Materials and methods for treatment of Usher syndrome type 2A and/or non-syndromic autosomal recessive retinitis pigmentosa (ARRP)

Patent metadata

Field	Value
Publication number	US-11118177-B2
Application number	US-202016905258-A
Country	US
Kind code	B2
Filing date	Jun 18, 2020
Priority date	Dec 21, 2017
Publication date	Sep 14, 2021
Grant date	Sep 14, 2021

Abstract

The present application provides materials and methods for treating a patient with one or more of Usher Syndrome Type 2A and ARRP, both ex vivo and in vivo; materials and methods for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299. In addition, the present application provides one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; a therapeutic comprising at least one or more gRNAs or sgRNAs for editing an USH2A gene containing a guanine deletion at nucleotide position c.2299; and a therapeutic for treating a patient with one or more of Usher Syndrome Type 2A and ARRP. The present application also provides a kit for treating a patient with one or more of Usher Syndrome Type 2A and ARRP.

First claim

What is claimed is:

1. A composition comprising a first gRNA and a second gRNA, wherein the first gRNA and the second gRNA are selected from the group consisting of:
(a) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5313;
(b) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5314;
(c) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5313;
(d) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5314;
(e) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5276;
(f) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5275;
(g) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5277;
(h) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5278;
(i) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5287;
(j) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5286;
(k) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5290;
(I) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5291;
(m) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5292;
(n) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5294;
(o) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5296;
(p) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5302;
(q) a first gRNA comprising SEQ ID NO: 5295 and a second gRNA comprising SEQ ID NO: 5310;
(r) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5276;
(s) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5275;
(t) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5277;
(u) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5278;
(v) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5287;
(w) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5286;
(x) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5290;
(y) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5291;
(z) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5292;
(aa) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5294;
(bb) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5296;
(cc) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5302; and
(dd) a first gRNA comprising SEQ ID NO: 5299 and a second gRNA comprising SEQ ID NO: 5310.

2. The composition of claim 1 , wherein the gRNAs are sgRNAs.

Assignees

• Crispr Therapeutics Ag
• Bayer Healthcare Llc

Inventors

• Allocca Mariacarmela
• Noma Akiko
• Scaria Abraham

Classifications

• A61P27/02

  Ophthalmic agents · CPC title

• C12N15/11 Primary

  DNA or RNA fragments; Modified forms thereof (DNA or RNA not used in recombinant technology, C07H21/00); {Non-coding nucleic acids having a biological activity} · CPC title

• A61K38/00

  Medicinal preparations containing peptides (peptides containing beta-lactam rings A61K31/00; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, A61K31/00; ergot alkaloids of the cyclic peptide type A61K31/48; containing macromolecular compounds having statistically distributed amino acid units A61K31/74; medicinal preparations containing antigens or antibodies A61K39/00; medicinal preparations characterised by the non-active ingredients, e.g. peptides as drug carriers, A61K47/00) · CPC title

• C12N15/86

  Viral vectors · CPC title

• C12N2750/14132

  Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent · CPC title