FDA grants Fast Track Designation to AAVantgarde Bio for AAVB-039

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The US Food and Drug Administration (FDA) has granted Fast Track Designation to AAVantgarde Bio for AAVB-039, the company’s gene therapy program for Stargardt disease secondary to biallelic mutation in ABCA4.

Recently, the Investigational New Drug (IND) application for AAVB-039 was cleared to proceed by the FDA. 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.

Natalia Misciattelli, PhD, CEO of AAVantgarde, commented on the FDA decision in a press release from the company.

“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. This designation represents an important milestone for our program and enables us to accelerate development efforts as we work to bring a potentially transformative therapy to patients as quickly as possible.”

AAVB-039 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. Additionally, AAVantgarde is conducting STELLA, a prospective natural history study across the US, Europe, and the UK. STELLA has informed the clinical trial design of CELESTE.

AAVantgarde also has its first clinical-stage program, AAVB-081, for the treatment of retinitis pigmentosa in phase 1/2 development. AAVB-081 represents the first-ever dual AAV gene therapy tested clinically in an ocular indication.

References:

1. AAVantgarde Bio Announces FDA Fast Track Designation for AAVB-039 for the Treatment of Stargardt Disease. Published August 12, 2025. Accessed August 12, 2025. https://www.globenewswire.com/news-release/2025/08/12/3131528/0/en/AAVantgarde-Bio-Announces-FDA-Fast-Track-Designation-for-AAVB-039-for-the-Treatment-of-Stargardt-Disease.html