AGC Biologics to manufacture dual AAV therapies for AAVantgarde

AGC Biologics has signed a manufacturing agreement with  AAVantgarde, strengthening its leadership in the adeno-associated virus (AAV) sector.

Under the collaboration, AGC Biologics will provide GMP-compliant production for AAVantgarde’s two dual AAV gene therapies targeting previously untreatable inherited retinal diseases:

• AAVB-039 for Stargardt disease, the most common inherited form of macular degeneration and a leading cause of vision loss in young adults. This therapy is currently in Phase 1/2 clinical trials in the US, UK, and Europe.

• AAVB-081 for retinitis pigmentosa caused by Usher Syndrome Type 1B, a rare condition primarily affecting children. AAVB-081 is the first dual AAV gene therapy clinically tested for an ocular disease.

Both programs leverage AGC Biologics’ BravoAAV™ suspension platform combined with an innovative dual AAV vector approach. This strategy addresses a key limitation of conventional AAV therapies: the limited packaging capacity of ~4.7 kilobases. In this dual vector system, the therapeutic gene is split into two halves, each packaged in a separate AAV vector and delivered simultaneously. Once inside the target cell, the two halves are reassembled into a complete, functional gene—enabling treatment of diseases previously untreatable due to vector size constraints.

While financial terms were not disclosed, batch costs are expected to range between US$1–5 million due to the complexity of dual vector therapies.

AGC Biologics’ Milan Cell and Gene Center of Excellence will support clinical supply across early and late development stages. The BravoAAV™ platform is a suspension-based AAV manufacturing system designed for high yields and rapid scale-up. It supports both single and dual vector approaches, allows clinical supply within nine months of gene identification, and is optimized for large and complex genes that cannot fit into conventional single AAV vectors. The Milan facility brings three decades of experience, over ten EMA and FDA-approved products, and extensive expertise in complex cell and gene therapy projects.

“Partnering with AGC Biologics provides us with access to high-quality manufacturing capacity and accelerates the advancement of our innovative gene therapy pipeline,” said Natalia Misciattelli, CEO of AAVantgarde.