FDA Approves First Gene Therapy for Young Children with Sickle Cell Disease

Andelyn Biosciences announced it is now manufacturing FAYUVI, Ultragenyx Pharmaceutical Inc.'s FDA-approved gene therapy for Sanfilippo syndrome type A, also known as mucopolysaccharidosis type IIIA, for commercial supply at its Columbus, Ohio facility. The move follows the U.S. Food and Drug Administration's approval of FAYUVI, making it the first FDA-approved gene therapy manufactured using the Andelyn AAV Curator Platform process. Sanfilippo syndrome type A is a rare, fatal lysosomal storage disease that primarily affects the central nervous system and is marked by rapid neurodegeneration beginning in early childhood; it is estimated to affect 3,000 to 5,000 patients worldwide, with a median life expectancy of 15 years. Andelyn Chief Executive Officer Wade Macedone said the company is proud to manufacture an FDA-approved gene therapy for commercial use using an AAV Curator Platform process, calling the milestone a reflection of the purpose behind Andelyn's founding. Andelyn, a full-service FDA-inspected cell and gene therapy commercial CDMO with more than 20 years of experience, has produced clinical and commercial material for more than 500 cGMP batches and 85 global clinical trials.

The U.S. Food and Drug Administration has approved FAYUVI, also known as UX111, a gene therapy developed by Ultragenyx Pharmaceutical Inc. for the treatment of patients with Sanfilippo syndrome type A, or MPS IIIA, a rare and progressive neurodegenerative lysosomal storage disorder. Abeona Therapeutics Inc. congratulated Ultragenyx on the approval, noting that the therapy originated from pioneering research by Drs. Haiyan Fu and Douglas McCarty at The Ohio State University and Nationwide Children's Hospital and was advanced through clinical development by Abeona as ABO-102 before Abeona out-licensed global development and commercialization rights to Ultragenyx in May 2022. Abeona Chief Executive Officer Vish Seshadri called the approval an extraordinary milestone for patients and families affected by a devastating disease that has lacked effective therapeutic options. The approval also represents a meaningful value-creation event for Abeona, which under its licensing agreement with Ultragenyx is eligible to receive certain commercial milestone payments and royalties tied to future product sales. FAYUVI is designed to deliver a functional copy of the SGSH gene to the central nervous system via an AAV9 viral vector, addressing the root genetic cause of MPS IIIA.

Eli Lilly has entered an exclusive research collaboration with QurCan Therapeutics to develop genetic medicines for nervous system diseases. The agreement centers on QurCan's polymer lipid nanoparticle delivery platform for central and peripheral nervous system targets, with Eli Lilly responsible for later development and commercialization. Lilly is also making a strategic investment in QurCan Therapeutics, tying capital directly to progress in genetic medicine delivery technology. The deal pushes Lilly further toward expansion in neurodegenerative and other specialty drugs alongside its well known GLP 1 portfolio, though pricing pressure and payer pushback still hang over Mounjaro, Zepbound and Foundayo. Investors will want to see a first concrete output such as a nominated development candidate or an announced IND timeline for at least one nervous system genetic therapy.