Patents Assigned to SpliSense Ltd.
  • COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS
    Publication number: 20230142669
    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.
    Type: Application
    Filed: March 25, 2021
    Publication date: May 11, 2023
    Applicant: SpliSense Ltd.
    Inventors: Yifat OREN, Ofra BARCHAD-AVITZUR, Efrat OZERI-GALAI
  • COMBINATION TREATMENTS FOR CYSTIC FIBROSIS CHARACTERIZED BY A 3849 + 10KB C-TO-T CFTR MUTATION
    Publication number: 20220220486
    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.
    Type: Application
    Filed: March 31, 2022
    Publication date: July 14, 2022
    Applicants: YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM, SPLISENSE LTD.
    Inventors: Bat Sheva KEREM, Efrat OZERI-GALAI, Yifat OREN, Ofra BARCHAD-AVITZUR
  • RESTORATION OF THE CFTR FUNCTION BY SPLICING MODULATION
    Publication number: 20220213479
    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.
    Type: Application
    Filed: May 5, 2020
    Publication date: July 7, 2022
    Applicants: YISSUM RESEARCH DEVELOPMENT COMPANY OF THE HEBREW UNIVERSITY OF JERUSALEM, SpliSense Ltd.
    Inventors: Bat Sheva KEREM, Efrat OZERI-GALAI, Yifat OREN, Ofra BARCHAD-AVITZUR
  • COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS
    Publication number: 20220064647
    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.
    Type: Application
    Filed: March 29, 2020
    Publication date: March 3, 2022
    Applicant: SpliSense Ltd.
    Inventors: Yifat OREN, Ofra BARCHAD-AVITZUR, Efrat OZERI-GALAI
  • COMPOSITIONS AND METHODS FOR TREATING CYSTIC FIBROSIS
    Publication number: 20220040219
    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 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.
    Type: Application
    Filed: March 29, 2020
    Publication date: February 10, 2022
    Applicants: SpliSense Ltd., THE HOSPITAL FOR SICK CHILDREN
    Inventors: Christine BEAR, Onofrio LASELVA, Steven MOLINSKI, Yifat OREN, Ofra AVITZUR-BARCHAD, Efrat OZERI-GALAI