FDA approves CASGEVY for children as young as two with sickle cell disease and beta thalassemia

Ultragenyx Pharmaceutical received full U.S. FDA approval in September 2026 for FAYUVI, also known as UX111, the first-ever treatment for pediatric patients with Sanfilippo syndrome Type A, a previously untreated ultra-rare and fatal neurodegenerative disease. The approval came with a Priority Review Voucher and expands Ultragenyx's commercial gene therapy portfolio and U.S. manufacturing footprint. The FAYUVI clearance follows the accelerated FDA approval of GENGLYCOS, also known as DTX401, for GSDIa in August 2026, giving the company two new gene therapies approved within weeks, each generating its own Priority Review Voucher. Ultragenyx's investment narrative projects $1.2 billion in revenue and $43.8 million in earnings by 2029, with a $27.00 fair value estimate implying 86% upside to the current price. The company's biggest immediate risk remains high cash burn and potential dilution if launches underperform or costs do not come down, with adoption, reimbursement and scaling of the new therapies shaping whether it can move closer to its 2027 profitability goal.

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.

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.