Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DFP0R6
|
|||
| Drug Name |
RP-L401
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Infantile malignant osteopetrosis [ICD-11: LD24.10] | Phase 1 | [1] | |
| Company |
Rocket Pharmaceuticals
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | T-cell immune regulator 1 (TCIRG1) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04525352) A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene. U.S.National Institutes of Health. | |||
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