Denali Therapeutics DNL310 gets FDA orphan drug and rare pediatric disease designations

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Denali Therapeutics (NASDAQ:DNLI) received FDA orphan drug designation and rare pediatric disease designation for DNL310 for the treatment of Hunter syndrome.

Hunter syndrome is a genetic lysosomal storage disease caused by a mutation in the gene coding for iduronate 2-sulfatase (IDS), and characterized by progressive cognitive impairment. Standard enzyme replacement therapy is limited by its ability to cross the blood-brain-barrier (BBB).

DNL310, delivered intravenously, uses Denali’s enzyme transport vehicle (TV) technology to transport IDS across the BBB.

“We believe that our BBB TV technology provides a non-invasive solution to deliver higher concentrations and broader distribution of protein-based therapeutics in the brain and will therefore improve the chance of success to achieve efficacy for patients with neurodegenerative diseases, including many lysosomal storage diseases," Ryan Watts, Denali’s CEO, said in a statement.

Denali plans to conduct a Phase 1/2 study of DNL310 in Hunter syndrome in 2020.

Jennifer Poland