An international clinical trial co-led by Mass General Brigham and Fudan University’s Eye & ENT Hospital found that a one-time gene therapy restored hearing in about 90% of participants born with a rare genetic form of deafness, with benefits lasting up to 2.5 years. The trial, the largest and longest-followed study of its kind, treated 42 people aged 0.8 to 32 years who have mutations in the OTOF gene, delivering a working copy of the gene via a single injection into the inner ear. Most participants regained hearing within weeks and went on to develop or improve speech, with younger children and those treated in both ears showing the strongest gains. The results were published in the journal Nature.
Why it matters: OTOF mutations cause only a small share of inherited deafness, but this is the strongest evidence yet that a single gene therapy injection can produce durable hearing and speech gains, a step researchers hope will guide therapies for other genetic causes of hearing loss.
Source: Mass General Brigham →
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