Denali Therapeutics, Inc. (DNLI) announced that the FDA has selected experimental candidate DNL126 in support of clinical trials advancing the rare disease therapeutics (START) Pilot Program. The candidate is an investigational enzyme replacement therapy designed to cross the blood-brain barrier (BBB) for the potential treatment of MPS IIIA (Sanfilippo syndrome type A). MPS III, also known as Sanfilippo syndrome, is a rare, genetic lysosomal storage disease that causes neurodegeneration. The newly launched ...