AAVantgarde

Pioneering large gene delivery to improve the life of patients with debilitating diseases, starting with inherited retinal disorders.

AAVantgarde is a clinical-stage international biotechnology company developing two proprietary Adeno-Associated Viral (AAV) vector platforms for large gene delivery. AAV gene therapy has proven to be transformative in the treatment of genetic illnesses, but the small vector payload limits its application. To solve the constraint of oversize gene delivery, AAVantgarde is developing two AAV-based large gene delivery platforms: one leveraging DNA recombination (dual-hybrid); and one based on protein trans-splicing (AAV-intein mediated). The company is validating these platforms in two programs: Usher Syndrome Type 1B-associated retinitis pigmentosa (USH1B) and Stargardt disease (STGD) respectively. USH1B is an autosomal-recessive genetic disorder due to mutations in the MYO7A gene characterized by profound hearing loss and adolescent-onset retinitis pigmentosa that results in loss of vision which is severe by midlife. STGD is the most prevalent inherited macular dystrophy and is an autosomal-recessive genetic disorder due to mutations in the ABCA4 gene characterized by progressive loss of central vision starting from childhood, leading to profound vision loss. They are pioneering the next generation of gene therapies, opening the opportunity for genetic medicine delivery to more underserved patients with debilitating diseases, starting with ophthalmology indications, but with the potential to expand to different therapeutic areas.

• Genetics/Genomics
• Engineered Biology
• Neurology
• Platform Technology
• Eye Health
• Rare diseases
• Translational Medicine