• 06 Oct 2025
• Admin
• News Article

AAVantgarde Bio Receives FDA Orphan Drug Designation for AAVB-039 in Stargardt Disease

AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing gene therapies for inherited retinal diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AAVB-039, its investigational gene therapy for Stargardt disease caused by biallelic ABCA4 mutations.

Targeting the Root Cause of Stargardt Disease

AAVB-039 is designed to address the root cause of Stargardt disease by delivering the full-length ABCA4 protein, which is too large for conventional AAV vectors. This approach may benefit patients with any ABCA4 mutation.

The therapy is currently being evaluated in the CELESTE interventional clinical trial, while patient recruitment continues in the STELLA observational study.

AAVantgarde has also received Clinical Trial Authorisation (CTA) from the UK Medicines and Healthcare products Regulatory Agency (MHRA), allowing clinical activities to expand in the UK.

Advancing Regulatory Milestones

“The Orphan Drug Designation and UK CTA approval represent two important regulatory milestones for AAVB-039. With Fast Track Designation already in place, we now have a suite of incentives that can accelerate development and deliver this potentially transformative therapy to patients as efficiently as possible.”

• Dr. Natalia Misciattelli, CEO of AAVantgarde Bio.

About the Regulatory Pathway

The FDA’s Orphan Drug Designation program supports the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S.
Key benefits include:

• Tax credits for qualified clinical testing
• Waived FDA application fees
• Seven years of U.S. market exclusivity upon approval

AAVB-039 holds both IND clearance in the U.S. and CTA approval in the UK, positioning it for international clinical progress.

About Stargardt Disease

Stargardt disease is the most common inherited form of macular degeneration and a leading cause of vision loss in children and young adults.

• Typically autosomal recessive, caused by mutations in the ABCA4 gene
• Leads to toxic retinoid byproduct accumulation in the retina
• Affects 60,000–75,000 individuals across the U.S. and EU
• No approved treatments currently exist

By restoring ABCA4 function, AAVB-039 aims to slow or halt disease progression and preserve vision.