Kyverna says one-year CAR-T data strengthen FDA filing for stiff-person syndrome on September 24, 2026 Autoimmune Diseases BLA Blackfoot language CAR-T Kyverna Therapeutics miv-cel RMAT Stiff-Person Syndrome United States Food and Drug Administration +
Beacon’s gene therapy advances toward FDA filing after pivotal XLRP trial success on September 21, 2026 Approved Beacon Therapeutics BLA Blackfoot language Blindness gene therapy Infrequent laru-zova United States Food and Drug Administration VISTA trial X-linked retinitis pigmentosa +
Ultragenyx Resubmits BLA for UX111 Gene Therapy Targeting Sanfilippo Syndrome Type A to FDA on February 02, 2026 Accelerated Approved BLA Blackfoot language gene therapy Mucopolysaccharidosis Type IIIA resubmission Ultragenyx United States Food and Drug Administration UX111 +