AAVantgarde Bio receives IND clearance for AAVB-039

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AAVantgarde Bio recently announced the US Food and Drug Administration (FDA) has cleared its investigational new drug (IND) application for AAVB-039.¹ AAVB-039 is AAVantgarde’s gene therapy program for the treatment of Stargardt disease. AAVantgarde is a clinical-stage biotechnology company focused on developing gene therapies for the treatment of inherited retinal disease.

“This FDA clearance marks a pivotal milestone for AAVantgarde and our Stargardt program,” Natalia Misciattelli, PhD, chief executive officer of AAVantgarde, said in a press release.

“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.”

AAVB-039 uses AAVantgarde’s proprietary AAV intein platform, enabling the delivery of large genes, and aims to address the underlying genetic cause of Stargardt disease by restoring the full-length ABCA4 protein.

The CELESTE phase 1/2 clinical trial will evaluate the safety, tolerability, and initial efficacy of AAVB-039 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 a phase 1/2 development. AAVB-081 represents the first-ever dual AAV gene therapy tested clinically in an ocular indication.

“The IND clearance for AAVB-039 is a testament to the scientific foundation and translational potential of our proprietary dual AAV intein platform,” Professor Alberto Auricchio, chief scientific officer and scientific founder of AAVantgarde, said in a press release.

“Delivering large genes like ABC4 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.”

Reference:

1. AAVantgarde Receives FDA IND Clearance to Progress Stargardt Disease program AAVB-039. AAVantgarde. Published July 15, 2025. Accessed July 17, 2025. https://www.aavantgarde.com/en/news/aavantgarde-receives-fda-ind-clearance-to-progress-stargardt-disease-program-aavb-039/