AAVantgarde Receives FDA Clearance to Progress Stargardt Disease Asset, AAVB-039, into Phase 1/2 Clinical Trial
AAVantgarde Bio announced that the 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 efficacy of AAVB-039 in patients with Stargardt disease.
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,” Natalia Misciattelli, PhD, Chief Executive Officer of AAVantgarde, said in a company news 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.”
Alongside CELESTE, AAVantgarde is conducting STELLA, a prospective natural history study at selected centers of excellence across the US, 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 dual AAV gene therapy tested clinically in an ocular indication, according to AAVantgarde. 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.