Denali Therapeutics Announces Data Presentations on Enzyme Transport Vehicle Programs

Presentations at upcoming WORLD Symposium to showcase progress on treatments for Hunter Syndrome, Sanfilippo Syndrome Type A, and Pompe Disease

Jan. 29, 2026 at 4:31pm

Got story updates? Submit your updates here. ›

Denali Therapeutics Inc. today announced it will present clinical and preclinical data from its Enzyme TransportVehicle (ETV) programs at the 22nd Annual WORLD Symposium in San Diego, California. The presentations will highlight continued follow-up data from Phase 1/2 studies of tividenofusp alfa (DNL310) for Hunter syndrome and DNL126 (ETV:SGSH) for Sanfilippo syndrome type A, as well as the Phase 1 study design and preclinical data for DNL952 (ETV:GAA) for Pompe disease.

Why it matters

Denali's TransportVehicle platform is designed to effectively deliver large therapeutic molecules, including enzymes, throughout the body and across the blood-brain barrier. Successful development of these ETV-enabled therapies could transform treatment options for patients with rare, often devastating neurological disorders like Hunter syndrome, Sanfilippo syndrome, and Pompe disease.

The details

The presentations will include two oral presentations discussing follow-up data from the Phase 1/2 study of tividenofusp alfa for Hunter syndrome and preliminary data from the ongoing Phase 1/2 study of DNL126 for Sanfilippo syndrome type A. Additionally, five poster presentations will cover topics such as the clinical and economic burden of Hunter syndrome, preclinical data on the ETV platform's ability to enhance treatment of Pompe disease, and a case study of tividenofusp alfa treatment in a non-neuronopathic sibling pair with Hunter syndrome.

  • The FDA is conducting a Priority Review of the Biologics License Application for tividenofusp alfa, with a decision expected by April 5, 2026.
  • The WORLD Symposium will be held February 2-6, 2026 in San Diego, California.

The players

Denali Therapeutics Inc.

A biotechnology company pioneering a new class of biotherapeutics designed to cross the blood-brain barrier using its proprietary TransportVehicle platform.

TransportVehicle Platform

Denali's proprietary technology designed to effectively deliver large therapeutic molecules such as antibodies, enzymes and oligonucleotides throughout the whole body, including the brain, by crossing the blood-brain barrier after intravenous administration.

tividenofusp alfa (DNL310)

Denali's enzyme replacement therapy for Hunter syndrome (mucopolysaccharidosis type II, or MPS II) that is currently under FDA Priority Review.

DNL126 (ETV:SGSH)

Denali's enzyme replacement therapy for Sanfilippo syndrome type A (MPS IIIA) that is in a Phase 1/2 clinical study.

DNL952 (ETV:GAA)

Denali's enzyme replacement therapy for Pompe disease that is in a Phase 1 clinical study.

Got photos? Submit your photos here. ›

The takeaway

Denali's TransportVehicle platform has the potential to transform treatment options for patients with rare neurological disorders by enabling effective delivery of therapeutic enzymes across the blood-brain barrier. The upcoming data presentations at the WORLD Symposium will provide important updates on the progress of Denali's ETV-enabled programs for Hunter syndrome, Sanfilippo syndrome, and Pompe disease.