drugset / Trial / NCT06041620
Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells
Unknown
2 enrolled
Institute of Hematology & Blood Diseases Hospital, China
Shanghai Vitalgen BioPharma Co., Ltd. · collab
NaSingle-groupOpen-labelTreatment
Summary
This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.
Timeline
- Start
- 2023-08-31
- Primary completion
- 2025-12-31
- Completion
- 2026-06-30
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | VGB-Ex01 | Unknown | — | — |