Denali Therapeutics Reports Enzyme Activity in CNS from Phase 1/2 Trials for Hunter and Sanfilippo Syndromes

Denali Therapeutics has provided updates on its Enzyme Transport Vehicle (ETV) platform during the 2026 WORLDSymposium, highlighting progress across three clinical programs aimed at treating lysosomal storage disorders. The company presented data from ongoing studies targeting Hunter syndrome, Sanfilippo syndrome type A, and Pompe disease. These programs utilize Denali’s proprietary ETV technology to deliver therapeutic enzymes across the blood-brain barrier, addressing neurological symptoms associated with these rare genetic disorders.

The updates included interim results from Phase 1/2 clinical trials for Hunter syndrome and Sanfilippo syndrome type A, as well as preclinical findings for Pompe disease. For Hunter syndrome, Denali reported that its investigational therapy demonstrated enzyme activity in both peripheral tissues and the central nervous system (CNS). Similarly, early data for Sanfilippo syndrome type A showed evidence of enzyme delivery to the CNS. Preclinical studies for Pompe disease indicated promising enzyme uptake in relevant tissues. The company stated that these findings support further development of its ETV platform in addressing unmet medical needs in lysosomal storage disorders.

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Date: February 5, 2026

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GOAI