Capsida Biotherapeutics Presents New Preclinical Evidence Indicating Novel First-in-Class IV-Administered Gene Therapy Effectively Treats Genetic Epilepsy Due to STXBP1 Mutations
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星期二, 五月 7, 2024
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THOUSAND OAKS, Calif., May 7, 2024 /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.
Key Points:
- Gene therapy for genetic epilepsy due to STXBP1 mutations has not been previously possible because earlier generation adeno-associated viruses (AAVs) or wild-type AAVs could not achieve the level of widespread neuronal transduction required to modify the disease.
- CAP-002 is a first-in-class next-generation intravenous (IV)-administered gene therapy that achieves brain-wide neuronal expression while simultaneously detargeting the liver.
- Capsida's wholly owned program is currently in IND-enabling studies and is expected to enter the clinic in the first half of 2025.
- This level of gene expression raises neuronal STXBP1 protein to levels comparable to those that reversed disease phenotype in the mouse model.