Gene therapy for ocular disorders

Patent metadata

Field	Value
Publication number	US-12403204-B2
Application number	US-202117369525-A
Country	US
Kind code	B2
Filing date	Jul 7, 2021
Priority date	Dec 14, 2015
Publication date	Sep 2, 2025
Grant date	Sep 2, 2025

Abstract

Compositions and methods are provided for treating ocular disorders in a subject are provided. In one aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGA3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding CNGB3. In another aspect, an adeno-associated viral vector is provided which includes a nucleic acid molecule comprising a sequence encoding REP-1. In desired embodiments, the subject is human, cat, dog, sheep, or non-human primate.

First claim

What is claimed is:

1. A method for treating achromatopsia in a human subject in need thereof, said method comprising administering to the human subject subretinally an AAV vector comprising an AAV capsid and a nucleic acid sequence packaged therein, wherein the nucleic acid sequence comprises i) AAV ITR sequences, ii) a nucleic acid sequence encoding human cyclic nucleotide gated channel alpha 3 (CNGA3), and iii) expression control sequences that direct expression of the CNGA3 in a host cell, wherein the nucleic acid sequence encoding the human CNGA3 sequence comprises SEQ ID NO: 9 or SEQ ID NO: 11.

2. The method of claim 1 , wherein nucleic acid sequence encoding human CNGA3 sequence comprises SEQ ID NO: 9 or SEQ ID NO: 11.

3. The method of claim 1 , wherein the expression control sequences comprise an ocular cell-specific promoter.

4. The method of claim 1 , wherein the AAV vector is administered in combination with another therapy.

5. The method of claim 1 , wherein the AAV vector is administered at a dosage of about 10 9 to about 10 13 vector genomes (VG).

6. The method of claim 1 , wherein the AAV vector is administered in a volume of about 100 μL to about 500 μL.

7. The method of claim 1 , wherein the AAV vector is administered more than once.

8. The method of claim 1 , wherein the expression control sequences comprise a chicken β-actin (CBA) promoter with cytomegalovirus (CMV) enhancer elements.

9. The method of claim 1 , wherein the expression control sequences comprise a rhodopsin kinase promoter.

10. The method of claim 1 , wherein the AAV capsid is an AAV2 capsid, an AAV5 capsid, an AAV8 capsid, an AAV9 capsid, an AAV8 bp capsid, an AAV7m8 capsid, or a variant thereof.

Assignees

• Univ Pennsylvania

Inventors

• Bennett Jean
• Bennicelli Jeannette
• Sun Junwei

Classifications

• C12N2840/007

  cell or tissue specific · CPC title

• C12N2840/002

  controllable or inducible · CPC title

• C12N2800/22

  Vectors comprising a coding region that has been codon optimised for expression in a respective host · CPC title

• C12N2800/107

  for mammalian · CPC title

• C12N2799/04

  in vivo · CPC title