A Breakthrough in Hearing Loss: What Gene Therapy Means for Families
- 2 hours ago
- 3 min read
Laurie Hanin, PhD, CCC-A, CHC Executive Director
The article “New Gene Therapy Enables Children With a Rare Form of Deafness to Hear” written by Gina Kolata and published in the New York Times on April 23, 2026 discusses the intriguing news that the Food and Drug Administration recently approved a gene therapy that can significantly reduce the hearing loss caused by Otoferlin, a rare, inherited form of deafness. This gene is responsible for approximately 2-8% of congenital hearing loss.
Gene Therapy is Changing What's Possible
I recently spoke with CHC's Medical Director, Dr. Thomas Roland, who shared insight on this development.
Otoferlin-related deafness is caused by a problem in a specific gene, and it can severely affect a child’s hearing. But with this new treatment, there’s now a chance to fix the genetic issue and help these children hear more clearly. This therapy was discussed by Dr. Roland in a 2024 Q&A published on the CHC blog. Dr. Roland wrote about a research study showing promising results with gene therapy, even before this breakthrough treatment received FDA approval. At the time, the company Akous was working on the treatment. Now, another company, Regeneron, has made it a reality.
New Therapy On the Horizon
Another interesting mention in the article is that a similar gene therapy trial for a different kind of genetic hearing loss, targeting the GJB2 gene, may soon begin in the United States. GJB2-related deafness is caused by a mutation in the GJB2 gene, and results in a deafness commonly referred to as Connexin26 related hearing loss. Around 30-40% of children with profound prelingual hearing loss have this genetic mutation, which makes it one of the most common causes of autosomal recessive non syndromic hereditary (inherited) deafness.
What this Means for Families
For parents who aren’t sure what’s causing their child’s hearing loss, we encourage taking action. It’s a good idea to meet with a geneticist or genetics counselor to get a clearer picture of whether the hearing loss could be due to a genetic mutation. If you haven’t spoken with a genetics expert before, your audiologist and otolaryngologist can help connect you with a genetics team that understands testing for hearing loss. Understanding the genetic cause of hearing loss could open the door to potential treatments, including gene therapy, that could improve hearing in the future.
Contact Us
If you have questions about gene therapy or any aspect of your child’s hearing, we’re here to support you. Our experts can provide guidance and connect you with trusted resources and specialists.
About the Author
Laurie Hanin, PhD, CCC-A, is the Executive Director of the Center for Hearing and Communication (CHC) and has been with CHC since 1992. She has been an audiologist for over 35 years. She received her Masters degree in Audiology in 1979 from Queens College of the City University of New York, and her Ph.D. degree in Speech and Hearing Sciences from the Graduate Center of the City University of New York in 1988. Dr. Hanin has been a researcher in the areas of hearing loss and cochlear implantation and focused much of her clinical audiological work in pediatrics. She recently received the New York State Distinguished Clinician Award, is well published, and has made frequent presentations at major conferences and conventions in addition to numerous television appearances as a leading expert in audiology, particularly on The Today Show.
