Capsida Receives FDA Approval to Start Clinical Trial for Gene Therapy for STXBP1-Related Disorders

Written By Russ Novy

Capsida Biotherapeutics has announced that it received approval from the FDA for its Investigational New Drug (IND) Application for its gene therapy CAP-002, developed for STXBP1-RD. CAP-002 uses Capsida’s engineered AAV capsid that crosses the blood–brain barrier while detargeting the liver and sensory nerves, enabling widespread neuronal delivery after an IV infusion. This is more than industry news -- it's a fresh dose of hope for every family living with STXBP1, and an affirmation that STXBP1 research is moving forward in a truly meaningful way.

This clinical trial is the result of multiple years of research starting in Dr. Viviana Gradinaru's lab at California Institute of Technology, and a strong collaboration with Dr. Mingshan Xue's lab at Baylor College of Medicine.

Congratulations to the Capsida team on this significant milestone!

This first genetic therapy trial for STXBP1 disorders is a significant step in our mission to advance therapies and one day find a cure for STXBP1 disorders. As outlined in the foundation's STXBP1 Fast Forward strategic plan, we must continue to progress all of our therapy strategies while strengthening our existing biopharma relationships and building new ones. 

The Capsida trial marks the first instance of testing a treatment aimed at restoring the STXBP1 protein to sufficient levels in humans, with the goal of minimizing seizures and enhancing daily functioning. The initial dosing of a patient is expected to occur in the third quarter of this year. While the study will start small and focus on safety, it signals that the years we’ve spent building patient registries, sharing data, and funding early science are paying off. Right now, there’s nothing families need to do except stay informed: join the Contact List, keep genetic reports and medical records current, and remain engaged with the STARR and ESCO natural history studies. Your support laid the groundwork that made this milestone possible. We’ll keep everyone updated through email, social channels, and our website. Together, we’re turning possibility into progress.

We encourage you to read Capsida's press release and review the other links below.

Charlene Son Rigby, President, and Mark Quinlan, Executive Director 

Additional Information

Russ Novy
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