Pharmacology and Safety profile of AMP-101, Dok-7 gene therapy to stabilise the neuromuscular junction


Translational Research

Poster Number: 305


Patricio Sepulveda, PhD, MBA, Amplo Biotechnology

Amplo Biotechnology is developing AMP-101, an Adeno Associated Viral Vector to augment the levels of Dok-7 (AAV-DOK7). Dok-7 is a non-catalytic cytoplasmic adaptor protein that is essential for the formation of neuromuscular synapses.
Dok-7 mutations lead to a severe form of congenital myasthenic syndrome (CMS). Delivery of AAV-DOK7 has shown therapeutic benefits in CMS, Emery-Dreyfuss, Amyotrophic Lateral Sclerosis, Spinal Muscle Atrophy, sarcopenia, and nerve injury.
Amplo has developed AMP-101, which is a clinically optimised version of AAV-DOK7. Our findings support a pharmacology and safety profile compatible with clinical and commercial development.