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1. WO2020102723 - COMPOSITIONS AND METHODS FOR TREATING WILSON'S DISEASE

Publication Number WO/2020/102723
Publication Date 22.05.2020
International Application No. PCT/US2019/061808
International Filing Date 15.11.2019
IPC
C12N 9/14 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
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
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
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
Applicants
  • ENCODED THERAPEUTICS, INC. [US]/[US]
Inventors
  • RAMAMOORTHI, Kartik
  • TAGLIATELA, Stephanie
  • TANENHAUS, Anne
  • YOUNG, Andrew
  • CHEN, Szu-Ying
  • ZHANG, Chi
  • MARTIN, Stephanie
  • OBERKOFLER, David
  • WONG, Victoria
  • LI, Jianmin
Agents
  • BARKER, Adrian
Priority Data
62/768,74416.11.2018US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR TREATING WILSON'S DISEASE
(FR) COMPOSITIONS ET MÉTHODES POUR LE TRAITEMENT DE LA MALADIE DE WILSON
Abstract
(EN)
Provided herein are nucleic acid molecules and vectors comprising variant coppertransporting ATPase 2 (ATP7B) nucleic acid sequences. Such sequences have been optimized for expression in mammalian cells, liver cells, and/or from an adeno associated viral vector (AAV), including truncated and/or codon optimized variants. Also provided are viral vectors comprising such ATP7B variant nucleic acid sequences, and methods of use thereof for treating disorders associated with an ATP7B deficiency, such as Wilsons disease.
(FR)
L'invention concerne des molécules d'acide nucléique et des vecteurs comprenant des séquences d'acides nucléiques d'ATPase 2 de transport du cuivre (ATP7B) variant. De telles séquences ont été optimisées pour l'expression dans des cellules de mammifères, des cellules hépatiques, et/ou à partir d'un vecteur viral adéno-associé (AAV), comprenant des variants tronqués et/ou à codon optimisé. L'invention concerne également des vecteurs viraux comprenant de telles séquences d'acides nucléiques variants d'ATP7B, et des procédés d'utilisation correspondants pour traiter des troubles associés à une déficience d'ATP7B, telle que la maladie de Wilson.
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