Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RP-L301 · 1 trial · 1 indication
The number of participants with treatment-related adverse events as assessed by United States (US) National Cancer Institute (NCI) v.5.0.
| Arm | Type | Description |
|---|---|---|
| RP-L301 | EXPERIMENTAL | RP-L301 is a gene therapy product containing autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene |
| Name | Type | Description |
|---|---|---|
| RP-L301 | BIOLOGICAL | Autologous genetically modified CD34+ hematopoietic stem cells containing the corrected PKD gene |
Inclusion Criteria: * PKD diagnosis with a confirmed PKLR mutation. * Adult Cohort ≥18 years old and \<50 years for the initial 2 patients enrolled; Pediatric Cohort ≥8-17 years for the next 2-3 patients. * History of severe, transfusion-dependent anemia, defined as: 1. At least 6 red blood cell...
RP-L301 is an investigational gene therapy being studied for the treatment of Pyruvate Kinase Deficiency, a rare inherited disorder that affects red blood cells. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
RP-L301 is being developed by Rocket Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol RCKT. The company is focused on developing gene therapies for rare diseases.
RP-L301 is in Phase 1 clinical development. A Phase 1 clinical trial for this investigational gene therapy has been completed. It is not yet approved and remains in the clinical research stage.
RP-L301 has been studied in one clinical trial, registered as NCT04105166, titled "Gene Therapy for Pyruvate Kinase Deficiency (PKD)." This Phase 1 trial was completed and enrolled 4 participants with Pyruvate Kinase Deficiency in the United States and Spain.
RP-L301 is not FDA approved. It is an investigational gene therapy that has completed a Phase 1 clinical trial. The drug remains in clinical development and has not received marketing authorization from the FDA or any other regulatory agency.