ALN-SOD

ALN-SOD is an investigational small interfering RNA (siRNA) therapeutic developed by Alnylam Pharmaceuticals for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. The drug works via RNA interference to selectively inhibit the expression of SOD1, a gene whose mutations are implicated in a subset of ALS cases known as SOD1-ALS. By silencing SOD1 mRNA, ALN-SOD aims to reduce toxic gain-of-function effects caused by mutant SOD1 protein accumulation in motor neurons. The drug is administered intrathecally and is currently being evaluated for safety and tolerability in first-in-human Phase I clinical trials[3][4][5][7]. Regeneron Pharmaceuticals has also been involved as a sponsor/collaborator in recent clinical studies[5].