AAVantgarde Receives FDA Clearance to Progress Stargardt Disease Asset Into Phase 1/2 Clinical Trial  

AAVantgarde Bio recently announced the US 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 US, to evaluate the safety, tolerability, and initial efficacy 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,” said Dr. Natalia Misciattelli, Chief Executive Officer of AAVantgarde. “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 AAV-based, 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.”

“The IND clearance for AAVB-039 is a testament to the scientific foundation and translational potential of our proprietary dual AAV intein platform,” said Professor Alberto Auricchio, Chief Scientific Officer and Scientific Founder of AAVantgarde. “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 offers a potential therapeutic that addresses the genetic root cause for patients with Stargardt disease.”

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 efficacy. Together, these programs reflect AAVantgarde’s commitment to advancing scientifically rigorous, best-in-class genetic medicines for currently untreatable diseases.