Key Points Ocugen has dosed the first patient in ArMaDa3, its global Phase 3 registrational trial of OCU410 for geographic atrophy caused by dry age-related…
September 01, 2026
April 23, 2026
Gene Therapy Turns Hearing Back On: Regeneron Wins First‑in‑Class FDA Approval for OTOF‑Related Deafness
Regeneron Pharmaceuticals (NASDAQ: REGN ) has won FDA approval for DB‑OTO, a first-in-class gene therapy designed to treat a rare congenital hearing loss caused by…
February 09, 2026
Ultragenyx’s Gene Therapy UX111 Shows Durable Efficacy in Sanfilippo Syndrome Studies
Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) reported that its investigational gene therapy UX111 demonstrated sustained cognitive improvements and significant biomarker gains in long-term follow-up from two…