The Californer - New Preclinical Data Demonstrate Potential of Capsida's IV Gene Therapy for STXBP1-DEE to Correct all Disease Manifestations

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THOUSAND OAKS, Calif., Dec. 6, 2024 ~ Capsida Biotherapeutics, a leading biotechnology company, has announced promising new preclinical data on their first-in-class gene therapy candidate, CAP-002. This next-generation therapy is specifically designed to treat developmental and epileptic encephalopathy (DEE) caused by mutations in the syntaxin-binding protein 1 (STXBP1) gene.

The new data, gathered from non-human primates (NHPs) and human cells, demonstrate the potential of CAP-002 to significantly improve symptoms associated with STXBP1-DEE. These symptoms include seizures, motor abnormalities, and developmental/intellectual disabilities. The therapy is expected to enter clinical trials in the first half of 2025.

Capsida recently completed a pre-Investigational New Drug (IND) meeting with the U.S. Food and Drug Administration (FDA), where they received Orphan Drug Designation for CAP-002. This designation is granted to therapies that show promise in treating rare diseases with no approved treatments.

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In vitro studies using diseased human neurons showed that CAP-002 was able to restore STXBP1 protein levels to normal and fully correct neuronal network activity. In NHPs, IV administration of CAP-002 resulted in dose-dependent distribution and expression throughout the brain while minimizing effects on off-target tissues such as the liver and dorsal root ganglia (DRGs). These findings are consistent with previous mouse studies that have shown the potential for CAP-002 to treat and even fully correct STXBP1-DEE through gene supplementation.

Furthermore, the therapy was found to be safe and well-tolerated in NHPs, with significantly lower targeting to the liver and DRGs compared to AAV9 – a commonly used viral vector for gene therapy. No adverse histopathological findings were observed.

Peter Anastasiou, Chief Executive Officer of Capsida Biotherapeutics, emphasized the impact of these findings on patients and their families. "STXBP1-DEE is a life-altering condition with no approved treatments," he stated. "These new data demonstrate the potential of CAP-002 to safely address all aspects of the disease, and we are excited to move forward with clinical trials in 2025."

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The data will be presented at the American Epilepsy Society (AES) 2024 Annual Meeting in Los Angeles, CA. The oral presentation, titled "Systemic Gene Therapy with Engineered AAV Demonstrates Preclinical Efficacy and Safety Supporting a Disease-Modifying Treatment for STXBP1 Developmental and Epileptic Encephalopathy," will be given by Dr. Allison Knoll, Director of Preclinical Research at Capsida. The poster presentation will also be given by Dr. Knoll and will feature the same title.

The presentations will take place on Saturday, December 7, 2024, during the Translational Research session and Poster Session 1, respectively. Abstracts can be found on the AES website at www.aesnet.org.

Capsida Biotherapeutics is dedicated to developing innovative gene therapies for rare diseases like STXBP1-DEE. With promising preclinical data and FDA support, CAP-002 has the potential to provide much-needed relief for patients suffering from this debilitating condition.

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