AAVantgarde Bio receives FDA fast track designation for AAVB-039 in stargardt disease

The therapy delivers the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations

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

The FDA has also cleared the Investigational New Drug (IND) application for AAVB-039. The therapy delivers the full-length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations.

“We are thrilled that the FDA has granted Fast Track Designation for AAVB-039, as it underscores the urgent unmet need and important potential of our program due to the serious nature of challenges faced by patients living with Stargardt disease,” said Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde.

AAVB-039 is currently being evaluated in the Phase 1/2 CELESTE clinical trial to assess safety, tolerability, and preliminary efficacy in Stargardt disease patients. The FDA’s Fast Track program is intended to speed the development and review of treatments for serious conditions that address unmet medical needs.