- AAVB-039 delivers the full-length ABCA4 -protein, addressing the root cause of the disease and enabling treatment of all patients, independent of mutation
- CELESTE is informed by the STELLA prospective natural history study, which is currently enrolling patients in the United States and Europe
- AAVB-039 uses AAVantgarde’s proprietary dual AAV intein platform. The platform has achieved unprecedented preclinical data demonstrating high transduction, protein expression, and safety in multiple large animal models
MILAN, July 15, 2025 — AAVantgarde Bio (AAVantgarde), a clinical-stage biotechnology company developing 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, the company’s gene therapy program for Stargardt disease.
AAVantgarde is now initiating ‘CELESTE’, a first-in-human Phase 1/2 clinical trial in the United States, to evaluate the safety, tolerability, and initial eicacy of AAVB-039 in patients with Stargardt disease. This is the most common inherited form of macular degeneration and a leading cause of vision loss in children and young adults.
AAVB-039 aims to address the underlying genetic cause of the disease by restoring the full length ABCA4 protein and has the potential to benefit all patients with ABCA4 mutations. AAVB-039 utilizes AAVantgarde’s proprietary dual AAV intein platform, which enables the delivery of large genes.
This FDA clearance marks a pivotal milestone for AAVantgarde and our Stargardt program. With AAVB-039 now in clinical development, we are advancing our second therapeutic candidate toward addressing the urgent needs of patients with inherited retinal diseases. Moreover, the news reinforces our evolution from pioneering dual AAVbased, retina-targeting therapies into a clinical-stage company with a growing pipeline of candidates. Looking ahead, we remain focused on generating meaningful clinical data and building a portfolio of transformative treatments that can redefine what’s possible for patients living with severe genetic disorders.
Dr. Natalia Misciattelli
Chief Executive Officer of AAVantgarde
The IND clearance for AAVB-039 is a testament to the scientific foundation and translational potential of our proprietary dual AAV intein platform. Delivering large genes like ABCA4 has been a challenge in the field. Our approach, validated by rigorous preclinical studies showing quantified high transduction, expression, and long-term safety in multiple relevant models oers a potential therapeutic that addresses the genetic root cause for patients with Stargardt disease.
Professor Alberto Auricchio
Chief Scientific Officer and Scientific Founder of AAVantgarde
Alongside CELESTE, AAVantgarde is conducting STELLA, a prospective natural history study at selected centers of excellence across the U.S., Europe and the UK. The study has informed the clinical trial design and continues to better characterize Stargardt disease. Additionally, the company’s first clinical-stage program, AAVB-081, is in Phase 1/2 development for retinitis pigmentosa caused by Usher syndrome type 1B and represents the first-ever dual AAV gene therapy tested clinically in an ocular indication. Early clinical data demonstrates good preliminary safety and encouraging signs of eicacy. Together,these programs reflect AAVantgarde’s commitment to advancing scientifically rigorous, best-in-class genetic medicines for currently untreatable diseases.
Contact
Magda Blanco – Head of Corporate Development
Email: info@aavantgarde.com