Capsida Biotherapeutics Presents New Preclinical Evidence Indicating Novel First-in-Class IV-Administered Gene Therapy Effectively Treats Genetic Epilepsy Due to STXBP1 Mutations
Retrieved on:
Mardi, mai 7, 2024
Doctor of Philosophy, Brain, Traffic barrier, Mouse, Phenotype, Poster, MD, Model, Cell, Deficit spending, Associate professor, STXBP1, AAV, DSM-IV codes, Vivisection, Genetics, Disease, Patient, Liver, Baylor College of Medicine, Society, Gene, Treatment, Neurology, Seizure, Gene expression, Neuron, Annual general meeting, Texas Children's Hospital, Baylor University, Baltimore Convention Center, Vaccine
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.