Gene Therapy Pipeline

Pioneering Gene Therapy for Hearing Loss

Developing Gene Therapies for the Hearing Loss Population – Rescue Hearing’s Gene Therapy Pipeline focuses on addressing the root cause of genetic hearing loss. Our team of physicians, scientists, researchers, and business advisors are utilizing advanced medical technologies and drug development expertise to provide therapeutic options for those with hearing loss.

A Game-Changer in Hearing Care

Gene therapy is rapidly emerging as a groundbreaking solution for hearing loss, becoming both medically and commercially viable. With advancements in gene-editing tools like CRISPR/Cas9, developing gene therapy products is now more realistic, efficient, and affordable than ever before.

Genetic Testing & Hearing Loss

Up to 50% of hearing loss is linked to genetic factors. Medical providers are increasingly using genetic testing as a cost-effective tool to identify and address hearing-related mutations.

Regulatory Support & Innovation

Regulatory agencies like the FDA and EMA are streamlining the approval process for gene therapy technologies, accelerating their path to clinical application.

Investment Surge in Gene Therapy

Private institutional investment in gene and cell therapy companies has skyrocketed, growing from $362M in 2020 to $68B in 2021—nearly 33% of all life science investments.

With cutting-edge innovations and growing global support, gene therapy is shaping the future of hearing restoration and personalized medicine.

RHI Product Pipeline

Current as of: 4/6/2025

RHI Product Pipeline

Rescue Hearing Inc. is advancing a cutting-edge pipeline of gene therapy solutions targeting various forms of hearing loss. From progressive hearing impairment to balance disorders and neurodegenerative conditions, each product is designed to revolutionize hearing care through innovation and scientific breakthroughs.

* Common in Cochlear Implant Population

Our Five Products

Our pipeline features five breakthrough candidates, led by DFNB8 (most common genetic deafness in cochlear implant patients) and USH1B (addressing Usher Syndrome hearing and balance disorders). These flagship programs, alongside three additional candidates, position us as leaders in hearing restoration with substantial market potential.

DFNB8: Post Lingual Deafness

Most frequent genetic cause of deafness in the Cochlear Implant Population.

USH1B: Usher Sydnrome and Vestibular Disorders

Gene therapy will focus on rescue of vestibular function in Usher Syndrome patients.

DFNB16: 2nd Leading Cause of Genetic Deafness

Mutations of the STRC gene are the second leading cause of genetic deafness.

DFNB77: Nonsyndromic Hearing Loss

A nonsyndromic form of genetic hearing loss seen in the cochlear implant population.

Neurotrophic Disorders

Gene therapy will focus on treatment of inner ear cells.