AMP-101 / Amplo Biotech - LARVOL DELTA

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