专利检索 ap:("YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD." OR "SpliSense Ltd.") AND inv:"Bat Sheva KEREM" 第 1 页

1.

发明申请
RESTORATION OF THE CFTR FUNCTION BY SPLICING MODULATION 有权

公开(公告)号：US20220213479A1

公开(公告)日：2022-07-07

申请号：US17607908

申请日：2020-05-05

申请人： YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM LTD. , SpliSense Ltd.

发明人： Bat Sheva KEREM , Efrat OZERI-GALAI , Yifat OREN , Ofra BARCHAD-AVITZUR

IPC分类号： C12N15/113 , A61K31/7088 , A61K45/06 , A61P11/00

摘要： 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 +10 Kb C-to-T, optionally in combination with additional CF therapeutics.

2.

发明申请
COMBINATION TREATMENTS FOR CYSTIC FIBROSIS CHARACTERIZED BY A 3849 + 10KB C-TO-T CFTR MUTATION 有权

公开(公告)号：US20220220486A1

公开(公告)日：2022-07-14

申请号：US17709485

申请日：2022-03-31

申请人： YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM , SPLISENSE LTD.

发明人： Bat Sheva KEREM , Efrat OZERI-GALAI , Yifat OREN , Ofra BARCHAD-AVITZUR

IPC分类号： C12N15/113 , A61K31/4439 , A61K31/404 , A61K31/47 , A61P11/00

摘要： The present invention provides methods for treating Cystic Fibrosis (CF) and methods for suppressing the inclusion of a cryptic exon between exon 22 and 23 as a result of the mutation 3849+10 Kb C-to-T comprising the step of administering a pharmaceutical composition comprising synthetic oligonucleotides complementary to a region of the CFTR comprising the 3849+10 Kb C-to-T mutation oligonucleotides and a composition comprising one or more CFTR modifiers.