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Strimvelis
- Development stage
- Phase 2
- Lead developer
- GSK
- Modality
- Gene Silencing → Gene Therapies, Stem Cell Therapies → Cell Therapies, Gene Editing → Gene Therapies, Gene Addition/Replacement → Gene Therapies
- Administration
- Intravenous
01
Overview
Strimvelis is an ex vivo gammaretroviral vector-based gene therapy indicated for the treatment of patients with severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) for whom no suitable HLA-matched related stem cell donor is available. The therapy consists of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) that have been transduced with a retroviral vector carrying a functional copy of the human adenosine deaminase (ADA) gene. By reintroducing these modified cells into the patient, the therapy restores the production of the ADA enzyme, which is essential for the development and maintenance of a functional immune system. Strimvelis was the first ex vivo gene therapy to receive regulatory approval in the European Union, marking a significant milestone in genetic medicine.
- Brand names
- Strimvelis
- Other names
- ADA-SCID gene therapyautologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA sequence
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