AGC Biologics inks gene therapy manufacturing agreement with AAVantgarde

Seattle-headquartered contract development and manufacturing organization AGC Biologics will provide GMP manufacturing for clinical-stage biotechnology company AAVantgarde’s two novel candidates designed to address progressive and irreversible vision loss, for which there are currently no approved therapies.

The projects will leverage AGC Biologics’ BravoAAV suspension platform and use an innovative dual adeno-associated virus (AAV) vector approach to overcome a common obstacle in gene therapies — genes that are too large for a single AAV vector — by splitting the therapeutic gene into two halves.

“Each half is then packaged into its own separate AAV vector,” AGC explains in its press release. “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.”

According to AGC, the CDMO’s BravoAAV platform provides ready-to-use, high-yield GMP processes — with adhesion manufacturing scales up to 750L and suspension scales up to 2,000L — that can accelerate timelines from gene to clinic to as few as nine months. The platform offers GMP cell lines, representative scale-down processes, in-house pDNA, production and purification protocols, small-scale and large-scale manufacturing, in-house analytics, as well as fill-finish services.

AGC’s Biologics Milan Cell and Gene Center of Excellence in Italy will support manufacturing for early and late-phase clinical supply for the two therapeutics: AAVB-039 for Stargardt disease which is in a first-in-human Phase 1/2 clinical trial, and AAVB-081 for retinitis pigmentosa which is in Phase 1/2 development and represents the first-ever dual AAV gene therapy tested clinically in an ocular indication.

In addition to its Milan facility, AGC has production and manufacturing centers in Denmark, Germany, and Japan, as well as its U.S. facilities in Seattle and Boulder and Longmont, Colorado.  

“We are delighted to partner with AGC Biologics, a world-class CDMO with deep expertise in viral vector manufacturing,” CEO Natalia Misciattelli said in a statement.  “This partnership marks an important milestone for AAVantgarde as we continue to advance our innovative gene therapy pipeline in the clinic.”