AAV-ATOH1

**AAV-ATOH1** is an investigational inner-ear **gene therapy** that uses an adeno-associated virus vector to deliver the **ATOH1** transcription factor with the goal of regenerating vestibular hair cells from supporting cells in the vestibule. The program was developed by **Decibel Therapeutics** and became part of **Regeneron** following Regeneron's acquisition of Decibel. Preclinical materials describe it as a **regenerative gene therapy** intended primarily for **bilateral vestibulopathy** and related balance disorders by restoring lost vestibular hair cells rather than replacing a missing monogenic structural protein. Mechanistically, forced but ideally temporally controlled expression of ATOH1 is intended to reprogram inner-ear supporting cells toward a hair-cell fate and promote maturation of regenerated vestibular hair cells.

Other names

AAV-Atoh1AAV-Atoh-1AAV-Atoh 1