AGC Biologics to Manufacture AAVantgarde’s Dual-Vector Gene Therapies for Inherited Retinal Disorders

AGC Biologics has entered into a manufacturing agreement with AAVantgarde, a biotechnology company focused on gene therapies for inherited retinal diseases. The collaboration follows AAVantgarde’s recent Series B financing round and supports the development of two investigational therapies using a dual-vector AAV approach.

Agreement Covers Two Gene Therapy Candidates in Clinical Development

As part of the agreement, AGC Biologics will provide GMP (Good Manufacturing Practice) manufacturing services for AAVantgarde’s two lead candidates targeting inherited retinal conditions with no currently approved therapies:

       • AAVB-039 for Stargardt Disease: AAVB-039 is designed to treat Stargardt disease, the most common inherited form of macular degeneration. The condition affects approximately 1 in 6,500 individuals, often causing vision loss in children and young adults. This candidate is being evaluated in a first-in-human Phase 1/2 clinical trial taking place in the United States, United Kingdom, and Europe.

       • AAVB-081 for Retinitis Pigmentosa Caused by Usher Syndrome Type 1B: AAVB-081 is intended for patients with retinitis pigmentosa resulting from Usher syndrome type 1B, a rare condition that affects around 1 in 50,000 individuals, typically under the age of 10. The therapy is in Phase 1/2 development and is reported to be the first dual AAV gene therapy to enter clinical testing for an ocular disease.

Dual AAV Vector Strategy Enables Delivery of Large Genes

The gene therapy candidates are based on a dual-vector AAV delivery platform that addresses a known limitation in traditional AAV gene therapies, the limited cargo capacity of AAV vectors, which is approximately 4.7 kilobases.

The approach involves:

       • Dividing the therapeutic gene into two parts.

       • Packaging each part into a separate AAV vector.

       • Delivering both vectors to the same cell, where the full-length gene is reassembled and can produce the desired protein.

This dual-vector technique is designed to enable the treatment of conditions that cannot be addressed using single-vector systems due to size constraints.

The programs will utilize AGC Biologics’ BravoAAV™ suspension platform, which supports high-yield production processes and aims to streamline development timelines.

Manufacturing to Be Conducted at AGC Biologics’ Milan Facility

Production will take place at the AGC Biologics Milan Cell and Gene Center of Excellence, a site with more than three decades of experience in biologics and a track record that includes 10 product approvals from the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA).

The BravoAAV platform, developed by AGC Biologics, is intended to support accelerated development, with timelines from gene sequence to clinical material in as few as nine months.

“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.