Our innovative delivery system
Adeno-associated virus (AAV) is a small virus that infects humans but does not cause disease. It is uniquely suited for gene therapy application – because it cannot replicate on its own, it is unlikely to spread within the body. Human clinical trials have consistently found AAV to be safe. The same clinical trials have found that vectors (gene delivery vehicles based on AAV) are capable of causing long-term therapeutic protein expression in humans, meaning long-lasting treatment for patients. Therefore, AAV vectors have advantages compared to many other viral and non-viral based gene delivery platforms in both function and safety for correcting genetic-based diseases.
AGTC has developed an AAV vector manufacturing platform comprised of a proprietary, patented production method and proven downstream purification operations integrated into a GMP-compliant format capable of AAV vector generation at a commercial scale. The manufacturing platform is flexible and efficient, accommodating multiple genetic targets and AAV variants, or serotypes, while producing vector in sufficient quantity for treatment of both confined-space and systemic disease candidates. AGTC is currently partnering with SAFC Pharma to make its proprietary production system available to potential academic and corporate partners.