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Denali Therapeutics Announces Data Presentations on Enzyme TransportVehicle™ Programs for Hunter Syndrome, Sanfilippo Syndrome Type A and Pompe Disease at Upcoming 2026 WORLDSymposium™
Globenewswire· 2026-01-29 21:00
Core Insights - Denali Therapeutics Inc. is set to present clinical and preclinical data from its Enzyme Transport Vehicle™ (ETV) programs at the 22nd Annual WORLDSymposium™ from February 2-6, 2026, showcasing the potential of ETV for delivering enzyme replacement therapies throughout the body, including the brain [1] Group 1: Clinical Data Presentations - Two oral presentations will cover follow-up data from the Phase 1/2 clinical study of tividenofusp alfa (DNL310) for Hunter syndrome (MPS II) and preliminary data from the ongoing Phase 1/2 study of DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA) [2][5] - The U.S. FDA is conducting a Priority Review of the Biologics License Application (BLA) for tividenofusp alfa, with a decision anticipated by April 5, 2026 [2] Group 2: Presentation Details - The platform presentations include a study on intravenous tividenofusp alfa for MPS II and preliminary results from the DNL126 study in children with MPS IIIA, both scheduled for February 5, 2026 [3] - Poster presentations will cover various topics, including a retrospective cohort study on unmet needs in Hunter syndrome, enhanced correction of skeletal muscle and brain pathology in a Pompe mouse model, and a case study involving a male sibling pair with MPS II [4][6] Group 3: Satellite Symposium - Denali will sponsor a satellite symposium titled "Transforming Patient Care in MPS II" on February 5, 2026, featuring expert speakers [7] Group 4: TransportVehicle™ Platform - Denali's TransportVehicle™ platform is designed to cross the blood-brain barrier, enabling the delivery of large therapeutic molecules, including antibodies and enzymes, to the brain, demonstrating significantly improved brain exposure compared to traditional methods [8] - The platform has been clinically validated, with five TV-enabled programs currently in clinical development [8] Group 5: Company Overview - Denali Therapeutics is a biotechnology company focused on developing biotherapeutics that can cross the blood-brain barrier, aiming to transform the lives of individuals with neurodegenerative and lysosomal storage diseases [9]