Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RP-L102 · 1 trial · 1 indication
Bone Marrow (BM) colony-forming cell (CFC) mitomycin-C (MMC) resistance ≥20% at 12 months post-infusion with a confirmatory result at 18 or 21 months (MMC at 10 nM concentration)
| Arm | Type | Description |
|---|---|---|
| RP-L102 | EXPERIMENTAL | RP-L102 is CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a lentiviral vector carrying the FANCA gene |
| Name | Type | Description |
|---|---|---|
| RP-L102 | BIOLOGICAL | CD34+ enriched cells from subjects with Fanconi anemia subtype A transduced ex vivo with a lentiviral vector carrying the FANCA gene |
Inclusion Criteria: 1. Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent 2. Subject of the complementation group FA-A 3. Minimum age: 1 year and a minimum weight of 8 kg 4. At least 30 CD34+ cells/μL are det...
RP-L102 is an investigational gene therapy being developed for Fanconi Anemia Complementation Group A, a rare inherited bone marrow failure disorder. It is designed to address the underlying genetic cause of the condition. The drug is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.
RP-L102 targets the FANCA gene, which is mutated in patients with Fanconi Anemia Complementation Group A. The therapy aims to deliver a functional copy of the FANCA gene to correct the genetic defect. This approach is intended to restore the body's ability to repair DNA and produce healthy blood cells.
RP-L102 is being developed by Rocket Pharmaceuticals, Inc., a biopharmaceutical company focused on rare diseases. The company's stock is traded under the ticker symbol RCKT. Rocket Pharmaceuticals is conducting clinical trials to evaluate the safety and efficacy of RP-L102 in patients with Fanconi Anemia Complementation Group A.
RP-L102 is currently in Phase 2 clinical development. It is an investigational therapy, meaning it has not received FDA approval and is still being studied in clinical trials. The ongoing Phase 2 trial is active but not recruiting participants, indicating that enrollment has been completed and the study is continuing to follow patients.
RP-L102 is being evaluated in a Phase 2 clinical trial registered as NCT04248439, titled "Gene Therapy for Fanconi Anemia, Complementation Group A." This single-center study is being conducted in the United States and has an enrollment of 5 participants. The trial is active but not recruiting, and it is open to patients aged 1 year and older of all sexes.
RP-L102 is a specific gene therapy product being developed for Fanconi Anemia Complementation Group A. It is not the same as other gene therapies that may target different complementation groups of Fanconi anemia. The ongoing clinical trial NCT04248439 specifically evaluates RP-L102 in patients with this particular genetic subtype.