公开(公告)号：US20220064647A1

公开(公告)日：2022-03-03

申请号：US17598274

申请日：2020-03-29

Applicant: SpliSense Ltd.

Inventor： Yifat OREN ,  Ofra BARCHAD-AVITZUR ,  Efrat OZERI-GALAI

IPC: C12N15/113 ,  A61K45/06

Abstract: 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：US20240352461A1

公开(公告)日：2024-10-24

申请号：US18765286

申请日：2024-07-07

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

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

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

CPC classification number: C12N15/113 ,  A61K31/7088 ,  A61K45/06 ,  A61P11/00 ,  C12N2310/11 ,  C12N2320/31

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

公开(公告)号：US20220220486A1

公开(公告)日：2022-07-14

申请号：US17709485

申请日：2022-03-31

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

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

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

Abstract: 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：US20240409930A1

公开(公告)日：2024-12-12

申请号：US18669584

申请日：2024-05-21

Applicant: SpliSense Ltd.

Inventor： Gili HART ,  Efrat OZERI-GALAI ,  Yifat OREN ,  Ofra BARCHAD-AVITZUR ,  Chava STAMPFER

IPC: C12N15/113 ,  A61K45/06 ,  A61P11/00

Abstract: The present invention provides specific synthetic oligonucleotides, as well as vectors, cells, and pharmaceutical compositions comprising the oligonucleotides, and their use in methods of treating, suppressing, inhibiting, ameliorating, or slowing progression of a lung disease or disorder, such as chronic obstructive pulmonary disease (COPD), asthma, idiopathic pulmonary fibrosis (IPF), and non-cystic fibrosis bronchiectasis (NCFB).

公开(公告)号：US20230142669A1

公开(公告)日：2023-05-11

申请号：US17911665

申请日：2021-03-25

Applicant: SpliSense Ltd.

Inventor： Yifat OREN ,  Ofra BARCHAD-AVITZUR ,  Efrat OZERI-GALAI

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

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

Abstract: 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

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

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

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

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