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1. WO2022011151 - MYBPC3 POLYPEPTIDES AND USES THEREOF

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
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
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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