Amplo Biotechnology, a privately held US-based biotech focused on developing AAV (Adeno-associated Virus)-based gene therapies for diseases affecting the neuromuscular junction, today announced the successful closing of an undisclosed Series Seed financing. The Seed round was led by BioBrit, LLC., joined by Casdin Capital, SeedFolio and small group of sophisticated, biotech angel investors. In conjunction with the financing, Dan Bradbury (BioBrit, LLC.) has been appointed Chair of the Board. It was also announced that Amplo was awarded a NIH Fast-Track Phase I/II SBIR grant for the development of AMP-101 gene therapy.
The latest funding round will be used to advance AMP-101, an AAV carrying Dok-7, to large scale manufacturing and to complete IND-enabling studies. AMP-101 was invented as a means to treat Dok-7 congenital myasthenic syndrome. AMP-101 has also shown to revert age-related loss in muscle function by improving the neuromuscular junction and decreasing denervation in mice. AMP-101 has also shown to increase survival and functional benefits in mouse models of Amyotrophic Lateral Sclerosis and Emery-Dreyfuss muscular dystrophy.
Amplo secured FDA orphan drug designation for AMP-101 in January 2020. Amplo, plans to begin clinical trials in Dok-7 congenital myasthenic syndrome in 2023, with aims to move into other neuromuscular conditions once initial safety and efficacy are demonstrated.
"When we think about AMP-101, we see the potential to treat many neuromuscular diseases with a single gene therapy. The closing of the Seed financing will allow us to rapidly execute on a plan that has received FDA and MHRA input," said Patricio Sepulveda Ph.D. MBA, CEO of Amplo Biotechnology.
In addition, AMP-101, Amplo is developing other AAVs to treat diseases of the neuromuscular junction.
About Amplo Biotechnology Amplo is a late preclinical AAV gene therapy company focused on developing therapies for congenital myasthenic syndromes and conditions of the neuromuscular junction.