AMT-162

AMT-162 is an investigational, one-time, intrathecally administered gene therapy designed for the treatment of amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene. It utilizes a recombinant adeno-associated virus serotype rh10 (AAVrh10) vector to deliver a microRNA (miRNA) that specifically targets and degrades SOD1 messenger RNA, thereby reducing the production of toxic mutant SOD1 protein. This approach aims to slow or potentially halt disease progression in patients with SOD1-mutant ALS. The therapy is being developed by uniQure and originated from research at the University of Massachusetts Medical School. AMT-162 has received Orphan Drug designation and Fast Track status from regulatory authorities due to its potential benefit for this rare and fatal neurodegenerative disorder[1][3][6][7][8].

Other names

ABP 102ABP102ABP-102