Compositions and methods for treating cystic fibrosis
AssigneeSpliSense Ltd.
InventorsYifat OREN, Ofra BARCHAD-AVITZUR, Efrat OZERI-GALAI
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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.
Claims25
A method for inducing the skipping of exon 24 of the cystic fibrosis transmembrane conductance regulator (CFTR) pre-mRNA in a cell or a method for treating cystic fibrosis (CF) in a subject in need thereof, comprising contacting said cell with or administering to said subject an effective amount of a synthetic antisense oligonucleotide (ASO) comprising 14-24 or 17-22 contiguous nucleobases having at least 75% complementary to an equal-length portion of a nucleic acid sequence from SEQ ID NO:
- 1, thereby inducing the skipping of exon 24 of the CFTR pre-mRNA in said cell.
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A pharmaceutical composition comprising a synthetic antisense oligonucleotide (ASOI) comprising 17 to 22 contiguous nucleobases having at least 80% complementary to an equal-length portion of a nucleic acid sequence from SEQ ID NO:
- 1 and a pharmaceutically acceptable carrier.
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A kit comprising:
- a. at least one synthetic antisense oligonucleotide (ASO);
- and at least one of:
- b. at least one CFTR modifier;
- or
- c. at least one CF drug,
- wherein said ASO is selected from the group consisting of SEQ ID NOs:
- 2-16, and said CFTR modifier is a CFTR potentiator, a CFTR corrector, a Translational Read-Through agent, or a CFTR amplifier.
Dependencies are inferred from the claim text.