AAVantgarde’s AAVB-039 gene therapy, targeting the root cause of Stargardt disease, receives FDA IND clearance to start the CELESTE clinical trial in the US

AAVantgarde Bio, a clinical-stage biotech focused on gene therapies for inherited retinal diseases, has secured FDA clearance to initiate its Phase 1/2 CELESTE trial evaluating AAVB-039 for Stargardt disease.

The therapy uses AAVantgarde’s proprietary dual AAV intein platform to deliver the full-length ABCA4 protein, addressing the genetic cause of the disease and potentially benefiting all patients with ABCA4 mutations, regardless of mutation type.

Stargardt disease is the most common inherited macular degeneration and a leading cause of vision loss in children and young adults. The CELESTE trial will assess safety, tolerability, and early signs of efficacy.

Dr Natalia Misciattelli, CEO of AAVantgarde, said the FDA clearance is a pivotal step towards expanding the company’s pipeline and delivering transformative treatments for inherited retinal disorders.

Professor Alberto Auricchio, CSO and scientific founder, highlighted the platform’s ability to overcome challenges in delivering large genes like ABCA4, supported by strong preclinical data showing high transduction, protein expression, and safety in large animal models.

The CELESTE study design is informed by the ongoing STELLA natural history study, which is enrolling patients across the US, Europe, and the UK to better characterize Stargardt disease progression.

AAVantgarde’s first clinical program, AAVB-081 for Usher syndrome type 1B, also employs the dual AAV platform and is showing encouraging early safety and efficacy signals.

Together, these programs reinforce AAVantgarde’s commitment to advancing innovative gene therapies for severe, currently untreatable genetic eye diseases.