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1. WO2022011151 - MYBPC3 POLYPEPTIDES AND USES THEREOF

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Publication Number WO/2022/011151
Publication Date 13.01.2022
International Application No. PCT/US2021/040906
International Filing Date 08.07.2021
IPC
A61K 38/17 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
A61K 48/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
48Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
A61P 9/06 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
9Drugs for disorders of the cardiovascular system
06Antiarrhythmics
C12N 15/86 2006.1
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
79Vectors or expression systems specially adapted for eukaryotic hosts
85for animal cells
86Viral vectors
CPC
A01K 2217/072
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2217Genetically modified animals
07Animals genetically altered by homologous recombination
072maintaining or altering function, i.e. knock in
A01K 2217/075
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2217Genetically modified animals
07Animals genetically altered by homologous recombination
075inducing loss of function, i.e. knock out
A01K 2227/105
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2227Animals characterised by species
10Mammal
105Murine
A01K 2267/0306
AHUMAN NECESSITIES
01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
2267Animals characterised by purpose
03Animal model, e.g. for test or diseases
0306Animal model for genetic diseases
A61K 35/761
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
66Microorganisms or materials therefrom
76Viruses; Subviral particles; Bacteriophages
761Adenovirus
A61K 38/1709
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
17from animals; from humans
1703from vertebrates
1709from mammals
Applicants
  • CHILDREN'S MEDICAL CENTER CORPORATION [US]/[US]
Inventors
  • PU, William, T.
  • LU, Fujian
  • BEZZERIDES, Vassilios
Agents
  • EL-HAYEK, Roque
  • BAKER, C., Hunter
  • AMUNDSEN, Eric, L.
  • ATTISHA, Michael, J.
  • ALAM, Saad
Priority Data
63/049,39808.07.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) MYBPC3 POLYPEPTIDES AND USES THEREOF
(FR) POLYPEPTIDES MYBPC3 ET LEURS UTILISATIONS
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Abstract
(EN) Provided herein are compositions and methods for treating a disorder associated with abnormal RYR2 function (e.g., arrhythmia or heart failure). In some embodiments, method comprises administering to a subject in need thereof an effective amount of a polypeptide comprising a C -terminal domain of Cardiac Myosin binding protein C (MYBPC3) or a nucleic acid or an rAAV encoding such polypeptide.
(FR) L'invention concerne des compositions et des méthodes de traitement d'un trouble associé à une fonction RYR2 anormale (par exemple, une arythmie ou une insuffisance cardiaque). Selon certains modes de réalisation, la méthode consiste à administrer à un sujet qui en a besoin une quantité efficace d'un polypeptide comprenant un domaine de terminaison C de la protéine C de liaison à la myosine cardiaque (MYBPC3) ou un acide nucléique ou un VAAr codant pour un tel polypeptide.
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