AAVantgarde Bio, a clinical-stage biotechnology company focused on next-generation gene therapies for inherited retinal diseases, has received FDA Investigational New Drug (IND) clearance for AAVB-039, its gene therapy program for Stargardt disease. This pivotal clearance enables AAVantgarde to initiate CELESTE, a first-in-human Phase 1/2 clinical trial in the United States. The trial will evaluate the safety, tolerability, and initial efficacy of AAVB-039 in patients with Stargardt disease, the most common inherited form of macular degeneration and a leading cause of vision loss in children and young adults.

AAVB-039 is designed to address the fundamental genetic cause of Stargardt disease by delivering the full-length ABCA4 protein. This is crucial because the ABCA4 gene is too large to be packaged within a standard AAV vector, a challenge that AAVantgarde overcomes using its proprietary dual AAV intein platform. This innovative platform has demonstrated impressive preclinical data, showing high transduction, robust protein expression, and long-term safety across multiple large animal models, suggesting a strong potential to benefit all patients with ABCA4 mutations, regardless of the specific mutation.

The design of the CELESTE trial is informed by the insights gathered from STELLA, a prospective natural history study currently enrolling patients across the U.S., Europe, and the UK. This ongoing study helps to further characterize Stargardt disease and refine the clinical trial approach.

This FDA clearance represents a significant milestone for AAVantgarde, marking the advancement of their second therapeutic candidate into clinical development. Their first clinical-stage program, AAVB-081, is already in Phase 1/2 development for retinitis pigmentosa caused by Usher syndrome type 1B, representing the first-ever dual AAV gene therapy tested clinically in an ocular indication with early promising safety and efficacy signals. Together, these programs underscore AAVantgarde's commitment to developing scientifically rigorous, best-in-class genetic medicines for currently untreatable inherited retinal diseases, aiming to redefine what's possible for patients with severe genetic disorders.