CRISPR/Cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa

Patent metadata

Field	Value
Publication number	US-12545912-B2
Application number	US-202117341244-A
Country	US
Kind code	B2
Filing date	Jun 7, 2021
Priority date	Mar 5, 2014
Publication date	Feb 10, 2026
Grant date	Feb 10, 2026

Abstract

CRISPR/Cas-related compositions and methods for treatment of Usher Syndrome and/or Retinitis Pigmentosa are disclosed herein.

First claim

What is claimed is:

1 . A guide RNA (gRNA) molecule comprising a targeting domain which is complementary with a target domain from the USH2A gene, wherein the target domain is within 200 nucleotides of a guanine deletion at nucleotide position 2299 (2299delG) in the USH2A gene, wherein the targeting domain comprises SEQ ID NO: 938.

2 . The gRNA molecule of claim 1 , wherein the gRNA molecule is a modular gRNA molecule or a chimeric gRNA molecule.

3 . The gRNA molecule of claim 1 , wherein the targeting domain comprises 17 nucleotides to 26 nucleotides in length.

4 . A nucleic acid comprising (a) a sequence encoding a first guide RNA (gRNA) molecule comprising a first targeting domain which is complementary with a first target domain from the USH2A gene, wherein the first target domain is within 200 nucleotides of a guanine deletion at nucleotide position 2299 (2299delG) in the USH2A gene, wherein the targeting domain comprises SEQ ID NO: 938.

5 . The nucleic acid of claim 4 , wherein the first gRNA molecule is a modular gRNA molecule or a chimeric gRNA molecule.

6 . The nucleic acid of claim 4 , wherein the first targeting domain comprises 17 nucleotides to 26 nucleotides in length.

7 . The nucleic acid of claim 4 , further comprising: (b) a sequence that encodes a Cas molecule.

8 . The nucleic acid of claim 7 , wherein the Cas molecule is a Cas9 molecule.

9 . The nucleic acid of claim 7 , further comprising (c) a sequence that encodes a second gRNA molecule comprising a second targeting domain that is complementary to a second target domain of the USH2A gene.

10 . The nucleic acid of claim 9 , further comprising (d) a template nucleic acid.

11 . A composition comprising a first guide RNA (gRNA) molecule comprising a first targeting domain which is complementary with a first target domain from the USH2A gene, wherein the first target domain is within 200 nucleotides of a guanine deletion at nucleotide position 2299 (2299delG) in the USH2A gene, wherein the targeting domain comprises SEQ ID NO: 938.

12 . The composition of claim 11 , further comprising a Cas molecule.

13 . The composition of claim 11 , further comprising a nucleic acid encoding a Cas molecule.

14 . The composition of claim 13 , wherein the Cas molecule is a Cas9 molecule.

15 . The composition of claim 14 , further comprising (d) a template nucleic acid.

16 . The composition of claim 11 , wherein the targeting domain comprises 17 nucleotides to 26 nucleotides in length.

17 . A nucleic acid comprising a nucleotide sequence that is complementary with a first target domain from the USH2A gene, wherein the first target domain is within 200 nucleotides of a guanine deletion at nucleotide position 2299 (2299delG) in the USH2A gene, wherein the nucleotide sequence comprises SEQ ID NO: 938.

18 . The nucleic acid of claim 17 , wherein the nucleotide sequence comprises 17 nucleotides to 26 nucleotides in length.

19 . The nucleic acid of claim 18 , wherein the nucleic acid comprises one or more nucleotide modifications and wherein the one or more nucleotide modifications comprises a sugar modification, a phosphate backbone modification, or a combination thereof.

20 . The nucleic acid of claim 19 , wherein the one or more nucleotide modifications is a phosphorothioate modification, a 2′-O-methyl modification, a 2′-O-methoxyethyl modification, or a combination thereof.

Assignees

• Editas Medicine Inc

Inventors

• Maeder Morgan L
• Bumcrot David A

Classifications

• C12Y301/00

  Hydrolases acting on ester bonds (3.1) · CPC title

• C12N2750/14143

  viral genome or elements thereof as genetic vector · CPC title

• C12N9/22

  Ribonucleases {[RNase]; Deoxyribonucleases [DNase]} · CPC title

• C12N7/00

  Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof (preparing medicinal viral antigen or antibody compositions, e.g. virus vaccines, A61K39/00) · CPC title

• A61K47/26

  Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin · CPC title