Gene therapy targeting OTOF mutations safely restores hearing in children and adults, with improvements from complete deafness to moderate loss (50 dB HL) within one month, as shown in a July 2025 UCI trial with 10 participants aged 1.5-23.91
Regeneron plans FDA submission by end of 2025 for DB-OTO after Phase I/II CHORD trial where 11/12 children achieved clinically meaningful hearing improvements, with some reaching normal levels and no longer needing cochlear implants46
January 2026 study confirms gene therapy restores hearing in adolescent and adult OTOF-related deafness patients, with optimal results in children aged 5-87
UF Health's 2025 gene-editing EV platform preserves hearing in mice and is set for human trials in ~3 years, adaptable to various mutations2
Single-injection therapies via AAV delivery to the inner ear reverse deafness rapidly, improving sound perception from 106 dB to 52 dB on average after six months8
Sources:
1. https://medschool.uci.edu/news/advancing-gene-therapy-address-deafness
2. https://ufhealth.org/news/2025/gene-editing-platform-may-help-treat-hearing-loss
4. https://www.biospace.com/drug-development/regeneron-readies-fda-run-for-hearing-loss-gene-therapy-that-could-transform-patients-lives
6. https://www.uc.edu/news/articles/2025/12/the-gene-therapy-poised-to-rewrite-childhood-deafness.html
7. https://geneticliteracyproject.org/2026/01/27/gene-therapy-can-restore-hearing-in-some-people-born-with-congenital-deafness/
8. https://www.sciencedaily.com/releases/2025/07/250702214148.htm