Ultragenyx Gene Therapy DTX301 Hits Key Endpoint in Phase 3 Trial for Ornithine Transcarbamylase Deficiency on March 13, 2026 AAV gene therapy Ammonia level finding DTX301 Enh3ance trial gene therapy Ornithine carbamoyltransferase deficiency Phase 3 Ultragenyx +
Ultragenyx Announces Layoff of 130 Employees Amid FDA Delays on UX111 Resubmission on February 13, 2026 layoffs Rare Diseases Ultragenyx United States Food and Drug Administration UX111 Workers +
Ultragenyx Resubmits BLA for UX111 Gene Therapy with New Long-Term Data for Sanfilippo Syndrome Type A on February 03, 2026 Accelerated Approved Blackfoot language gene therapy Mucopolysaccharidosis III resubmission Ultragenyx United States Food and Drug Administration UX111 +
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 +
Ultragenyx and Mereo BioPharma Stocks Crash After Late-Stage Setrusumab Trials Fail in Osteogenesis Imperfecta on January 02, 2026 Bone Density clinical trial failure Mereo BioPharma Osteogenesis Imperfecta setrusumab stock crash Ultragenyx +
FDA Declines Approval of Ultragenyx's Sanfilippo Gene Therapy Due to Manufacturing Issues on July 14, 2025 Approved gene therapy manufacturing issues Mucopolysaccharidosis Type IIIA Regulatory Setback Response Letter Ultragenyx United States Food and Drug Administration UX111 +