drugset / Trial / NCT05682144
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
Non-randomizedSequentialOpen-labelTreatment
Summary
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Timeline
- Start
- 2023-04-12
- Primary completion
- 2029-06-01
- Completion
- 2044-06-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Autologous Plasmablasts (B cells) | Cell therapy | 5e+07 cells/kg | — |
| Subject | Autologous Plasmablasts (B cells) | Cell therapy | 1e+08 cells/kg | — |
| Subject | Autologous Plasmablasts (B cells) | Cell therapy | 2e+08 cells/kg | — |