LIN-2003

LIN-2003 is a recombinant, engineered arylsulfatase A (ARSA) enzyme replacement therapy (ERT) being developed by Linno Pharmaceuticals for the treatment of metachromatic leukodystrophy (MLD). MLD is a rare and fatal lysosomal storage disorder characterized by mutations in the ARSA gene, which leads to the accumulation of sulfatides in the nervous system and subsequent progressive demyelination. LIN-2003 is uniquely designed using Linno's proprietary Nano Brain-pass (NBP) technology, which enables the enzyme to cross the blood-brain barrier (BBB) following systemic administration. This approach addresses a major limitation of traditional ERTs that cannot effectively reach the central nervous system. By restoring ARSA enzymatic activity, LIN-2003 aims to reduce toxic sulfatide levels and prevent neurological degeneration. The drug received FDA Orphan Drug Designation in December 2023 and is currently undergoing Phase 1 clinical evaluation.

Other names

recombinant arylsulfatase Aengineered ARSA enzyme