There are no approved pharmacological therapies for congenital hearing loss and this situation is unlikely to change any time soon. Still, a glimmer of hope has been provided with signs of hearing improvement in a single patient with profound genetic deafness after treatment with Regeneron Pharmaceuticals, Inc.’s gene therapy, DB-OTO.
Regeneron Strikes A CHORD With Deafness Gene Therapy
A very early success with a gene therapy for a rare form of deafness puts Regeneron ahead of Lilly and Sensorion.

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The promise of innovative therapies seems to have been constrained not by efficacy or safety concerns, but because the high price of treatments is incongruous with the reimbursement of short-course therapies.
The founder and CEO of Ultragenyx talked with Scrip as the company approaches a pivotal data readout and awaits US FDA action on its first gene therapy.
Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.
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Miltenyi’s leadership calls for a streamlined regulatory framework as India gears to scale in cell and gene therapy. Executives from the German group also talked to Scrip about helping advance local capabilities, delivering point-of-care CAR- T cell therapy to hospitals and pricing dynamics.
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The gene therapy for Duchenne muscular dystrophy passed the blockbuster sales threshold in 2024. Analysts said the drug’s overall risk/benefit profile still appears solid.