GNK-301

GNK-301 is a humanized monoclonal antibody developed by GeNeuro for the treatment of amyotrophic lateral sclerosis (ALS). It specifically targets and neutralizes the HERV-K ENV protein—a neurotoxic envelope protein encoded by human endogenous retrovirus K (HERV-K)—which has been implicated in neuronal cell death and blood-brain barrier dysfunction in ALS. Preclinical studies have demonstrated that GNK-301 can both detect the presence of HERV-K ENV in cerebrospinal fluid and neutralize its harmful effects, thereby protecting neurons and maintaining blood-brain barrier integrity. This precision medicine approach aims to identify ALS patients with detectable HERV-K ENV who may benefit from targeted therapy with GNK-301. The drug is currently in preclinical development[1][2][5][6][7][8].