AVGN7 / AAVogen - LARVOL DELTA

Codon-optimized human Smad7 gene therapy enhances skeletal muscle mass and function in a murine model of Duchenne muscular dystrophy. (PubMed, Gene Ther) - "These data suggest that AVGN7.2 is capable of enhancing dystrophic muscle function without exacerbating muscle degeneration. Although these functional effects were partial, they resembled those of several dystrophin-targeting drugs and suggest that combinatorial approaches may safely yield further benefit."

Journal • Preclinical • Duchenne Muscular Dystrophy • Fibrosis • Gene Therapies • Genetic Disorders • Immunology • Muscular Dystrophy • SMAD7

Murine toxicology assessment of avgn7.2, a novel gene therapeutic for inclusion body myositis and other muscle wasting diseases. (PubMed, Gene Ther) - "Vector biodistribution mirrored previously published patterns with liver followed by striated muscle having the highest levels, although overexpression of hSMAD7 and the S6RP biomarker only occurred in muscle. These data suggest that AVGN7.2 was well-tolerated even at doses known to elicit clinical toxicities with muscle-tropic AAV capsids other than AAV6."

Journal • Preclinical • Gene Therapies • Hematological Disorders • Immunology • Myositis • Ophthalmology • Rare Diseases • SMAD7

Validation of a Göttingen Minipig Gene Therapy Biodistribution Assay for AVGN7 Using Droplet Digital Polymerase Chain Reaction. (PubMed, AAPS J) - "High recovery of AVGN7 during nucleic acid extraction was also demonstrated for all tissues. 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."

Journal • Polymerase Chain Reaction • Gene Therapies • SMAD7

Case Study on an Insect Cell-Produced AAV6 Gene Therapy Vector for Neuromuscular Disease (ASGCT 2025) - "This presentation provides a case study of the production and activity of AVGN7, an AAV6-based gene therapeutic for treating rare neuromuscular diseases...The combination of an upstream process capitalizing on high cell densities with enrichment procedures specific to the AAV6 serotype results in a high yield of vector exceptionally enriched for full capsids. Disease Focus of Abstract:Neuromuscular Disorders"

Case study • Clinical • Gene therapy • CNS Disorders • Gene Therapies • Infectious Disease • SMAD7

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