AAVantgarde Bio has received clearance from the US Food and Drug Administration (FDA) to begin a phase 1/2 clinical trial for AAVB-039, a gene therapy for Stargardt disease. The company announced in a press release it will launch the CELESTE trial in the United States to evaluate safety, tolerability, and initial efficacy.

AAVB-039 uses AAVantgarde’s proprietary dual AAV intein platform to deliver the full-length ABCA4 protein, addressing the genetic cause of Stargardt disease. The inherited retinal disorder, which affects 60,000 to 75,000 people in the United States and Europe, leads to vision loss and has no approved treatments.

CELESTE builds on insights from the ongoing STELLA natural history study. Preclinical studies of AAVB-039 demonstrated high levels of gene expression and long-term safety in large animal models.

This marks AAVantgarde’s second gene therapy program to reach clinical testing. Its first candidate, AAVB-081, is in development for retinitis pigmentosa due to Usher syndrome type 1B. The company said early data from that program show encouraging safety and efficacy signals.