ETV:IDS (DNL310) granted Fast Track Designation in the US for the treatment of Hunter Syndrome (MPS II)
The investigational enzyme replacement therapy, which crosses the blood brain barrier, is under evaluation in a Phase 1/2 study in patients with Hunter syndrome as a potential treatment for both CNS and peripheral manifestations of the disease.
Source:
Biospace Inc.
SPS commentary:
Hunter syndrome (MPS II) is a rare neurodegenerative lysosomal storage disorder caused by a mutation in the gene that encodes for the enzyme iduronate-2-sulfatase (IDS). DNL310 is a fusion protein composed of IDS fused to the company’s proprietary Enzyme Transport Vehicle (ETV), which is engineered to cross the BBB via receptor-mediated transcytosis into the brain.