AAVogen

AAVogen, Inc. is a preclinical-stage biotechnology firm founded in August 2015 by Dr. Buel "Dan" Rodgers, who also serves as the company's CEO. The company's mission is deeply personal, as it was established by a family affected by several muscle-wasting diseases, which drives its commitment to developing effective therapies. Dr. Rodgers brings over 27 years of research experience in skeletal and cardiac muscle development, having held faculty positions at Johns Hopkins University School of Medicine and Washington State University, where he founded the Washington Center for Muscle Biology.

The company focuses on developing gene therapies to combat muscle-wasting diseases such as inclusion body myositis (IBM), Duchenne muscular dystrophy (DMD), and cancer cachexia. AAVogen operates in the biotechnology market, specifically within the gene therapy sector, which utilizes adeno-associated viral (AAV) vectors to deliver therapeutic genes into tissues. Its business model involves a hybrid approach of product development and out-licensing, maintaining a virtual structure to keep operational costs low while pursuing seed funding and grants to support preclinical development. The company has successfully secured multiple grant awards from the National Institutes of Health (NIH), including from the National Institute on Aging (NIA) and the National Cancer Institute (NCI), to advance its research.

AAVogen's primary product candidate is AVGN7, a gene therapeutic designed to introduce the Smad7 gene into muscle tissue using a non-pathogenic AAV vector. The Smad7 protein works by blocking multiple molecular pathways that inhibit muscle growth and cause muscle protein degradation. This mechanism allows AVGN7 to not only prevent muscle wasting but also to restore muscle mass and strength. Pre-clinical studies have shown that AVGN7 can increase muscle mass, improve strength, and correct deficits in cardiac muscle structure and output. A key differentiator for AVGN7 is its design for muscle-specificity, which aims to avoid off-target effects that have been a challenge for other therapies in development. The company is advancing AVGN7 through preclinical, IND-enabling studies, with sporadic inclusion body myositis as its lead indication, alongside programs for DMD and cancer cachexia.

Keywords: gene therapy, muscle wasting, adeno-associated virus, AAVogen, AVGN7, Duchenne muscular dystrophy, inclusion body myositis, cancer cachexia, Buel Dan Rodgers, Smad7, neuromuscular disorders, orphan drugs, preclinical biotech, muscle biology, AAV vectors, myopathies, sarcopenia