Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RP-L201 · 1 trial · 1 indication
Evaluation of safety associated with treatment with RP-L201
, as determined by the proportion of subjects alive at least 1-year post investigational product infusion without allogeneic HSCT and alive at age 2 (24 months) without allogeneic HSCT for subjects less than 1 year of age at study enrollment.
Evaluation of safety associated with treatment with RP-L201
| Arm | Type | Description |
|---|---|---|
| RP-L201 | EXPERIMENTAL | RP-L201 is a gene therapy product containing autologous genetically modified CD34+ hematopoietic cells transduced with Chim-CD18-WPRE lentiviral vector administered as a single intravenous infusion |
| Name | Type | Description |
|---|---|---|
| RP-L201 | BIOLOGICAL | CD34+ enriched hematopoietic stem cells from subjects with severe LAD-I transduced ex vivo with lentiviral vector carrying the ITGB2 gene, Chim-CD18-WPRE. |
Inclusion Criteria: * A confirmed diagnosis of severe LAD-I as demonstrated by flow cytometry indicating CD18 expression on \<2% neutrophils (polymorphonuclear neutrophils (PMNs)). Subjects in which CD18+ PMNs are \>2% will be considered eligible with \<2% CD11a or CD11b expressing PMNs and if ther...
RP-L201 is an investigational gene therapy being developed for Leukocyte Adhesion Defect Type I, a rare inherited disorder that affects the immune system. It is designed to address the underlying genetic cause of the condition. The therapy is currently in clinical development and has not been approved by the FDA.
RP-L201 is being developed by Rocket Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker RCKT. The company is focused on developing gene therapies for rare and debilitating disorders, and RP-L201 is one of its investigational candidates for Leukocyte Adhesion Defect Type I.
RP-L201 is in Phase 1 clinical development. A Phase 1 trial has been completed, and the therapy remains investigational. It has not received FDA approval and is still being studied for safety and efficacy in patients with Leukocyte Adhesion Defect Type I.
RP-L201 has been studied in a completed Phase 1 clinical trial with the identifier NCT03812263. The trial evaluated the safety and efficacy of RP-L201 in subjects with Leukocyte Adhesion Deficiency I and enrolled 9 participants across the United States, Spain, and the United Kingdom.
RP-L201 has received several FDA designations, including Regenerative Medicine Advanced Therapy (RMAT), Rare Pediatric Disease designation, Fast Track designation, and Orphan Drug designation. These designations are intended to support and expedite the development of therapies for serious or rare conditions.
RP-L201 is classified as a gene therapy, not a monoclonal antibody. It is being developed to treat Leukocyte Adhesion Defect Type I by targeting the genetic cause of the disease. The therapy is administered to patients with the condition and has been evaluated in a clinical trial.