AMP-201 / Amplo Biotech - LARVOL DELTA

Pre-clinical development of gene augmentation therapy to restore Acetyl Cholinesterase activity in Collagen Q Congenital Myasthenic Syndrome using a platform approach to AAV gene therapy development. (ESGCT 2024) - "Given the rarity of ColQ CMS (∼1,000 patients in the major economies) and its similarity to other CMS (e.g., Dok-7 CMS), we applied a platform approach to decrease the costs of development and time to the clinic. In this study, we report positive results from our pharmacology study and small-scale suspension manufacturing, and we will describe how we plan to leverage AMP-101’s (AAV Dok-7) development for AMP-201."

Gene therapy • Preclinical • Gene Therapies

Development of a platform gene therapy approach for Congenital Myasthenic Syndromes (ESGCT 2024) - "Seeking to increase capital efficacy and timeline to the clinic, Amplo has streamlined the process of testing new adeno-associated virus (AAV) therapeutics by applying a platform approach consisting of similar manufacturing processes and starting reagents (including the same combination of capsid, promoter, and enhancers), analytical and manufacturing methods and only changing the animal model and the transgene delivered. The development of our unified platform approach has proven to be an effective strategy for CMS, leading to the successful pre-clinical development of Amplo's first two gene therapies, AMP-101 (AAV Dok-7) and AMP-201 (AAV ColQ)."

Gene therapy • Gene Therapies