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AAV9-DWORF
- Development stage
- Preclinical
- Lead developer
- Tenaya Therapeutics
- Modality
- Gene Therapies
- Administration
- Intravenous
01
Overview
AAV9-DWORF is an adeno-associated virus serotype 9 (AAV9) mediated gene therapy designed to deliver the Dwarf Open Reading Frame (DWORF) gene. Developed by Tenaya Therapeutics, the therapy targets the sarco/endoplasmic reticulum calcium-ATPase (SERCA) pump, which is often dysfunctional in failing hearts. DWORF is a muscle-specific micropeptide that acts as an endogenous enhancer of SERCA activity by displacing the inhibitory peptide phospholamban (PLN). By restoring or enhancing SERCA function, AAV9-DWORF aims to improve calcium handling and cardiac contractility. It is currently in preclinical development for the treatment of cardiovascular diseases, including heart failure and various cardiomyopathies, as well as Duchenne muscular dystrophy (DMD) associated cardiomyopathy.
- Other names
- AAV9-DWORF gene therapyAAV-9-DWORF gene therapyAAV 9-DWORF gene therapyadeno-associated virus serotype 9-DWORF
02
Targets
PLN (Phospholamban)ATP2A2 (Sarco/endoplasmic reticulum calcium ATPase isoform 2a)
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