On the heels of AAVantgarde closing a Series B financing round, AGC Biologics announced a new manufacturing agreement with the biotechnology company, marking AGC Biologics’ latest advancement in the adeno-associated virus market. Under this agreement, AGC Biologics will provide Good Manufacturing Practice manufacturing for AAVantgarde’s two novel candidates designed to address progressive and irreversible vision loss where there are currently no approved therapies:

• AAVB-039 for Stargardt disease. This therapy addresses the most common inherited form of macular degeneration and a leading cause of vision loss in children and young adults, affecting approximately 1 in 6,500 individuals. AAVB-039 is in a first-in-human Phase 1/2 clinical trial ongoing in the U.S., UK and Europe.
• AAVB-081 for retinitis pigmentosa (caused by Usher syndrome type 1B). Retinitis pigmentosa from Usher syndrome affects approximately 1 in 50,000 individuals, typically under the age of 10. This therapy is in Phase 1/2 development and represents the first-ever dual AAV gene therapy tested clinically in an ocular indication.

The projects will leverage AGC Biologics’ BravoAAV™ suspension platform and use an innovative dual AAV vector approach to overcome a common obstacle in gene therapy: genes that are too large for a single AAV vector.

Because many therapeutic genes are too large to fit within a single AAV delivery vector, this method splits the therapeutic gene into two halves. Each half is then packaged into its own separate AAV vector. When administered, both vectors enter the target cell, where the two pieces are reassembled to form the complete, functional gene. This enables the treatment of diseases that were previously out of reach for the standard single-vector approach due to the AAV vector's limited capacity of 4.7 kilobases.

"We are delighted to partner with AGC Biologics, a world-class CDMO with deep expertise in viral vector manufacturing. This partnership marks an important milestone for AAVantgarde as we continue to advance our innovative gene therapy pipeline in the clinic,” said Natalia Misciattelli, CEO of AAVantgarde. “Working with AGC will ensure that we have access to the highest quality manufacturing capabilities, enabling us to deliver transformative therapies for patients."

The AGC Biologics Milan Cell and Gene Center of Excellence will support manufacturing for early and late-phase clinical supply for the two therapeutics. With a 30-year record of accomplishment and 10 product approvals from the European Medicines Agency and the U.S. Food and Drug Administration, the Milan facility is a global leader with deep expertise in complex cell and gene therapy projects. The BravoAAV™ platform offers a ready-to-use, high-yield process that can accelerate timelines from gene to clinic to as few as nine months.

“This collaboration with AAVantgarde allows us to contribute to the noble mission of addressing this significant unmet medical need and showcase our technical precision across many vector types as well as various stages of clinical development,” said Luca Alberici, Executive Vice President of AGC Biologics’ Global Cell & Gene Technologies Division. “As a dedicated team with a focus on reliability and stability, we are honored to develop, manufacture, and optimize lenti-, retro-, and adeno-associated viral vectors for developers with complex, cutting-edge projects.”