Title:
RESTORATION OF THE CFTR FUNCTION BY SPLICING MODULATION
Document Type and Number:
WIPO Patent Application WO/2020/225813
Kind Code:
A8
Abstract:
The present invention provides oligonucleotides capable of binding to and modulating the splicing of the pre-mRNA of the CFTR gene, including compositions comprising the oligonucleotides, and uses thereof, such as for suppressing the inclusion of a cryptic exon between exon 22 and 23 as a result of the mutation 3849 +10Kb C-to-T, optionally in combination with additional CF therapeutics.
Inventors:
KEREM BAT SHEVA (IL)
OZERI GALAI EFRAT (IL)
OREN YIFAT (IL)
BARCHAD-AVITZUR OFRA (IL)
OZERI GALAI EFRAT (IL)
OREN YIFAT (IL)
BARCHAD-AVITZUR OFRA (IL)
Application Number:
PCT/IL2020/050495
Publication Date:
November 17, 2022
Filing Date:
May 05, 2020
Export Citation:
Assignee:
YISSUM RES DEV CO OF HEBREW UNIV JERUSALEM LTD (IL)
SPLISENSE LTD (IL)
SPLISENSE LTD (IL)
International Classes:
C12N15/113; A61K31/00; A61K31/7088; A61P11/00
Attorney, Agent or Firm:
COHEN, Mark S. et al. (IL)
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