Systemic AAV Gene Therapy Using a CNS-targeted Engineered Capsid Significantly Increases GCase Activity to Support the Potential Treatment of PD-GBA (S37.007)
Capsida has developed a next-generation gene therapy candidate (CAP-003) for administration as a single intravenous (IV) infusion to Parkinson’s disease patients with GBA1 mutations (PD-GBA).
Authors
- P. Denis
- Brandon Wheeler
- Nicholas C. Flytzanis (ORCID: https://orcid.org/0000-0002-7921-9392)
- Nick Goeden
- Shelby Thomas
- Lubov Grigoryeva (ORCID: https://orcid.org/0000-0002-9083-8805)
- Swati Tole
- Matt Flynn (ORCID: https://orcid.org/0000-0003-4566-9464)
- H. Acharya
- Kimberly A. McDowell (ORCID: https://orcid.org/0009-0000-5412-105X)
- A. Armendariz
- Reed Ressler
- Pasha Tchourilov
Institutions
- Nanotherapeutics (United States) (US)
- Capsida Biotherapeutics (United States)
Publication Details
- Journal
- Neurology
- Published
- 2025-04-07
- DOI
- https://doi.org/10.1212/wnl.0000000000212169
- Citations
- 2
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 3.63