Processing

Please wait...

Settings

Settings

Goto Application

1. WO2020094693 - CODON-OPTIMIZED ABCB11 TRANSGENE FOR THE TREATMENT OF PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2 (PFIC2)

Publication Number WO/2020/094693
Publication Date 14.05.2020
International Application No. PCT/EP2019/080341
International Filing Date 06.11.2019
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
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
Applicants
  • VIVET THERAPEUTICS [FR]/[FR]
Inventors
  • GONZALEZ ASEGUINOLAZA, Gloria
  • SMERDOU, Cristian
  • PALOMO DÍAZ, Laura
Agents
  • PLASSERAUD IP
Priority Data
18306458.307.11.2018EP
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) CODON-OPTIMIZED ABCB11 TRANSGENE FOR THE TREATMENT OF PROGRESSIVE FAMILIAL INTRAHEPATIC CHOLESTASIS TYPE 2 (PFIC2)
(FR) TRANSGÈNE ABCB11 À CODONS OPTIMISÉS POUR LE TRAITEMENT DE LA CHOLESTASE INTRAHÉPATIQUE FAMILIALE DE TYPE 2 (PFIC2)
Abstract
(EN)
The present disclosure relates to gene therapy vector for use in the treatment of progressive familial intrahepatic cholestasis type 2. More specifically, the present invention relates to an adeno-associated virus vector comprising codon-optimized sequence encoding for the BSEP for the treatment of PFIC2.
(FR)
La présente invention concerne un vecteur de thérapie génique destiné à être utilisé dans le traitement du type 2 de cholestase intrahépatique familiale progressive. Plus spécifiquement, la présente invention concerne un vecteur de virus adéno-associé comprenant un codage de séquence à codon optimisé pour le BSEP pour le traitement de PFIC2.
Latest bibliographic data on file with the International Bureau