News Related to Cerebrospinal Fluid Heparan Sulfate

Spruce Biosciences Receives Positive FDA Feedback on TA-ERT for Sanfilippo Syndrome Type B

Feb 18, 2026

Spruce Biosciences completed two Type B meetings with the FDA regarding tralesinidase alfa enzyme replacement therapy for Sanfilippo syndrome type B. The company now anticipates BLA submission in Q4 2026 to accommodate drug product process performance qualification requirements.

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 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.

FDA Places Clinical Holds on Regenxbio Gene Therapies for Rare Metabolic Disorders

Jan 29, 2026

The FDA placed clinical holds on Regenxbio's RGX-111 and RGX-121 gene therapies following a tumor case in an MPS I patient. The company's stock fell nearly 18% on the news.