MILAN, August 12, 2025 — AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing next-generation gene therapies for inherited retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for AAVB-039, the company’s gene therapy program for Stargardt disease secondary to biallelic mutation in ABCA4. The Investigational New Drug (IND) application for AAVB-039 was cleared to proceed by the FDA. Stargardt's disease is the most common inherited form of macular degeneration and a leading cause of vision loss in children and young adults. AAVB-039 addresses the underlying genetic cause of the disease by providing the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations.
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 due to the serious nature of challenges faced by patients living with Stargardt disease. This designation represents an important milestone for our program and enables us to accelerate development efforts as we work to bring a potentially transformative therapy to patients as quickly as possible.
Dr. Natalia Misciattelli
Chief Executive Officer of AAVantgarde
The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs and biologics intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial, which is assessing safety, tolerability, and preliminary efficacy in patients with Stargardt disease.
Contact:
Magda Blanco – Head of Corporate Development
Email: info@aavantgarde.com