SOUTH SAN FRANCISCO, Calif. — January 29, 2026 — Leads & Copy —
Denali Therapeutics Inc. (Nasdaq: DNLI) presented clinical and preclinical data from its Enzyme TransportVehicle™ (ETV) programs at the 22nd Annual WORLDSymposium™ in San Diego, California. The presentations, which took place February 2-6, 2026, showcased the potential of the ETV to deliver enzyme replacement therapies throughout the body, including the brain.
Two oral presentations covered data from the Phase 1/2 clinical study of tividenofusp alfa (DNL310) for Hunter syndrome (mucopolysaccharidosis type II, or MPS II) and preliminary data from the Phase 1/2 study of DNL126 (ETV:SGSH) for Sanfilippo syndrome type A (MPS IIIA). In addition, three posters highlighted a case study with a sibling pair from the Phase 1/2 study of tividenofusp alfa, a community survey conducted with Project Alive on unmet needs among individuals living with MPS II and their caregivers, and a health outcomes analysis evaluating the clinical and economic burden among individuals treated for MPS II.
The FDA is conducting a Priority Review of the Biologics License Application (BLA) for tividenofusp alfa, with a decision expected by April 5, 2026. Denali also detailed the Phase 1 study design of DNL952 (ETV:GAA) for Pompe disease and supporting preclinical data in two posters.
Specifics on the WORLDSymposium presentations include:
Platform Presentations
Phase I/II Study of Intravenous Tividenofusp Alfa for Mucopolysaccharidosis Type II
Presentation #258
Date: Thursday, February 5, 2026
Session Time: 11:00 AM-noon PST
Preliminary Results From Phase I/II, First-in-Human, Open-Label Study of DNL126 in Children With Mucopolysaccharidosis Type IIIA (MPS IIIA)
Presentation #183
Date: Thursday, February 5, 2026
Session Time: 11:00 AM-noon PST
Poster Presentations
Persistent Clinical Burden and Unmet Needs in Hunter Syndrome (MPS II) in the United States: A Retrospective Cohort Study
Poster #052
Date: Tuesday, February 3, 2026
Time: 3:30-5:30 PM PST
Enhanced Correction of Skeletal Muscle and Brain Pathology in a Pompe Mouse Model Using Transferrin Receptor-Mediated Delivery of GAA
Poster #290
Date: Wednesday, February 4, 2026
Time: 3:30-5:30 PM PST
Tividenofusp Alfa Treatment in a Male Sibling Pair with Non-neuronopathic Mucopolysaccharidosis Type II (MPS II)
Poster #065
Date: Thursday, February 5, 2026
Time: 3:30-5:30 PM PST
Quality of Life (QoL), Unmet Needs, and Treatment Experience of People Living with Mucopolysaccharidosis Type II (MPS II) and Their Caregivers: A Community Survey
Poster #248
Date: Thursday, February 5, 2026
Session Time: 3:30-5:30 PM PST
A Phase 1, Multi-center, Open-label Study Design to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants with Late-Onset Pompe Disease
Poster #034
Date: Thursday, February 5, 2026
Time: 3:30-5:30 PM PST
Denali will sponsor a satellite symposium event titled “Transforming Patient Care in MPS II” on Thursday, February 5, 2026, from 6:45-7:45 AM PST. Speakers include Barbara Burton, M.D.; Gwen Gunn, Ph.D., M.S. and Paul Harmatz, M.D.
Denali Therapeutics Inc. is a biotechnology company focused on developing biotherapeutics designed to cross the blood-brain barrier using its TransportVehicle™ platform. The company is advancing its portfolio of therapeutic candidates across all stages of development to deliver medicines for neurodegenerative, lysosomal storage and other diseases.
The TransportVehicle™ (TV) platform is designed to deliver large therapeutic molecules, such as antibodies, enzymes, and oligonucleotides, throughout the body, including the brain, by crossing the blood-brain barrier (BBB) after intravenous administration. The TV platform is based on engineered Fc domains that bind to specific natural transport receptors, such as transferrin receptor and CD98 heavy chain amino acid transporter, which are expressed at the BBB and deliver the TV and its therapeutic cargo to the brain through receptor-mediated transcytosis. In animal models, antibodies and enzymes engineered with the TV platform demonstrate more than 10- to 30-fold greater brain exposure than similar antibodies and enzymes without this technology. Oligonucleotides engineered with the TV platform demonstrate more than a 1,000-fold greater brain exposure in primates than systemically delivered oligonucleotides without this technology. Five TV-enabled programs are currently in clinical development.
PDFs of the presentations will be available on the Events page in the Investor section of Denali’s corporate website once the WORLDSymposium embargo lifts.
Source: Denali Therapeutics
