NI-002

NI-002 is a preclinical-stage recombinant glycoprotein and enzyme replacement therapy (ERT) being developed by NeuImmune. It is designed to address the central nervous system (CNS) manifestations of various lysosomal storage disorders (LSDs) beyond Niemann-Pick disease. The therapeutic approach utilizes a proprietary platform that targets Intercellular Adhesion Molecule 1 (ICAM-1) to facilitate the transport of lysosomal enzymes across the blood-brain barrier (BBB). These enzymes are produced using NeuImmune's specialized geCHO (genetically engineered Chinese Hamster Ovary) cell line technology, which is optimized for the production of therapeutic glycoproteins. NI-002 represents an expansion of the company's lead ICAM-1-targeted ERT platform into additional LSD indications where CNS involvement remains a significant unmet medical need.

Other names

Additional LSDs ProgramAdditional LSDs Program (ICAM-1/ERTs)