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PERM1
- Development stage
- Preclinical
- Lead developer
- Virginia Tech
- Modality
- Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
- Administration
- Intravenous
01
Overview
PERM1 (PPARGC1 and ESRR-induced regulator, muscle 1) is a striated muscle-specific protein that acts as a regulator of mitochondrial bioenergetics and cardiac contractility. As a therapeutic candidate, it is developed as an adeno-associated virus (AAV)-mediated gene therapy designed to treat heart failure with reduced ejection fraction (HFrEF). The mechanism involves PERM1 acting as a signaling nexus that links metabolic regulation to sarcomeric function. Specifically, PERM1 enhances contractility by downregulating myosin-binding protein C3 (MYBPC3)—a protein that normally limits actin-myosin cross-bridge formation—and by anchoring creatine kinase B (CKB) to the sarcomere to facilitate ATP delivery for muscle contraction. Research conducted at Virginia Tech suggests that AAV-mediated overexpression of PERM1 can restore mechano-energetic coupling and improve cardiac output in failing hearts.
- Other names
- PPARGC1 and ESRR-induced regulator, muscle 1
02
Targets
PPARA (Peroxisome proliferator-activated receptor alpha)ESRRA (Estrogen-related receptor alpha)BAG6 (Large proline-rich protein BAG6)TNC (Tenascin-C fibronectin type III domains)PPARGC1A (Peroxisome proliferator–activated receptor gamma coactivator 1-alpha)
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