Amplo Biotechnology, a leading US-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-NINDS. This highly competitive grant will provide substantial 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, Amplo 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.
“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 revolutionize the lives of CMS patients,” expressed Dr. Patricio Sepulveda, CEO of Amplo Biotechnology, who holds a Ph.D. in the field and an MBA.