FDA Grants Fast Track Designation to AAVantgarde Bio’s AAV Gene Therapy for Stargardt Disease

MILAN, Aug. 12, 2025 (GLOBE NEWSWIRE)  – In a significant move for the inherited retinal disease community, AAVantgarde Bio announced that its gene therapy program, AAVB-039, has been granted Fast Track Designation by the U.S. Food and Drug Administration (FDA). The therapy is designed to treat Stargardt disease, the most common form of inherited macular degeneration and a leading cause of vision loss in children and young adults.

The FDA’s decision, which follows the clearance of an Investigational New Drug (IND) application for AAVB-039, is a major step toward addressing a critical unmet medical need.

A Novel Approach for a Challenging Disease

Stargardt disease is caused by mutations in the ABCA4 gene. This gene is exceptionally large, making it impossible to fit into a single standard gene therapy vector. AAVantgarde Bio’s solution is a next-generation, dual AAV gene therapy that can deliver the full-length ABCA4 protein.

“We are thrilled that the FDA has granted Fast Track Designation for AAVB-039, as it underscores the urgent unmet need and important potential of our program,” said Dr. Natalia Misciattelli, CEO of AAVantgarde. “This enables us to accelerate development efforts as we work to bring a potentially transformative therapy to patients as quickly as possible.”

There are currently no approved treatments for Stargardt disease, which affects an estimated 60,000 to 75,000 individuals across the U.S. and E.U. AAVB-039 has the potential to benefit all patients with ABCA4 mutations.

AAVB-039 is currently being evaluated in a Phase 1/2 clinical trial, named CELESTE, to assess its safety, tolerability, and preliminary effectiveness.