Amplo Biotechnology, a San Diego, CA-based biotech company dedicated to developing innovative AAV-based gene therapies for neuromuscular junction disorders, announced the award of a Fast Track Phase I/II STTR grant from the NIH-NIAMS.
This grant will provide funding for the advancement of AMP-201, an AAV-ColQ gene therapy designed to address the severe congenital myasthenic syndrome (CMS) caused by Collagen Q (ColQ) Deficiency.
Under an exclusive licensing agreement, Amplo Biotechnology has gained access to AAV-ColQ data developed by Professor Kinji Ohno’s laboratory at Nagoya University, which forms the basis of AMP-201. In the Fast Track STTR project, the company will collaborate with Professor Ricardo Maselli, a CMS expert neurologist and researcher at UC-Davis.
Amplo Biotechnology’s development of AMP-201 builds upon it’s progression of AMP-101, an AAV-DOK7 gene therapy targeting Dok-7 congenital myasthenic syndrome, which Amplo expects to bring to clinical trial in 2024. Upon demonstrating initial safety and efficacy, the company plans to expand into other neuromuscular conditions, leveraging the vast potential of AMP-101 and AMP-201 to significantly enhance the quality of life for the majority of CMS patients.
Led by Dr. Patricio Sepulveda, CEO, Amplo Biotechnology is a late-stage preclinical AAV gene therapy company, committed to leveraging gene therapy advancements to improve neuromuscular junction function across various medical conditions. The company’s flagship program, AMP-101, has undergone rigorous preclinical studies, enabling the submission of a Clinical Trial Application in the UK for Dok7 CMS. Amplo Biotechnology’s technologies are rooted in ground-breaking research conducted at renowned institutions such as the University of Tokyo, Oxford University, and Nagoya University.
FinSMEs
29/05/2023