AAVantgarde Receives FDA Clearance to Advance AAVB-039 for Stargardt Disease into Phase 1/2 Trial
AAVantgarde Bio has announced that the US 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. With this clearance, AAVantgarde is now initiating CELESTE, a first-in-human phase 1/2 clinical trial in the United States designed to evaluate the safety, tolerability, and initial efficacy of AAVB-039 in patients with Stargardt disease.
About AAVB-039 and Its Mechanism
AAVB-039 is designed to address the underlying genetic cause of Stargardt disease by restoring the full-length ABCA4 protein, offering the potential to benefit all patients with ABCA4 mutations. The therapy leverages AAVantgarde’s proprietary dual AAV intein platform, which enables the delivery of large genes that exceed the packaging capacity of traditional single AAV vectors.
Executive Insights on Advancing Clinical Programs
“This FDA clearance marks a pivotal milestone for AAVantgarde and our Stargardt program,” said Natalia Misciattelli, PhD, 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.”
Supporting Studies and Pipeline Development
In parallel with CELESTE, AAVantgarde is conducting STELLA, a prospective natural history study at selected centers of excellence across the United States, Europe, and the UK. This study has helped inform the clinical trial design of CELESTE and continues to deepen the understanding of Stargardt disease.
The company also highlighted progress on its first clinical-stage program, AAVB-081, which is in phase 1/2 development for retinitis pigmentosa caused by Usher syndrome type 1B. According to AAVantgarde, this represents the first dual AAV gene therapy tested clinically in an ocular indication, with early data demonstrating good preliminary safety and encouraging signs of efficacy.
