US 12,351,803 B2
Restoration of the CFTR function by splicing modulation
Bat Sheva Kerem, Mevaseret Zion (IL); Efrat Ozeri-Galai, Jerusalem (IL); Yifat Oren, Jerusalem (IL); and Ofra Barchad-Avitzur, Jerusalem (IL)
Assigned to YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD., Jerusalem (IL); and SpliSense Ltd., Jerusalem (IL)
Filed by YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD., Jerusalem (IL); and SpliSense Ltd., Jerusalem (IL)
Filed on Jul. 7, 2024, as Appl. No. 18/765,286.
Application 18/765,286 is a continuation of application No. 17/607,908, previously published as PCT/IL2020/050495, filed on May 5, 2020.
Claims priority of provisional application 62/843,469, filed on May 5, 2019.
Prior Publication US 2024/0352461 A1, Oct. 24, 2024
This patent is subject to a terminal disclaimer.
Int. Cl. C12N 15/113 (2010.01); A61K 31/7088 (2006.01); A61K 45/06 (2006.01); A61P 11/00 (2006.01)
CPC C12N 15/113 (2013.01) [A61K 31/7088 (2013.01); A61K 45/06 (2013.01); A61P 11/00 (2018.01); C12N 2310/11 (2013.01); C12N 2320/31 (2013.01)] 13 Claims
 
1. A synthetic oligonucleotide molecule complementary to a pre-mRNA transcript of a cystic fibrosis transmembrane conductance regulator (CFTR) gene having a 3849+10Kb C-to-T mutation, wherein the sequence of said synthetic oligonucleotide molecule consists of the sequence as set forth in SEQ ID NO: 1 or SEQ ID NO: 40 and characterized by increasing the percentage of correctly spliced mature CFTR mRNA by at least 10%; and decreasing the level of aberrantly spliced mature CFTR mRNA by at least 20%.