SHERIDAN, WYOMING - December 3, 2025 - AGC Biologics has signed a new manufacturing agreement with AAVantgarde Bio to produce dual-vector AAV gene therapies for Stargardt disease and retinitis pigmentosa, positioning its BravoAAV™ platform at the center of next-generation treatments for inherited retinal disorders with no approved therapies.
Dual-vector AAV platform targets previously "untreatable" retinal genes
Under the deal, AGC Biologics will provide GMP manufacturing for AAVantgarde's two clinical-stage candidates: AAVB-039 for Stargardt disease, the most common inherited form of macular degeneration in children and young adults, and AAVB-081 for retinitis pigmentosa caused by Usher syndrome type 1B. Both programs are in Phase 1/2 trials, with AAVB-039 being tested in the U.S., UK and Europe, and AAVB-081 representing the first-ever dual AAV gene therapy in an ocular indication.
Many genes implicated in inherited retinal disease are too large to fit inside a single AAV vector, given the roughly 4.7 kb payload limit. AAVantgarde's approach splits these oversized genes into two halves, packages each into a separate AAV vector and relies on cellular machinery to reassemble the full-length gene once both vectors enter the target photoreceptor cells. This dual-vector strategy enables developers to address disease-causing genes that were previously out of reach for standard AAV therapies.
BravoAAV™ suspension platform underpins rapid clinical scaling
AGC Biologics will run the projects from its Milan Cell and Gene Center of Excellence, applying its BravoAAV™ suspension platform to deliver high-yield AAV production for both early and late-phase clinical supply. With 30 years of experience and 10 product approvals from the European Medicines Agency and the U.S. Food and Drug Administration, the site is positioned as a global hub for complex cell and gene therapy manufacturing.
The BravoAAV™ platform provides a ready-to-use, scalable process designed to compress timelines from gene to clinic to as few as nine months. For emerging ophthalmology biotechs, that combination of speed, regulatory track record and technical depth is critical in highly specialized indications where patient populations are small and trial execution windows are tight.
AAVantgarde secures industrial backbone for first-in-class ocular programs
For AAVantgarde, fresh off a Series B financing round, the partnership locks in the industrial backbone needed to move its lead programs through early proof-of-concept and toward pivotal development. "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."
By aligning dual-vector innovation with an established AAV manufacturing platform, AAVantgarde aims to de-risk scale-up while maintaining the flexibility to adapt dose, construct design and formulation as clinical data emerge from its Stargardt and Usher type 1B programs.
Strengthening AGC Biologics' AAV leadership across modalities and phases
For AGC Biologics, the deal is another step in a deliberate strategy to expand leadership in the AAV market and position BravoAAV™ as a go-to solution for complex vector architectures. "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."
Beyond AAV, the Milan site supports lentiviral and retroviral vector programs, giving sponsors a single partner capable of handling multi-modality pipelines as they expand beyond ophthalmology into other rare and genetic disease indications.
Strategic impact for inherited retinal disease pipelines
The AAVantgarde partnership underscores how specialized CDMOs are becoming central to the industrialization of dual-vector and other advanced AAV formats. For inherited retinal diseases such as Stargardt and retinitis pigmentosa, where patients face progressive and irreversible vision loss and no approved therapies, the ability to reliably produce dual-vector constructs at GMP scale is a key precondition for moving beyond scientific promise toward real-world impact.
By combining AAVantgarde's disease-focused genetic strategies with AGC Biologics' BravoAAV™ platform and regulatory experience, the collaboration is designed to shorten the path from discovery to late-stage development-and ultimately broaden access to gene therapies for patients with inherited blindness.
For more information on AGC Biologics' BravoAAV™ platform and AAV manufacturing services, visit https://www.agcbio.com/capabilities/bravo-adeno-associated-viral-vector-platform-cdmo.