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1. WO2020194320 - COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS

Publication Number WO/2020/194320
Publication Date 01.10.2020
International Application No. PCT/IL2020/050381
International Filing Date 29.03.2020
IPC
A61K 31/7088 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
70Carbohydrates; Sugars; Derivatives thereof
7088Compounds having three or more nucleosides or nucleotides
C12N 15/113 2010.01
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
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A61P 11/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
11Drugs for disorders of the respiratory system
A61K 31/00 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31Medicinal preparations containing organic active ingredients
Applicants
  • SPLISENSE LTD. [IL]/[IL]
Inventors
  • OREN, Yifat
  • AVITZUR-BARCHAD, Ofra
  • OZERI-GALAI, Efrat
Agents
  • KESTEN, Dov
  • GEYRA, Assaf
Priority Data
62/825,24228.03.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS
(FR) COMPOSITIONS ET PROCÉDÉS DESTINÉS AU TRAITEMENT DE LA FIBROSE KYSTIQUE
Abstract
(EN)
The present invention is directed to a method for treating cystic fibrosis (CF) using a splicing modulator, such as an antisense oligonucleotide, capable of inducing the skipping of exon 23, exon 24, or both, of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA. Also provided are a composition and a kit comprising the splicing modulator, and a method of producing thereof.
(FR)
La présente invention concerne un procédé de traitement de la fibrose kystique (CF) à l'aide d'un modulateur d'épissage, tel qu'un oligonucléotide antisens, pouvant induire le saut de l'exon 23, de l'exon 24, ou des deux, du pré-ARNm du régulateur de la conductance transmembranaire de la fibrose kystique (CFTR). L'invention concerne également une composition et un kit comprenant le modulateur d'épissage, et un procédé de production de ceux-ci.
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