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1. WO2021081401 - METHODS FOR TREATING PATIENTS HAVING CFH MUTATIONS WITH CFH-ENCODING VECTORS

Publication Number WO/2021/081401
Publication Date 29.04.2021
International Application No. PCT/US2020/057161
International Filing Date 23.10.2020
IPC
A61K 48/00 2006.01
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
C07K 14/47 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
46from vertebrates
47from mammals
C12N 15/86 2006.01
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
C12N 15/864 2006.01
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
864Parvoviral vectors
Applicants
  • GEMINI THERAPEUTICS INC. [US]/[US]
Inventors
  • MCLAUGHLIN, James
  • HUANG, Lisa
  • KATTI, Suresh
  • STRAPPS, Walter
  • DECK, Melissa
Agents
  • XU, Lily
  • SHUSTER, Michael, J.
  • HUBL, Susan
  • KABLER, Kevin
  • HURT, Nicholas, S.
Priority Data
62/925,07323.10.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) METHODS FOR TREATING PATIENTS HAVING CFH MUTATIONS WITH CFH-ENCODING VECTORS
(FR) PROCÉDÉS DE TRAITEMENT DE PATIENTS AYANT DES MUTATIONS DE CFH AVEC DES VECTEURS DE CODAGE DE CFH
Abstract
(EN)
The present disclosure provides methods for treating, preventing, or inhibiting diseases in patients having one or more mutations in complement factor H (CFH), complement component 3 (C3), and complement factor B (CFB) by administering to the patients a recombinant adeno-associated virus (rAAV) vector encoding a CFH polypeptide or biologically active fragment and/or variant thereof.
(FR)
La présente invention concerne des méthodes de traitement, de prévention ou d'inhibition de maladies chez des patients présentant une ou plusieurs mutations du facteur de complément H (CFH), du composant de complément 3 (C3) et du facteur de complément B (CFB) par l'administration aux patients d'un vecteur de virus adéno-associé recombinant (rAAV) codant pour un polypeptide de CFH ou un fragment biologiquement actif et/ou un variant de celui-ci.
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