AVGN7 / AAVogen - LARVOL DELTA

Validation of a Göttingen Minipig Gene Therapy Biodistribution Assay for AVGN7 Using Droplet Digital Polymerase Chain Reaction (ASGCT 2025) - "These data not only validate the assay for tracking and quantifying AVGN7 in preclinical studies with Göttingen minipigs, but additionally address challenges of using pig models in general while providing a procedural road map for developing other AAV biodistribution assays. Disease Focus of Abstract:Muscle Disorders"

Gene therapy • Polymerase Chain Reaction • Gene Therapies • SMAD7

Case Study on Insect Cell-Produced AAV6 Gene Therapy Vector in a Rhabdovirus-Free Sf9 Cell Line (ASGCT 2024) - "This presentation provides a case study of production and activity of AVGN7, an AAV6-based gene therapeutic for treating rare neuromuscular diseases, using this system...Preliminary studies demonstrate the ability to produce high levels of AAV expression using a high cell density, high MOI (multiplicity of infection) methodology. Downstream processes for purification will be discussed along with assessments of bioactivity and post-translational processing of capsid proteins."

Case study • Gene therapy • Preclinical • CNS Disorders • Gene Therapies • Infectious Disease

Development and validation of a model gene therapy biodistribution assay for AVGN7 using digital droplet polymerase chain reaction. (PubMed, Mol Ther Methods Clin Dev) - "The procedural road map provided exceeds recently established standards. It is also relevant to many IND-enabling processes, as validated ddPCR assays can be used in biodistribution studies and with vector titering and manufacturing quality control."

Gene therapy • Journal • Polymerase Chain Reaction • Gene Therapies • SMAD7