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AAV.104
- Development stage
- Preclinical
- Lead developer
- Regeneron Pharmaceuticals
- Modality
- Gene Therapies
- Administration
- Cochleostomy, Round Window Membrane Injection, Semicircular Canal Injection
01
Overview
AAV.104 is a preclinical, adeno-associated virus (AAV) gene therapy being developed to restore hearing in individuals with congenital hearing loss caused by biallelic mutations in the stereocilin (STRC) gene. It delivers a functional STRC transgene to cochlear hair cells to restore stereocilin expression and thereby reconstitute normal hair bundle function and mechanoelectrical transduction in the inner ear. The program was originally developed by Decibel Therapeutics and is now part of Regeneron’s hearing loss gene therapy portfolio, alongside other AAV-based candidates targeting monogenic forms of deafness.[1][2][7][9]
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