Gene therapy breakthrough seen in Leber's congenital amaurosis

One-year KYSA-8 and KYSA-6 data showed durable miv-cel benefit in SPS and gMG, supporting Kyverna's rolling BLA submission targeted for Q4 2026.

FDA approved Fayuvi (rebisufligene etisparvovec-hopf), a one-time AAV9 gene therapy, for pediatric patients with Sanfilippo syndrome type A, based on cognitive data from an open-label trial in children 2 to 5 years old.

Ocugen dosed the first patient in ArMaDa3, a global phase 3 trial of the one-time gene therapy OCU410 for geographic atrophy, following FDA RMAT designation and phase 2 data showing a 31% reduction in lesion growth versus control.

Novartis suspended several autoimmune and neurology trials of rap-cel after 3 patient deaths linked to immune effector cell-associated hemophagocytic syndrome, and BMS separately paused zola-cel studies after observing inflammatory safety events.

The FDA placed a new clinical hold on REGENXBIO's RGX-121 for Hunter syndrome after asymptomatic spine MRI findings were identified in 5 trial participants, the gene therapy's second regulatory setback this year.

The FDA extended its review of deramiocel for Duchenne muscular dystrophy to November 22, 2026, after accepting additional HOPE-3 data as a major BLA amendment.

PTC Therapeutics wins Sangamo's BLA-stage Fabry disease gene therapy ST-920 for $111 million in a bankruptcy auction, with STAAR data supporting accelerated approval.

FDA granted accelerated approval to pariglasgene brecaparvovec-opnr, a one-time liver-directed AAV8 gene therapy, based on a 31% reduction in cornstarch intake versus placebo.