EHT101

EHT101 is an investigational adeno-associated virus (AAV) vector-based gene therapy developed by Euhearing Therapeutics for the treatment of congenital monogenic hearing loss. It specifically targets mutations in the *OTOF* gene, which encodes the otoferlin protein. Otoferlin is essential for the calcium-triggered release of neurotransmitters at the synapse between inner hair cells and auditory nerve fibers; its absence leads to profound sensorineural hearing loss or auditory neuropathy. EHT101 is designed to deliver a functional copy of the *OTOF* gene directly to the inner ear hair cells via intracochlear injection, aiming to restore otoferlin expression and recover auditory function. The candidate is currently being evaluated in investigator-initiated trials (IIT) in China.

Other names

OTOF gene therapy