AAVantgarde Bio Gains FDA IND Clearance for Dual AAV Gene Therapy AAVB-039 for Stargardt Disease

MILAN – July 15, 2025 – AAVantgarde Bio, a clinical-stage biotechnology company focused on next-generation gene therapies for inherited retinal diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for AAVB-039. This significant milestone enables the company to initiate ‘CELESTE’, a first-in-human Phase 1/2 clinical trial in the United States, to evaluate the safety, tolerability, and initial efficacy of AAVB-039 in patients with Stargardt disease. Stargardt disease is 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 underlying genetic cause of Stargardt disease by restoring the full-length ABCA4 protein. This is crucial as the ABCA4 gene is too large to fit into a standard single AAV vector. AAVB-039 overcomes this challenge by utilizing AAVantgarde’s proprietary dual AAV intein platform, which uniquely enables the delivery of large genes by splitting the gene across two AAV vectors, then reassembling the functional protein inside the cell.

“This FDA clearance marks a pivotal milestone for AAVantgarde and our Stargardt program,” said Dr. Natalia Misciattelli, CEO of AAVantgarde. She emphasized that AAVB-039 is the company’s second therapeutic candidate advancing into the clinic, reinforcing their evolution into a clinical-stage company with a growing pipeline.

Professor Alberto Auricchio, CSO and Scientific Founder of AAVantgarde, highlighted the strength of their dual AAV intein platform, noting its validation through rigorous preclinical studies demonstrating high transduction, expression, and long-term safety.

In addition to CELESTE, AAVantgarde is conducting STELLA, a natural history study informing the trial design and further characterizing Stargardt disease. The company’s first clinical-stage program, AAVB-081, an AAV-based dual hybrid gene therapy, is already in Phase 1/2 development for retinitis pigmentosa caused by Usher syndrome type 1B, showing encouraging early safety and efficacy. These programs underscore AAVantgarde’s commitment to developing transformative genetic medicines for currently untreatable inherited retinal diseases.