AAVantgarde Bio received FDA clearance for its Investigational New Drug (IND) application for AAVB-039, a gene therapy for Stargardt disease. The company is initiating CELESTE, a Phase 1/2 clinical trial in the U.S., to assess the therapy’s safety, tolerability, and efficacy. Stargardt disease is the most common inherited macular degeneration and currently lacks approved treatments.
This clinical trial initiation is crucial because it represents a potential turning point for Stargardt disease patients, who currently face progressive vision loss without effective treatment options. AAVB-039’s potential to address the root cause of the disease by delivering the full ABCA4 protein offers a significant advancement over current management strategies. The trial also highlights the growing potential of gene therapies to address previously untreatable inherited retinal diseases. This progress could stimulate further research and development in the field, leading to more effective treatments for a range of inherited conditions.
AAVB-039 utilizes AAVantgarde’s dual AAV intein platform, which allows for the delivery of large genes. This platform has demonstrated promising preclinical results, including high transduction, protein expression, and safety in animal models. The CELESTE trial design is informed by the ongoing STELLA natural history study, which is gathering data on Stargardt disease progression. AAVantgarde also has another gene therapy, AAVB-081, in Phase 1/2 development for retinitis pigmentosa caused by Usher syndrome type 1B.
The FDA clearance and subsequent trial launch represent a significant step forward for AAVantgarde. Positive clinical trial data could validate the company’s dual AAV intein platform and pave the way for potential regulatory approval of AAVB-039. This success could establish AAVantgarde as a leader in gene therapy for inherited retinal diseases and encourage further investment in the development of new treatments for these conditions. The outcome of the CELESTE trial will be critical for both the company’s future and the future of Stargardt disease treatment.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
