Key Takeaways

  • AAVantgarde Bio has completed recruitment of approximately 150 patients in the global STELLA natural history study for Stargardt disease
  • The study will generate longitudinal data to better understand disease progression and support development of the gene therapy candidate AAVB-039
  • Insights from STELLA are expected to guide future clinical strategies and strengthen AAVantgarde’s pipeline in inherited retinal diseases

AAVantgarde Bio announced the completion of patient recruitment for its STELLA natural history study.

STELLA, a multicenter, prospective observational study, is designed to enhance understanding of the progression of Stargardt disease. The study has enrolled approximately 150 participants across multiple global clinical sites, underscoring strong engagement from both the medical community and patient population.

The completion of enrollment marks a key milestone for the company as it works to build a comprehensive dataset to support the development of AAVB-039, its investigational gene therapy for Stargardt disease.

“The successful completion of recruitment for the STELLA study represents a meaningful step forward in building a comprehensive understanding of Stargardt disease,” said Jayashree Sahni, Chief Medical Officer of AAVantgarde. “Natural history studies like STELLA are critical in shaping the development of effective gene therapies, and we are encouraged by the strong collaboration across the global clinical and patient communities. The data generated will play a vital role in informing the future development of AAVB-039 and advancing our mission to address significant unmet needs in inherited retinal diseases.”

The STELLA study is designed to collect longitudinal clinical, functional, and biomarker data from patients with Stargardt disease caused by biallelic mutations in the ABCA4 gene (STGD1). By evaluating prognostic factors and disease progression over time, the study aims to better characterize the patient population and inform the design of future clinical trials, supporting the development of new therapeutic approaches.

AAVantgarde is developing a pipeline of gene therapies targeting inherited retinal diseases, utilizing proprietary adeno-associated viral (AAV) vector platforms designed for large gene delivery. Its lead programs include LUCE-1, aimed at treating retinitis pigmentosa associated with Usher syndrome type 1B, and CELESTE, focused on Stargardt disease. Both programs are currently in Phase 1/2 clinical trials.