AAVantgarde Bio has received two regulatory milestones for its investigational gene therapy AAVB-039, aimed at treating Stargardt disease, the most common inherited form of macular degeneration. The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for AAVB-039, while the UK Medicines and Healthcare products Regulatory Agency (MHRA) approved a Clinical Trial Authorisation (CTA).

AAVB-039 is designed to address the underlying cause of Stargardt disease by delivering the full-length ABCA4 protein, offering potential benefit to all patients with ABCA4 mutations. The therapy is currently being tested in the Phase 1/2 CELESTE clinical trial, which evaluates safety, tolerability, and preliminary efficacy across three dose levels. Recruitment continues in the ongoing STELLA observational study.

Dr. Natalia Misciattelli, chief executive of AAVantgarde, said: “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. 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.”

Orphan Drug Designation in the U.S. supports the development of treatments for rare diseases affecting fewer than 200,000 people, offering benefits such as tax credits for qualified clinical testing, waiving of certain FDA application fees, and, if approved, seven years of U.S. market exclusivity.

Stargardt disease primarily affects children and young adults and can lead to progressive vision loss. Current management focuses on symptom control, with no approved therapies targeting the underlying genetic cause. AAVB-039, by restoring ABCA4 protein function, aims to preserve or improve retinal health and slow disease progression.

The CELESTE and STELLA trials are part of AAVantgarde’s broader development programme for inherited retinal diseases. With the regulatory designations in place, the company is positioned to advance AAVB-039 through clinical development with support from both U.S. and European regulatory incentives.