NI-001

NI-001 is a preclinical-stage recombinant fusion protein being developed by NeuImmune as an enzyme replacement therapy (ERT) for the treatment of Niemann-Pick disease, specifically focusing on its central nervous system (CNS) manifestations. The drug candidate consists of a recombinant acid sphingomyelinase (ASM) enzyme, produced in glycoengineered Chinese Hamster Ovary (geCHO) cells, fused to a targeting moiety directed against intercellular adhesion molecule-1 (ICAM-1). This design is intended to facilitate the transport of the therapeutic enzyme across the blood-brain barrier (BBB) via ICAM-1-mediated receptor-mediated transcytosis. By delivering the functional ASM enzyme directly to the CNS, NI-001 aims to address the underlying sphingomyelinase deficiency and subsequent lysosomal storage accumulation that standard ERTs cannot effectively reach.

Other names

Niemann Pick Disease ProgramICAM-1/ASM programICAM-1-targeted recombinant acid sphingomyelinaseICAM1-targeted recombinant acid sphingomyelinaseICAM 1-targeted recombinant acid sphingomyelinase