Granted Patent
B2
US 12,576,161 · App. 18/297,347 · Granted Mar 17, 2026
Plakophillin-2 gene therapy methods and compositions
Inventors:
Zhihong Jane Yang (Menlo Park, CA); Jaclyn Ho (San Mateo, CA); Chris Reid (San Bruno, CA); Jin Yang (Belmont, CA)
Assignee:
Tenaya Therapeutics, Inc.
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Abstract
Provided herein are methods and compositions for plakophilin-2 gene therapy for treating heart diseases such as arrhythmogenic right ventricular cardiomyopathy (ARVC) or arrhythmogenic cardiomyopathy (ACM).
Claims (17)
1 . A gene therapy vector comprising a plakophilin 2 gene operatively linked to at least one promoter, wherein the plakophilin 2 gene has a sequence at least 90% identical to SEQ ID NO: 2.
2 . The gene therapy vector of claim 1 , wherein the gene therapy vector comprises a viral vector.
3 . The gene therapy vector of claim 2 , wherein the viral vector is selected from the group consisting of an adeno-associated virus, an adenovirus, a lentivirus, a pox virus, a vaccinia virus, and a herpes virus.
4 . The gene therapy vector of claim 2 , wherein the gene therapy vector is an adeno-associated virus.
5 . The gene therapy vector of claim 4 , wherein the adeno-associated virus is selected from the group consisting of an AAV6, an AAV8, and an AAV9.
6 . The gene therapy vector of claim 5 , wherein the adeno-associated virus is an AAV9 or a derivative thereof.
7 . The gene therapy vector of claim 1 , wherein the gene therapy vector targets cells in the myocardium, the epicardium, or both.
8 . The gene therapy vector of claim 1 , wherein the promoter is a cardiac specific promoter.
9 . The gene therapy vector of claim 8 , wherein the cardiac specific promoter directs gene expression in the myocardium, the epicardium, or both.
10 . The gene therapy vector of claim 8 , wherein the cardiac specific promoter is a troponin promoter or an alpha-myosin heavy chain promoter.
11 . The gene therapy vector of claim 1 , wherein the promoter is a PKP2 promoter.
12 . The gene therapy vector of claim 1 , wherein the promoter is a constitutive promoter.
13 . The gene therapy vector of claim 12 , wherein the constitutive promoter is a beta-actin promoter.
14 . The gene therapy vector of claim 1 , further comprising a cardiac specific enhancer.
15 . The gene therapy vector of claim 1 , wherein the gene therapy vector further comprises a 3′ element.
16 . The gene therapy vector of claim 15 , wherein the 3′ element comprises a Woodchuck Hepatitis Virus Posttranscriptional Regulatory Element (WPRE), a bovine growth hormone polyadenylation (bGH polyA) sequence, or a combination thereof.
17 . A composition comprising the gene therapy vector of claim 1 and a pharmaceutically acceptable carrier or excipient comprising a buffer, a polymer, a salt, or a combination thereof.
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Jul 24, 2023
From: YANG, ZHIHONG JANE; HO, JACLYN; REID, CHRIS; YANG, JIN
To: TENAYA THERAPEUTICS, INC.
Reel/Frame 064363/0702 →
Continuity (6)
Continuation
PCTUS2021053908
· Oct 7, 2021
Provisional Application
63227801
· Jul 30, 2021
Provisional Application
63216322
· Jun 29, 2021
Provisional Application
63172053
· Apr 7, 2021
Provisional Application
63089951
· Oct 9, 2020
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