RHI100

RHI100 is an investigational gene therapy designed for the treatment of DFNB8, a form of post-lingual progressive genetic hearing loss caused by mutations in the TMPRSS3 gene. Originally developed by Rescue Hearing Inc. and subsequently licensed to Myrtelle Inc., the therapy utilizes a recombinant adeno-associated virus (AAV2) vector to deliver a functional copy of the human TMPRSS3 gene directly to the inner ear via local administration. TMPRSS3 encodes a transmembrane serine protease that is essential for the survival of cochlear hair cells and spiral ganglion neurons. By restoring functional TMPRSS3 expression, RHI100 aims to rescue auditory function and promote cellular survival in the cochlea. The therapy is being developed as both a standalone treatment and a combination therapy for patients with cochlear implants who experience declining outcomes. The program has completed proof-of-concept studies and is currently in IND-enabling development.

Other names

DFNB8 gene therapyDFNB-8 gene therapyDFNB 8 gene therapyTMPRSS3 gene therapyTMPRSS-3 gene therapyTMPRSS 3 gene therapy