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Gene Therapy for Parkinson’s Disease Associated with GBA Mutations

Capsida Biotherapeutics („Capsida“) presented new IND-enabling data on its intravenously (IV) delivered gene therapy for Parkinson’s disease associated with GBA mutations (PD-GBA), CAP-003, that demonstrate its substantial therapeutic potential. Capsida’s next-generation therapy, using a proprietary engineered capsid, demonstrates best-in-class potential to effectively and safely treat PD-GBA. CAP-003 achieves unprecedented levels of GCase protein and activity while simultaneously detargeting the liver and dorsal root ganglion (DRGs).

These data were presented in a late-breaking poster presentation at the Society for Neuroscience „Neuroscience 2024“ annual meeting being held October 5-9, 2024 in Chicago, IL.

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