Children's Hospital of Philadelphia

Stories

Gene therapy restores hearing in deaf patients

First U.S. site to administer gene therapy for genetic hearing loss

A single injection into the inner ear can now restore hearing in children born deaf. In April 2026 the FDA approved Otarmeni, the first gene therapy for inherited hearing loss, after about 80% of 24 trial participants gained measurable hearing and 42% could pick up whispers.

Updated Oct 3

FDA creates new approval pathway for gene therapies tailored to individual rare disease patients

Rule Changes

Site of the first personalized CRISPR therapy for Baby KJ

For decades, the FDA required all drugs to prove effectiveness through controlled trials with sufficient sample sizes, a standard that made sense for common diseases but created an impossible barrier for ultra-rare conditions. On February 23, 2026, the FDA issued draft guidance for a new standard—the 'plausible mechanism' framework. Developers of gene-editing and ribonucleic acid (RNA) therapies could win full approval by showing that their treatment targets the root genetic cause, engages it, and improves outcomes compared to the disease's natural course.

Updated May 29