Denali macromolecule delivery technology that breaks through the blood-brain barrier is expected to advance to phase 2/3 clinical trials

Denali Therapeutics today announced updated long-term data from an ongoing Phase 1/2 clinical trial of its investigational enzyme replacement therapy DNL310

MPS II is a rare neurodegenerative lysosomal storage disorder caused by mutations in the gene encoding the IDS protease

The blood-brain barrier is critical in maintaining the brain microenvironment and protecting it from harmful substances and pathogens in the blood circulation

▲Denali's transfer carrier platform (Image source: Denali's official website)

All patients in the trial had neuronal disease, except for one patient in cohort B who had non-neuronal disease MPS II

Long-term data from the trial demonstrated durability of CSF biomarker responses up to one year of dosing

References:

[1] Denali Therapeutics Announces Continued Progress in DNL310 (ETV:IDS) Program for MPS II (Hunter Syndrome) Supporting Planned Initiation of Phase 2/3 Clinical Trial.

(Original abridged)