JUNCTOPHILIN-2 (JPH2) GENE THERAPY USING AAV VECTOR

Title:

JUNCTOPHILIN-2 (JPH2) GENE THERAPY USING AAV VECTOR

Document Type and Number:

WIPO Patent Application WO/2023/108029

Kind Code:

A3

Abstract:

Provided herein is a gene therapy for JPH2 (Junctophilin-2), e.g., using an adeno-associated virus (AAV) vector. The promoter of the vector may be a MHCK7 promoter or a cardiac troponin T (HTNNT2) promoter. The capsid may be an AAV9 or AAVrh74 capsid or a functional variant thereof. Other promoters or capsids may be used. Further provided are methods of treatment, such as by intravenous, intracoronary, intracarotid or intracardiac administration of the rAAV vector, and other compositions and methods.

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Inventors:

HERZOG CHRISTOPHER (US)
SACRAMENTO CHESTER (US)
RICKS DAVID (US)
PRABHAKAR RAJ (US)

Application Number:

PCT/US2022/081122

Publication Date:

July 27, 2023

Filing Date:

December 07, 2022

Export Citation:

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Assignee:

SPACECRAFT SEVEN LLC (US)

International Classes:

C12N15/85; A61K48/00; A61P21/00; C07K14/47; A61K38/17; C12N15/861

Foreign References:

US20190307899A1	2019-10-10
US20040043382A1	2004-03-04

Other References:

DATABASE NUCLEOTIDE ANONYMOUS : "Synthetic construct Homo sapiens clone IMAGE:9095596; MGC:199456 junctophilin 2 (JPH2) gene, encodes complete protein", XP093081559, retrieved from NCBI

Attorney, Agent or Firm:

LAHERTY, Carol et al. (US)

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