Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D6DO9A
|
|||
| Drug Name |
RP-L301
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Pyruvate kinase deficiency [ICD-11: 5C53.00] | Phase 1 | [1] | |
| Company |
Rocket Pharmaceuticals
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Pyruvate kinase PKLR (PKLR) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04105166) Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKD. U.S.National Institutes of Health. | |||
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