News Related to AAV9 gene therapy

Neurogene's NGN-401 Receives FDA Breakthrough Designation for Rett Syndrome

Mar 05, 2026

The FDA granted Breakthrough Therapy designation to NGN-401, Neurogene's gene therapy for Rett syndrome, based on interim data showing durable functional improvements. The company expects to complete pivotal trial dosing in Q2 2026.

Ultragenyx Reports Positive Long-Term Data for UX111 Gene Therapy in Sanfilippo Syndrome Type A

Feb 14, 2026

Ultragenyx Pharmaceutical announced positive long-term clinical data for UX111 gene therapy for Sanfilippo syndrome Type A, with up to 8.5 years of follow-up showing sustained biomarker reductions and functional improvements. The company resubmitted its BLA to the FDA in January 2026.

Ultragenyx Receives FDA Incomplete Response Letter for UX111 Gene Therapy, Announces Restructuring

Feb 14, 2026

Ultragenyx Pharmaceutical received an Incomplete Response Letter from the FDA for its UX111 gene therapy resubmission for Sanfilippo syndrome type A, requesting additional documentation. The company announced a 10% workforce reduction and expects profitability by 2027.

Ultragenyx Reports Q4 2025 Results, Resubmits UX111 Gene Therapy Application to FDA

Feb 13, 2026

Ultragenyx reported Q4 2025 revenues of $207 million, up 25% year-over-year, while announcing a 10% workforce reduction and resubmission of its UX111 gene therapy application following positive long-term clinical data.