AMP-101 / Amplo Biotech - LARVOL DELTA

The pre-clinical development of gene augmentation therapy to stabilize the neuromuscular junction in Dok-7 Congenital Myasthenic Syndrome reveals a mechanism of action that has the potential to treat many neuromuscular junction diseases using the same vector. (ESGCT 2024) - "Here, we report, for the first time, that Dok-7 is not only safe in non-human primates, but it is also active, and it leads to enlargement of the neuromuscular junction. This not only significantly increases the probability of success of AMP-101 for Dok-7 CMS but also reinforces the idea of AMP-101’s use in other conditions."

Preclinical • Gene Therapies • Genetic Disorders • Respiratory Diseases

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

Dose escalation pre-clinical trial of novel DOK7-AAV in mouse model of DOK7 congenital myasthenia. (PubMed, Brain Commun) - "Western blot analysis demonstrated robust expression of DOK7 in the diaphragm and tibialis anterior muscles. These data show that AMP-101 is an effective treatment in a mouse model for DOK7 congenital myasthenia, and suggests that AMP-101 is a promising candidate to move forward to clinic trials as a gene therapy for patients."

Journal • Preclinical • Gene Therapies • Genetic Disorders • Myasthenia Gravis