the invention may be useful in gene therapy for the treatment of dystrophin-associated muscular dystrophies, such as Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD), and X-linked cardiomyopathy.

Classes IPC  ?

• A61K 35/76 - VirusParticules sous-viralesBactériophages
• C07K 14/47 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains provenant de vertébrés provenant de mammifères
• C12N 15/864 - Vecteurs parvoviraux
• C12N 15/67 - Méthodes générales pour favoriser l'expression

Produits et services

printed publications, namely, books, brochures, informational pamphlets, informational cards, and advertisements being signs and boards of paper in the field of rare diseases and disorders

Produits et services

Research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of genome editing, gene editing, genome engineering, gene modulation, genetic diseases, gene therapy, and cell therapy biomedical research in the field of gene therapy design and delivery; gene therapy research, namely, design and delivery development of biochemical assays; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; scientific, medical, and pharmaceutical research in the nature of conducting clinical trials for others in the field of gene therapy design and delivery; medical and scientific research, namely, compiling and sharing of patient data for research purposes in the field of rare diseases and disorders; providing medical and scientific research information in the field of rare diseases and disorder

Produits et services

providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of liver, hepatological, cardiovascular, central nervous system, endocrine, inflammatory, metabolic, autoimmune, musculoskeletal, neurological, ophthalmologic, respiratory, and hematologic diseases and disorders

Produits et services

Downloadable software for creating, editing, uploading, downloading, accessing, viewing, posting, displaying, tagging, blogging, streaming, linking, annotating, indicating sentiment about, commenting on, embedding, transmitting, and providing electronic media information via computer, the internet and communication networks in the field of rare diseases and disorders; downloadable software for modifying and enabling transmission of images, audio, audio visual and video content and data in the field of rare diseases and disorders; downloadable computer software for use in inputting, gathering, monitoring, sharing, and analyzing patient data with respect to medical histories, health status and disease progression and treatment Licensing of intellectual property; online social networking services in the field of rare diseases and disorders

Produits et services

Chemical, biological, and biochemical preparations for use in gene delivery technology, namely, nucleic acids, including, coding and non-coding deoxyribonucleic acids, plasmids, vectors, promotors, enhancers and other regulatory sequences, for use in vitro laboratory use; chemical, biological, and biochemical preparations for use in the manufacture of pharmaceutical preparations Pharmaceutical preparations for the treatment of liver, hepatological, cardiovascular, central nervous system, inflammatory, autoimmune, musculoskeletal, ophthalmologic, respiratory, osteopathic, oncologic, endocrine, orthopedic, metabolic, lipid-related, genetic, neuromuscular, autosomal, lysosomal storage, enzyme, hematologic and long-chain fatty acid oxidation disorders (LC-FAOD) diseases and disorders; chemical, biological, and biochemical preparations for use in gene delivery technology for medical use, namely, nucleic acids, including coding and non-coding deoxyribonucleic acids, plasmids, vectors, promotors, enhancers and other regulatory sequences, for use in in vivo therapeutic use in gene therapy Printed publications, namely, books, brochures, informational pamphlets, informational cards, and advertisements being signs and boards of paper in the field of rare diseases and disorders Financial and insurance consultation services, namely, patient advisory, support and consultancy services regarding patient finances, insurance and access to medical treatment; providing financial and insurance information, namely, providing information and guidance in connection with health insurance benefit plans and other patient financial assistance resources; financial and insurance consultation services, namely, insurance and medical and pharmaceutical access consultation services, namely, assisting patients and their caregivers to identify the scope of coverage offered by patient medical and pharmaceutical insurance and to identify other financial assistance resources for payment of patient treatment; insurance consultation services, namely, assisting patients and their caregivers to obtain authorization and payment for medical and pharmaceutical costs Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of rare diseases and disorders; online journals, namely, blogs featuring information on rare diseases and disorders Pharmaceutical, scientific, and medical research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of genome editing, gene editing, genome engineering, gene modulation, genetic diseases, gene therapy, and cell therapy; biomedical research in the field of gene therapy design and delivery; gene therapy design and delivery development of biochemical assays; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; scientific research in the nature of conducting clinical trials for others in the field of gene therapy design and delivery; scientific and medical research, namely, compiling and sharing of patient data for research purposes in the field of rare diseases and disorders; medical and scientific research information in the field of rare diseases and disorders; computer services, namely, creating an on-line community for registered users to obtain feedback from their peers, interact with research data, and share data in the field of rare diseases and disorders; pharmaceutical research services, namely, recruitment of rare disease patients for panels such as pharmaceutical research panels; providing medical research and scientific research information in the field of hypophosphatemia Providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of liver, hepatological, cardiovascular, central nervous system, endocrine, inflammatory, metabolic, autoimmune, musculoskeletal, neurological, ophthalmologic, respiratory, and hematologic diseases and disorders; medical consulting services in the field of rare diseases and disorders; healthcare services, namely, providing an online database of medical and healthcare information in the field of rare diseases and disorders, and collecting data and information on rare diseases and disorders for screening, diagnosis and treatment purposes; medical information services in the nature of providing a database in the field of hypophosphatemia information based on collected patient data and information all for treatment and diagnostic purposes

Abrégé

Provided herein are methods for managing host immune responses to improve therapeutic outcomes in adeno-associated virus (AAV)-mediated gene therapy. Such methods may include administering a recombinant adeno-associated vims (rAAV) to a subject following administration of a CD 19 inhibitor, e.g., an anti-CD 19 antibody. The methods described herein can facilitate improved transgene expression, help overcome pre-existing NAbs, and/or permit redosing with the same or substantially similar rAAV or transgene.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 31/573 - Composés contenant des systèmes cycliques du cyclopenta[a]hydrophénanthrèneLeurs dérivés, p. ex. stéroïdes substitués en position 17 bêta par une chaîne à deux atomes de carbone, p. ex. prégnane ou progestérone substitués en position 21, p. ex. cortisone, dexaméthasone, prednisone ou aldostérone
• A61K 31/69 - Composés du bore
• A61K 31/675 - Composés du phosphore ayant l'azote comme hétéro-atome d'un cycle, p. ex. phosphate de pyridoxal
• A61K 31/5377 - 1,4-Oxazines, p. ex. morpholine non condensées et contenant d'autres hétérocycles, p. ex. timolol
• A61K 38/48 - Hydrolases (3) agissant sur des liaisons peptidiques (3.4)
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61K 38/45 - Transférases (2)
• A61K 38/46 - Hydrolases (3)
• A61K 38/53 - Ligases (6)
• A61K 38/44 - Oxydoréductases (1)
• A61K 38/18 - Facteurs de croissanceRégulateurs de croissance
• A61K 38/37 - Facteurs VIII
• A61K 38/36 - Facteurs de coagulation sanguine ou de fibrinolyse

Abrégé

This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL), or a fragment thereof having AGL activity. The polynucleotide and oligomer molecules are expressible to provide the human AGL or a fragment thereof having AGL activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with reduced activity of amylo-alpha-1, 6-glucosidase, 4-alpha-glucanotransferase (AGL) in a subject.

Classes IPC  ?

• C12N 9/10 - Transférases (2.)
• A61P 3/00 - Médicaments pour le traitement des troubles du métabolisme
• C12N 9/44 - Hydrolases (3.) agissant sur les composés glycosyliques (3.2) agissant sur les liaisons alpha-glucosidiques-1, 6, p. ex. iso-amylase, pullulanase
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées

Produits et services

Chemical, biological, and biochemical products for use in gene delivery technology, namely, nucleic acids, including coding and non-coding deoxyribonucleic acids, plasmids, vectors, promotors, enhancers and other regulatory sequences, for in vitro laboratory use and in vivo therapeutic use in gene therapy; chemical, biological, and biochemical products for use in the manufacture of pharmaceutical preparations. Research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of genome editing, gene editing, genome engineering, gene modulation, genetic diseases, gene therapy, and cell therapy; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; conducting clinical trials for others in the field of gene therapy design and delivery; biomedical research in the field of gene therapy design and delivery; gene therapy design and delivery; development of biochemical assays; compiling and sharing of patient data for research purposes in the field of medical science. Providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of genetic diseases and disorders; providing medical services, namely, gene therapy design and delivery services being gene therapy medical services in the field of liver, hepatological, cardiovascular, central nervous system, endocrine, inflammatory, metabolic, autoimmune, musculoskeletal, neurological, ophthalmologic, respiratory, and hematologic diseases and disorders; medical information services in the nature of providing a database in the field of gene therapy information based on collected patient data and information all for treatment and diagnostic purposes; medical information services. Licensing of intellectual property.

Abrégé

The present disclosure provides creatine prodrug analogs and their compositions useful for the treatment of creatine deficiencies.

Classes IPC  ?

• C07B 59/00 - Introduction d'isotopes d'éléments dans les composés organiques
• C07D 213/82 - AmidesImides en position 3
• C07D 273/02 - Composés hétérocycliques contenant des cycles comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle, non prévus par les groupes comportant deux atomes d'azote et un seul atome d'oxygène
• C07D 498/10 - Systèmes condensés en spiro
• C07C 279/22 - Y étant un atome d'hydrogène ou de carbone, p. ex. benzoylguanidines

Abrégé

This application provides polynucleotides comprising a coding sequence for a functionally active hereditary hemochromatosis protein (HFE) or a functionally active fragment thereof. The invention further provides compositions comprising said polynucleotides and their use in methods of preventing or treating hemochromatosis in a subject.

Classes IPC  ?

• C07K 14/74 - Complexe majeur d'histocompatibilité [MHC]
• A01K 67/027 - Nouvelles races ou races modifiées de vertébrés
• A61K 31/7088 - Composés ayant au moins trois nucléosides ou nucléotides

Abrégé

The present invention provides compositions and methods for treating tumor-induced osteomalacia. The method entails administering to a subject a pharmaceutical composition containing an anti-FGF23 ligand, wherein the dosing regimen of the pharmaceutical is designed to reach effective and efficient control of FGF23 activity.

Classes IPC  ?

• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
• C07K 16/22 - Immunoglobulines, p. ex. anticorps monoclonaux ou polyclonaux contre du matériel provenant d'animaux ou d'humains contre des facteurs de croissance

Abrégé

This application relates to adeno-associated viral vectors encoding a truncated yet functional ATP7B for use in gene therapy for treating Wilson disease (WD). The truncated ATP7B described herein has several advantages over the wild-type ATP7B such as higher efficacy and improved manufacturing yield.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes

Abrégé

The present disclosure generally relates to methods of protecting the genomic integrity and/or biological activity of AAV viral particles in a sample containing both AAV particles and helper virus particles during heat inactivation. The methods include heating, to a temperature greater than or equal to 45′C, a sample containing helper virus particles, AAV particles, and a buffer. The buffer includes a concentration of 10 mM or greater kosmotropic salts and/or a concentration of 10 mM or greater of divalent or trivalent cations.

Classes IPC  ?

• A61K 35/761 - Adénovirus
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 15/85 - Vecteurs ou systèmes d'expression spécialement adaptés aux hôtes eucaryotes pour cellules animales
• C12N 15/86 - Vecteurs viraux

Abrégé

This application relates to double-stranded small interfering RNAs that modulate DUX4 gene expression and describes methods of inhibiting DUX4 gene expression by contacting a cell with said double-stranded small interfering RNAs. The application further provides compositions comprising said double-stranded small interfering RNAs and their use in methods of preventing or treating a disease or disorder associated with aberrant expression of DUX4, such as facioscapulohumeral dystrophy (FSHD) or cancer, in a subject.

Classes IPC  ?

• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• A61P 21/00 - Médicaments pour le traitement des troubles du système musculaire ou neuromusculaire

Produits et services

Research and development in the field of gene therapy manufacturing technology; biomedical and scientific research in the fields of genome editing, gene editing, genome engineering, gene modulation, genetic diseases, gene therapy, and cell therapy; product development in the fields of genetic therapies and effectors to regulate, modulate, and characterize disease states; pharmaceutical research and development in the field of gene therapy; conducting clinical trials for others in the field of gene therapy design and delivery; biomedical research in the field of gene therapy design and delivery; gene therapy design and delivery development of biochemical assays; compiling and sharing of patient data for research purposes in the field of medical science

Abrégé

This invention provides a variety of novel adeno-associated vims (AAV) vectors for gene therapy applications in the treatment of glycogen storage disease type 1a (GSD-Ia). Disclosed herein are a number of recombinant nucleic acid molecules, vectors and recombinant AAV that incorporate a modified G6PC promoter/enhancer sequence. Utilization of the modified G6PC promoter/enhancer sequence results in enhanced AAV yield and quality when expressed from various host cell platforms. Also provided herein are compositions comprising the novel AAV of the invention and methods of treating GSD-Ia using the same.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C12N 9/16 - Hydrolases (3.) agissant sur les liaisons esters (3.1)
• C12N 15/11 - Fragments d'ADN ou d'ARNLeurs formes modifiées

Abrégé

This present disclosure provides recombinant adeno-associated virus (rAAV) and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a rAAV of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of PA caused by a mutation in propionyl-CoA carboxylase α-subunit (PCCA) or a mutation in propionyl-CoA carboxylase β-subunit (PCCB).

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C12N 15/113 - Acides nucléiques non codants modulant l'expression des gènes, p. ex. oligonucléotides anti-sens
• C12N 9/00 - Enzymes, p. ex. ligases (6.)ProenzymesCompositions les contenantProcédés pour préparer, activer, inhiber, séparer ou purifier des enzymes

Abrégé

The invention provides methods for the production of recombinant adeno-associated virus vectors (rAAV), comprising contacting a host cell with a solution comprising at least one compound of formula (I), (I- A), (I-B), (II), (III), or (IV), or a salt thereof, or a vitamin B, or any combination(s) thereof. Also provided are methods for increasing the production of rAAV by a host cell, comprising contacting a host cell with a solution comprising at least one compound of formula (I), (I-A), (I-B), (II), (III), or (IV), or a salt thereof, or a vitamin B, or any combination(s) thereof.

Classes IPC  ?

• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification
• C12N 5/00 - Cellules non différenciées humaines, animales ou végétales, p. ex. lignées cellulairesTissusLeur culture ou conservationMilieux de culture à cet effet

Abrégé

reprep DMA sequences in the production of recombinant adeno-associated vims (rAAV). Also provided are pharmaceutical compositions comprising an rAAV produced from a composition or method of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the prevention or treatment of a disease, condition, or disorder in a subject.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux

Abrégé

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (rAAV), and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a recombinant adeno-associated virus of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical (compositions may be useful in gene therapy for the treatment of PA caused by mutations in propionyl-CoA carboxylase α-subunit (PCCA) or mutations in propionyl-CoA carboxylase β-subunit (PCCB).

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 15/86 - Vecteurs viraux

Abrégé

The invention provides methods for the production of recombinant adeno-associated virus vectors (rAAV), comprising culturing producer cells in media with increased osmolality. Also provided are methods for decreasing the production of helper virus by a rAAV producer cell, comprising culturing the producer cell in media with increased osmolality.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• C12N 7/00 - Virus, p. ex. bactériophagesCompositions les contenantLeur préparation ou purification

Abrégé

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (rAAV), and methods of their use in gene therapy for treating CDKL5 deficiency disorder (CDD). Also provided are pharmaceutical compositions comprising an rAAV of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of CDD caused by mutations in CDKL.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 38/17 - Peptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'animauxPeptides ayant plus de 20 amino-acidesGastrinesSomatostatinesMélanotropinesLeurs dérivés provenant d'humains
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique

Abrégé

The present disclosure provides methods of reducing, minimizing, or inhibiting the formation of double-stranded ribonucleic acid (dsRNA) during the preparation of ribonucleic acid (RNA), such as messenger ribonucleic acid (mRNA), by adding at least one chaotropic agent to a starting reaction mixture. The present disclosure provides methods of reducing, minimizing, or inhibiting intramolecular base-pairing within a ribonucleic acid (RNA) transcript and/or intermolecular base-pairing between an RNA transcript and deoxyribonucleic acid (DNA) or another RNA during the preparation of ribonucleic acid (RNA), by adding at least one chaotropic agent to a starting reaction mixture.

Classes IPC  ?

• A61K 38/00 - Préparations médicinales contenant des peptides
• A61K 39/395 - AnticorpsImmunoglobulinesImmunsérum, p. ex. sérum antilymphocitaire
• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• A61P 35/00 - Agents anticancéreux
• A61P 43/00 - Médicaments pour des utilisations spécifiques, non prévus dans les groupes
• C07H 21/04 - Composés contenant au moins deux unités mononucléotide comportant chacune des groupes phosphate ou polyphosphate distincts liés aux radicaux saccharide des groupes nucléoside, p. ex. acides nucléiques avec le désoxyribosyle comme radical saccharide

Produits et services

Compiling data into computer databases for research purposes in the field of rare diseases and disorders; recruitment of rare disease patients for pharmaceutical research panels; collecting data and information into computer databases on rare diseases and disorders for treatment and diagnostic purposes. Providing access to a global computer network for sharing data in the field of rare diseases and disorders. Education and training services, namely, seminars, providing non-downloadable webinars, and workshops for healthcare professionals, caregivers, patients, and providers in the field of rare diseases and disorders; online publication of journals or diaries (blog services) featuring information on rare diseases and disorders. Medical and scientific research information in the field of rare diseases and disorders; computer services, namely, interactive hosting services which allow registered users to obtain feedback from their peers, interact with research data, and share data in the field of rare diseases and disorders. Providing medical and healthcare information for healthcare professionals, caregivers, patients, and providers in the field of rare diseases and disorders via a website; medical consulting services in the field of rare diseases and disorders; healthcare services, namely, providing medical and healthcare information in the field of rare diseases and disorders via website; providing medical and healthcare information concerning the screening, diagnosis, and treatment of rare diseases and disorders.

Produits et services

Clinical diagnostic reagents for medical and veterinary use, including in vitro diagnostic reagents.

Abrégé

Provided herein are methods for managing host immune responses to improve therapeutic outcomes in adeno-associated virus (AAV)-mediated gene therapy. Such methods may include administering a recombinant adeno-associated virus (rAAV) to a subject following administration of a CD 19 inhibitor, e.g., an anti-CD 19 antibody. The methods described herein can facilitate improved transgene expression, help overcome pre-existing NAbs, and/or permit redosing with the same or substantially similar rAAV or transgene.

Classes IPC  ?

• C12N 15/86 - Vecteurs viraux
• A61K 39/00 - Préparations médicinales contenant des antigènes ou des anticorps
• A61K 31/573 - Composés contenant des systèmes cycliques du cyclopenta[a]hydrophénanthrèneLeurs dérivés, p. ex. stéroïdes substitués en position 17 bêta par une chaîne à deux atomes de carbone, p. ex. prégnane ou progestérone substitués en position 21, p. ex. cortisone, dexaméthasone, prednisone ou aldostérone

Abrégé

The present disclosure provides creatine prodrug analogs and their compositions useful for the treatment of creatine deficiencies.

Classes IPC  ?

• C07B 59/00 - Introduction d'isotopes d'éléments dans les composés organiques
• C07D 213/82 - AmidesImides en position 3
• C07D 273/02 - Composés hétérocycliques contenant des cycles comportant des atomes d'azote et d'oxygène comme uniques hétéro-atomes du cycle, non prévus par les groupes comportant deux atomes d'azote et un seul atome d'oxygène
• C07D 498/10 - Systèmes condensés en spiro
• C07C 279/22 - Y étant un atome d'hydrogène ou de carbone, p. ex. benzoylguanidines

Abrégé

This application provides polynucleotides comprising a coding sequence for a functionally active hereditary hemochromatosis protein (HFE) or a functionally active fragment thereof. The invention further provides compositions comprising said polynucleotides and their use in methods of preventing or treating hemochromatosis in a subject.

Classes IPC  ?

• A61K 48/00 - Préparations médicinales contenant du matériel génétique qui est introduit dans des cellules du corps vivant pour traiter des maladies génétiquesThérapie génique
• C12N 15/90 - Introduction stable d'ADN étranger dans le chromosome
• C12N 5/071 - Cellules ou tissus de vertébrés, p. ex. cellules humaines ou tissus humains
• C12N 15/10 - Procédés pour l'isolement, la préparation ou la purification d'ADN ou d'ARN