NGN-201

NGN-201 is an adeno-associated virus (AAV)-mediated gene therapy in preclinical development by Neurogene for the treatment of Krabbe disease (globoid cell leukodystrophy), a rare, fatal lysosomal storage disorder of the nervous system caused by deficiency of the galactocerebrosidase (GALC) enzyme.[1][7][9][10] As a conventional gene therapy distinct from Neurogene’s EXACT-regulated programs, NGN-201 is designed to deliver a functional copy of the defective gene to restore GALC activity, reduce neurotoxic substrate accumulation, and prevent or ameliorate progressive demyelination and neurodegeneration characteristic of Krabbe disease.[1][9][10] The program is currently at the IND‑enabling/preclinical stage with no active clinical trials yet reported.[1][7][9][10]