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AMP-201

Development stage: Preclinical
Lead developer: Amplo Biotechnology
Modality: Gene Therapies
Administration: Intravenous

Overview

AMP-201 is an adeno-associated virus (AAV) gene therapy candidate being developed by Amplo Biotechnology for the treatment of Collagen Q (ColQ) Congenital Myasthenic Syndrome (CMS). ColQ CMS is a rare genetic neuromuscular disorder caused by mutations in the *COLQ* gene, which encodes the collagen Q protein responsible for anchoring acetylcholinesterase (AChE) at the neuromuscular junction. AMP-201 is designed to deliver a functional copy of the *COLQ* gene to restore the presence of AChE at the synapse, thereby improving synaptic transmission and motor function. The program received Phase I/II STTR funding in May 2023 and is currently in preclinical development for lead candidate selection.

Other names: AMP-201AMP201AMP 201AAV-ColQ

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