MILAN, October 3, 2025 — AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted AAVB-039, the company’s investigational gene therapy for the treatment of Stargardt disease secondary to biallelic mutation in ABCA4, Orphan Drug Designation (ODD). The company has also received Clinical Trial Authorisation (CTA) approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA).
Stargardt disease is the most common inherited form of macular degeneration and is a leading cause of vision loss in children and young adults. AAVB-039 addresses the root cause of the disease by delivering the full-length ABCA4 protein, with the potential to benefit all patients with ABCA4 mutations. The Investigational New Drug (IND) and CTA applications for AAVB-039 have been cleared to proceed by the FDA and the MHRA, respectively, and the program also holds Fast Track Designation in the US.
“The Orphan Drug Designation and UK CTA approval represent two important regulatory milestones for AAVB-039 and reflect the FDA’s and MHRA’s acknowledgement of the urgent need for treatments for patients living with Stargardt disease,” said Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde. “With Fast Track Designation already in place, we now have a suite of regulatory incentives that will help accelerate development and bring this potentially transformative therapy to patients and families as efficiently as possible.”
The FDA’s Orphan Drug Designation program is intended to advance the development of drugs and biologics for rare diseases affecting fewer than 200,000 people in the United States. Benefits of ODD include tax credits for qualified clinical testing, waiving of certain FDA application fees, and, if approved, seven years of U.S. market exclusivity.
AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial, which assesses safety, tolerability, and preliminary efficacy of AAVB-039 in patients with Stargardt disease across three dose levels.