AMP-101

AMP-101 is an investigational adeno-associated virus (AAV)-based gene therapy designed to treat congenital myasthenic syndromes (CMS) caused by mutations in the DOK7 gene. The therapy uses a recombinant AAV serotype rh74 vector to deliver human DOK7 cDNA under the control of a muscle-restricted promoter. DOK7 is a cytoplasmic adaptor protein essential for the formation and stabilization of neuromuscular junctions; mutations in this gene are among the most common causes of CMS worldwide. Preclinical studies have shown that AMP-101 can restore neuromuscular function and enlarge neuromuscular junctions in mouse models with severe DOK7-related CMS phenotypes, resulting in improved muscle strength and survival comparable to wild-type controls. The drug has demonstrated a favorable safety profile and consistent manufacturability at high yields and purity. Developed by Amplo Biotechnology under license from the University of Tokyo, AMP-101 has received orphan drug designation for congenital myasthenic syndromes and is expected to enter clinical trials soon[1][2][3][6][8][9].

Other names

AMP 101AMP101AMP-101AAVDok7 gene therapyAAVDok-7 gene therapyAAVDok 7 gene therapy