Capsida Biotherapeutics Presents New Preclinical Evidence Indicating Novel First-in-Class IV-Administered Gene Therapy Effectively Treats Genetic Epilepsy Due to STXBP1 Mutations

Key Facts

• Dose-dependent rescue of neurological phenotypes, including epileptic seizures, motor deficits, and cognitive impairments, with long-lasting effects in adult mice Capsida poster will also show new data with its next-generation gene therapy showing significant brain-wide expression of STXBP1 protein and liver detargeting after a single IV infusion Wholly owned program is first in class and currently in IND-enabling studies to support initiation of clinical trials in first half of 2025 , /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced new preclinical data supporting the potential of Capsida's gene therapy candidate, CAP-002, to achieve levels of gene supplementation necessary to correct neurological phenotypes associated with genetic epilepsy due to syntaxin-binding protein 1 (STXBP1) mutations.
• The data show that a single IV infusion of CAP-002 results in brain-wide STXBP1 gene expression and is capable of transducing up to 70% of neurons at therapeutically relevant doses in non-human primates (NHPs).
• "These data demonstrate that our STXBP1 program effectively crosses the blood-brain barrier in NHPs following IV delivery and achieves breakthrough levels of widespread brain transduction and STXBP1 protein expression needed to achieve disease-modifying impact as demonstrated in the mouse model data," said Capsida's Chief Scientific Officer Susan Catalano, Ph.D. "Coupled with significant detargeting of the liver compared to AAV9, CAP-002 holds the promise of effectively reversing disease and does so via less-invasive IV administration.
• About CAP-002: Capsida's Program for Genetic Epilepsy Due to STXBP1 Mutations CAP-002 is a wholly owned first-in-class next-generation intravenous (IV)-administered gene therapy that achieves brain-wide neuronal expression of the syntaxin-binding protein 1 (STXBP1) protein after a single infusion, with significant liver detargeting.

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