AAVantgarde’s AAVB-039 Gene Therapy for Stargardt Disease Receives Orphan Drug Designation from FDA

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AAVantgarde’s investigational gene therapy, AAVB-039, for the treatment of Stargardt disease. In addition, the therapy has received Clinical Trial Authorisation (CTA) from the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA), marking two key regulatory milestones in its development.

AAVB-039: Targeting the Genetic Root of Stargardt Disease

AAVB-039 is an intra-retinal, AAV8-intein-mediated gene therapy designed to target the ABCA4 gene, which is associated with Stargardt disease. By delivering the full-length ABCA4 protein, the therapy aims to address the underlying genetic cause of the condition. According to AAVantgarde, this approach has the potential to benefit all patients with ABCA4 mutations, offering a promising solution where no approved treatments currently exist.

CEO Comments on Regulatory Progress

Dr. Natalia Misciattelli, CEO of AAVantgarde, commented on the recent regulatory developments in a company press release, stating:

“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.”

Current Clinical Development: CELESTE and STELLA Studies

AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial. The study is assessing the safety, tolerability, and preliminary efficacy of the gene therapy in patients with Stargardt disease across three dose levels. Currently, the company has not disclosed a projected timeline for trial results or public updates.

In parallel, AAVantgarde is conducting STELLA, a prospective natural history study across the United States, Europe, and the United Kingdom. Insights from the STELLA study have informed the design of the CELESTE trial. Recruitment for STELLA remains ongoing.