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1. WO2021055609 - MRNA ENCODING ENGINEERED CFTR

Publication Number WO/2021/055609
Publication Date 25.03.2021
International Application No. PCT/US2020/051277
International Filing Date 17.09.2020
IPC
C12N 15/00 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
A61K 38/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
C07K 14/435 2006.01
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
CPC
A61K 31/47
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
33Heterocyclic compounds
395having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
435having six-membered rings with one nitrogen as the only ring hetero atom
47Quinolines; Isoquinolines
A61K 38/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
A61P 11/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
C07K 14/435
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
C12N 15/00
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
Applicants
  • TRANSLATE BIO, INC. [US]/[US]
Inventors
  • WOOSTER, Richard
  • DEROSA, Frank
  • BOEGLIN, Lianne
  • NANDURI, Priyaanka
  • DIAS, Anusha
  • TRAN, Khang Anh
Agents
  • KIM, Sang-A
  • BAUER, Steven
  • SHALEK, James
  • COUSINS, Abigail
  • MORIN, Randall
Priority Data
62/903,04720.09.2019US
62/984,63203.03.2020US
63/021,26307.05.2020US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) MRNA ENCODING ENGINEERED CFTR
(FR) ARNM CODANT POUR CFTR MODIFIÉE
Abstract
(EN)
The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding an engineered or mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.
(FR)
La présente invention concerne, entre autres, des méthodes et des compositions pharmaceutiques améliorées pour traiter la fibrose kystique sur la base d'un ARNm à codon optimisé codant pour une protéine régulatrice de la conductance transmembranaire de la fibrose kystique (CFTR) génétiquement modifiée ou mutante.
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