公开(公告)号：US20220064647A1

公开(公告)日：2022-03-03

申请号：US17598274

申请日：2020-03-29

申请人： SpliSense Ltd.

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

IPC分类号： C12N15/113 ,  A61K45/06

摘要： 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.

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.

公开(公告)号：US20230142669A1

公开(公告)日：2023-05-11

申请号：US17911665

申请日：2021-03-25

申请人： SpliSense Ltd.

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

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/47 ,  A61K45/06 ,  A61K31/443

CPC分类号： C12N15/1138 ,  A61K31/712 ,  A61K31/47 ,  A61K45/06 ,  A61K31/443 ,  C12N2310/11 ,  C12N2320/33

摘要： The present invention is directed to a method for inducing skipping of exon 24 of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA. Further, treating cystic fibrosis (CF) using a splicing modulator, such as an antisense oligonucleotide, capable of inducing the skipping of exon 24 of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA. Also provided are a composition and a kit comprising the splicing modulator.

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.