The company develops AAV-based gene therapies for neuromuscular junction disorders. Credit: THAVIS 3D on Unsplash.

Amplo Biotechnology has received a fast track phase I/II STTR grant from the NIH-NIAMS [National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases] for its gene therapy AMP-201.

The company will receive substantial funding to advance AAV-ColQ gene therapy.

AMP-201 addresses the severe congenital myasthenic syndrome (CMS) caused by collagen Q (ColQ) deficiency.

As part of an exclusive licensing agreement, Amplo Biotechnology secured access to AAV-ColQ data developed by Professor Kinji Ohno’s laboratory at Nagoya University, which forms the basis of the therapy.

Amplo Biotechnology will work with UC-Davis CMS expert neurologist and researcher, Professor Ricardo Maselli, on the fast track STTR project.

Amplo CEO Dr Patricio Sepulveda stated: “Amplo is thrilled to collaborate with UC-Davis in the development of AMP-201, addressing the critical unmet needs of patients suffering from ColQ CMS.

“By combining the power of Amp-101 and AMP-201, we are confident in our ability to revolutionise the lives of CMS patients.”

The company developed the gene therapy by building upon its progression of the AAV-DOK7 gene therapy AMP-101 that targets Dok-7 congenital myasthenic syndrome. It is expected to enter clinical trials in 2024.

Amplo Biotechnology plans to expand into other neuromuscular conditions, once the trial has demonstrated initial safety and efficacy.

It also develops adeno-associate virus-based gene therapies for neuromuscular junction disorders.

Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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