NP-C Therapeutics

NPC Therapeutics is a biotechnology company dedicated to developing curative gene therapies for Niemann-Pick Type C (NPC) disease, a rare and fatal neurodegenerative lysosomal storage disorder. The company was founded by a team of world-renowned scientists, including Dr. Peter Pentchev, who led the NIH team that discovered the NPC1 gene. Their pipeline is centered on AAV-based gene replacement therapies, with the lead program NPC-001 targeting the NPC1 gene and a secondary program NPC-002 targeting the NPC2 gene. NPC Therapeutics holds exclusive licenses for these technologies from the National Institutes of Health (NIH). The company aims to address the underlying genetic cause of the disease by restoring functional protein expression in the brain and other affected organs.