Clinical trials
Full profile accessFollow clinical development from study design and recruitment through results.
- Trial phase
- Status
- Readouts
Drug intelligence / Profile preview
AAV9-PERM1
- Development stage
- Preclinical
- Lead developer
- Virginia Tech
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-PERM1 is an adeno-associated virus serotype 9 (AAV9) gene therapy designed to overexpress PERM1 (PPARGC1 and ESRR-induced regulator in muscle 1), a striated muscle-specific regulator of mitochondrial bioenergetics. Developed by researchers at Virginia Tech and the Fralin Biomedical Research Institute (FBRI) at VTC, the therapy is intended to treat heart failure with reduced ejection fraction (HFrEF) by addressing both contractile dysfunction and mitochondrial impairment. In preclinical studies using a mouse model of pressure overload (transverse aortic constriction), AAV9-PERM1 administration preserved left ventricular ejection fraction (LVEF), blunted pathological hypertrophy, and maintained mitochondrial DNA copy number and biogenesis markers such as TFAM and PGC-1α.
- Other names
- AAV-PERM1AAV-PERM-1AAV-PERM 1
02
Targets
PERM1 (PPARGC1 and ESRR-induced regulator, muscle 1)TNC (Tenascin-C fibronectin type III domains)
Beyond the preview
Go deeper on AAV9-PERM1.
Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.
Indications & development
Full profile accessExplore development by indication, patient population, and geography.
- Indications
- Development status
- Countries
Licensing & deals
Full profile accessTrace asset ownership, licensing agreements, and commercial partnerships.
- Partners
- Deal terms
- Milestones
Patents & exclusivity
Full profile accessExplore the patent landscape and regulatory exclusivity around an asset.
- Patents
- Expiration dates
- Exclusivity
Competitive landscape
Full profile accessCompare development programs by target, modality, and indication.
- Competing assets
- Targets
- Development stage
Research & analysis
Full profile accessConnect source evidence and development news to your research questions.
- Publications
- News
- Analysis
Bring the full picture into focus.
See how Gosset can support your research on AAV9-PERM1.