AAVantgarde Announces FDA Orphan Drug Designation and UK CTA approval for AAVB-039 for the Treatment of Stargardt Disease

• AAVB-039 addresses the root cause of the disease, benefitting patients with any ABCA4 mutation
• AAVB-039 is currently being assessed in the CELESTE interventional clinical trial and we continue recruiting in our STELLA observational study

MILAN, Oct. 02, 2025 (GLOBE NEWSWIRE) -- 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.

About AAVantgarde Bio

AAVantgarde Bio is a clinical stage, biotechnology company advancing best-in-class therapies for patients with inherited retinal diseases (IRDs). The company’s lead programs target Stargardt disease and retinitis pigmentosa due to Usher syndrome type 1B, two severe, IRDs with no approved treatments. AAVB-039 and AAVB-081 are investigational, dual AAV gene therapies designed to address the root genetic causes of these diseases. With a strong foundation in translational science and a commitment to clinical excellence, AAVantgarde is working to bring transformative therapies to patients. For more information, please visit: www.aavantgarde.com

About Stargardt Disease

Stargardt disease is the most common form of inherited macular degeneration. Inherited in most cases as autosomal recessive, Stargardt disease is caused by mutations in the ABCA4 gene, leading to the accumulation of toxic retinoid byproducts in the retina and progressive vision loss. The ABCA4 gene is 6.8 kilobases in length, too large to be packaged within a standard, single AAV vector. Stargardt disease affects an estimated 60,000 to 75,000 individuals across the U.S. and E.U and currently there are no approved treatments.

Contact:
Magda Blanco – Head of Corporate Development
Email: info@aavantgarde.com

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