Drug intelligence / Profile preview

AAV9-Pak2

Development stage
Preclinical
Lead developer
The University of Manchester
Modality
Gene Therapies
Administration
Intravenous, Parenteral
01

Overview

AAV9-Pak2 (specifically the AAV9-Pak2-T402E construct) is a preclinical gene therapy agent designed for cardiac-specific overexpression of a constitutively active form of p21-activated kinase 2 (Pak2). It utilizes an adeno-associated virus serotype 9 (AAV9) vector and a cardiac troponin T (TnT) promoter to ensure expression within cardiomyocytes. The therapy aims to mitigate maladaptive endoplasmic reticulum (ER) stress and oxidative stress by modulating the IRE1-XBP1s signaling pathway and Nrf2-mediated responses. In mouse models, AAV9-Pak2 has demonstrated cardioprotective effects against cardiac hypertrophy, fibrosis, and diastolic dysfunction, particularly in the context of heart failure with preserved ejection fraction (HFpEF). It is currently an academic research tool and has no commercial sponsor.

Other names
AAV9-Pak2-T402EAAV-9-Pak2-T402EAAV 9-Pak2-T402E
02

Targets

PAK2

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