Gene Therapy for Hearing Loss
There is currently no FDA-approved treatment and no registered interventional gene-therapy trial specifically for TMPRSS3-related hearing loss.
However, the field moved forward significantly with the first FDA-approved genetic hearing-loss gene therapy for OTOF variants in April 2026.
Hearing Loss Gene Therapy News (TMPRSS3 DFNB8/10)
IU study finds possible treatment route for common type of genetic hearing loss (9/10/25)
The Natural History of TMPRSS3-related Hearing Loss (5/1/24)
Rescue of Auditory Function by a Single Administration of AAV- TMPRSS3 Gene Therapy in Aged Mice of Human Recessive Deafness DFNB8 (2/26/23)
Hearing Loss Gene Therapy News (Not TMPRSS3)
A clinical study testing an investigational therapy targeting GJB2-hearing loss - Sonix Study (6/2026)
Gene Therapy vs Cochlear Implantation in Restoring Hearing Function and Speech Perception for Individuals With Congenital Deafness (Otof Gene Therapy) - (9/1/25)
Companies developing treatments for genetic hearing loss and hearing regeneration
In the future, gene therapy will be an option for people. We are tracking the latest companies, organizations, and news about gene therapy. Gene therapy could potentially restore, improve, and preserve hearing in individuals. There are currently no TMPRSS3 clinical trials for gene therapy; however, this may change soon.
Acousia Therapeutics - Committed to identifying and developing novel compounds (small molecules) to preserve and enhance natural hearing for patients suffering from sensorineural hearing loss.
Eli Lilly / Akouos - Advancing Precision Genetic Medicines with the Potential to Restore, Improve, and Preserve Hearing. Testing (and possibly free testing) Resources. Resonate
Myrtelle - A gene therapy company whose mission is to transform the treatment of neurological diseases. Involved in DFNB8 research.
Otovia Therapeutics - A biotech company in China focusing on innovative drugs for diseases related to inner ear hearing loss.
Regeneron - A biotechnology company that invents, develops, and commercializes life-transforming medicines for people with serious diseases. Includes former Decibel Therapeutics programs.
Rescue Hearing - The RHI mission is to “Eliminate Hearing Loss as a Chronic Condition”. Involved in developing TMPRSS3 gene therapies. Its website lists DFNB8, licensed to Myrtelle, plus DFNB16 and USH1B programs.
Rinri Therapeutics - Rincell-1 cell therapy aims to replace auditory neurons; it is not a therapy that corrects a particular inherited mutation.
Salubritas Therapeutics - Developing treatments for hearing loss, including genome editing, inner ear regeneration, novel delivery, and gene therapy. Associated with researcher Dr. Zheng-Yi Chen and Dr. Xue Zhong Liu.
Seamless Therapeutics - Eli Lilly signed an agreement worth up to $1.12 billion with Seamless Therapeutics, a Germany-based startup, to develop and commercialize treatments for hearing loss using the biotech's gene-editing platform. The deal will give Lilly access to its proprietary technology to design specially engineered enzymes to correct certain gene mutations linked to hearing loss - (According to Reuters)
Sensorion - A French biotech company that offers innovative therapeutic solutions to provide long-term improvements for people living with, or at risk of, inner ear disorders. It prioritizes GJB2-related hearing loss.
Shanghai Euhearing Therapeutics - Develops gene therapies for congenital, single-gene hearing loss, including EHT102 for OTOF-related hearing loss.
Shionogi - A pharmaceutical collaborator. Its hearing-regeneration work is partnered with Salubritas Therapeutics.
Skylark Bio - Relevant for inherited hearing loss like GJB2, UBE3A (Angelman Syndrome), and SLC26A4. SKY-GJB2 is in Phase I/II development, with first-patient dosing announced in August 2026. It also has an SLC26A4/pendrin program.
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Inner Ear Gene Therapy Program - This laboratory, formerly part of the NIDCD Otolaryngology Surgeon-Scientist Program (OSSP), is focused on developing gene therapy as a treatment option for hearing loss and dizziness.
Somatic Cell Genome Editing (SCGE) Platform - The Somatic Cell Genome Editing (SCGE) Consortium is an NIH Common Fund program that aims to develop safe and effective methods to perform gene editing to treat genetic diseases in somatic cells.
Mapping of Clinical Trials of Hearing Disorders - Interesting database from The Hearing Foundation out of France