IP Library › Granted Patent US 12,545,912
Granted Patent B2
US 12,545,912 · App. 17/341,244 · Granted Feb 10, 2026

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

Inventors: Morgan L. Maeder (Jamaica Plain, MA); David A. Bumcrot (Belmont, MA)
Assignee: EDITAS MEDICINE, INC.
C12N15/113A61K38/465A61K47/26C12N7/00C12N9/22C12Y301/00C12N2310/10C12N2310/20C12N2320/34C12N2750/14143
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Quick Facts
Patent No.
US 12,545,912
App. No.
17/341,244
Granted
Feb 10, 2026
Kind
B2
Abstract

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

Claims (20)

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.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 15, 2021
From: MAEDER, MORGAN L.; BUMCROT, DAVID A.
To: EDITAS MEDICINE, INC.
Reel/Frame 056548/0929 →
Continuity (3)
Continuation 15123576
Provisional Application 61948520 · Mar 5, 2014
Related Publication 20220135970A1 · May 5, 2022
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