Denali Therapeutics Presents Enzyme TransportVehicle™ Progress Across Three Clinical Programs for Treatment of Lysosomal Storage Disorders

Data presented at 2026 WORLDSymposium™ highlight potential of ETV platform to enable delivery of enzyme replacement therapies to the whole body, including the brain

Feb. 5, 2026 at 10:23pm

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Denali Therapeutics presented data from programs in Hunter syndrome (MPS II), Sanfilippo syndrome type A (MPS IIIA) and Pompe disease that highlight the potential of its Enzyme TransportVehicle™ (ETV) to enable the delivery of enzyme replacement therapies (ERT) to the whole body, including the brain. The data showed progress across these three clinical programs, including reinforcing the potential for tividenofusp alfa (DNL310) to address the full disease spectrum in Hunter syndrome, preliminary Phase 1/2 study data for DNL126 (ETV:SGSH) in Sanfilippo syndrome type A, and the design of the ongoing DNL952 (ETV:GAA) Phase 1 clinical study in Pompe disease.

Why it matters

The data presented highlight the potential of Denali's Enzyme TransportVehicle platform to deliver enzyme replacement therapies to the brain and body, addressing unmet needs in rare lysosomal storage disorders like Hunter syndrome, Sanfilippo syndrome type A, and Pompe disease. This could lead to new treatment options for patients living with these debilitating diseases.

The details

The analysis from the Phase 1/2 study of tividenofusp alfa (DNL310) for Hunter syndrome showed rapid and substantial reduction in key biomarkers, as well as stabilization or improvement in clinical endpoints. The preliminary Phase 1/2 data for DNL126 (ETV:SGSH) in Sanfilippo syndrome type A demonstrated significant reductions in cerebrospinal fluid heparan sulfate and other biomarkers. Denali also presented the Phase 1 clinical study design for DNL952 (ETV:GAA) in Pompe disease, as well as preclinical data showing improved glycogen reduction compared to a second-generation enzyme replacement therapy.

  • The clinical data cut-off for the tividenofusp alfa (DNL310) Phase 1/2 study was March 28, 2025.
  • The clinical data cut-off for the DNL126 (ETV:SGSH) Phase 1/2 study was June 4, 2025.
  • Denali expects a Biologics License Application (BLA) submission and potential approval for DNL126 for MPS IIIA in 2027.

The players

Denali Therapeutics Inc.

A biopharmaceutical company developing therapies for neurodegenerative and lysosomal storage disorders.

Peter Chin, M.D.

Acting Chief Medical Officer and Head of Development of Denali Therapeutics.

Elizabeth Jalazo, M.D.

University of North Carolina at Chapel Hill and an investigator in the DNL126 (ETV:SGSH) Phase 1/2 study.

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What they’re saying

“The data presented at this year's WORLD Symposium reflect the strong momentum of our Enzyme TransportVehicle franchise as we continue to prepare for the potential commercial launch of tividenofusp alfa for Hunter syndrome and make meaningful progress across lysosomal storage disorders.”

— Peter Chin, M.D., Acting Chief Medical Officer and Head of Development of Denali Therapeutics

“These promising data show for the first time that treatment with the brain-penetrant enzyme replacement therapy DNL126 substantially reduced biomarkers of substrate accumulation in cerebrospinal fluid and peripheral tissues.”

— Elizabeth Jalazo, M.D., University of North Carolina at Chapel Hill and an investigator in the Phase 1/2 study

What’s next

Denali expects a Biologics License Application (BLA) submission and potential approval for DNL126 for MPS IIIA in 2027. Planning for a global Phase 3 confirmatory study is ongoing.

The takeaway

Denali's Enzyme TransportVehicle platform has shown promising results in delivering enzyme replacement therapies to the brain and body, potentially addressing unmet needs in rare lysosomal storage disorders like Hunter syndrome, Sanfilippo syndrome type A, and Pompe disease. This progress highlights the potential of Denali's technology to bring new treatment options to patients living with these debilitating diseases.