AAVantgarde gets fast track status for Stargardt gene therapy

The U.S. FDA has cleared the IND application and granted fast track designation for AAVantgarde Bio’s AAVB-039, the company’s gene therapy program for Stargardt disease secondary to biallelic mutation in ABCA4.

The most common form of inherited juvenile macular degeneration, Stargardt is caused by mutations in the ABCA4 gene. The condition leads to the progressive loss of central vision.

AAVantgarde’s AAVB-039 addresses the underlying genetic cause of the disease by providing the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations. The AAV gene therapy is currently being evaluated in the phase 1/2 CELESTE clinical trial, which is assessing safety, tolerability, and preliminary efficacy in patients with Stargardt disease.

AAVantgarde is not the only company going after Stargardt, a disease that has mostly remained elusive to genetic medicines due to the large size of the ABCA4 gene. 

Back in December 2024, Barcelona-based SpliceBio’s SB-007 became the first dual AAV gene therapy cleared by the FDA to enter clinical development for Stargardt disease. In February 2025, Ocugen aligned with the FDA on a plan to move forward with a phase 2/3 pivotal confirmatory clinical trial for the company’ modifier gene therapy in Stargardt disease, representing the first time this therapeutic approach has been explored in clinical trials for the genetic eye disorder.