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 studied for the treatment of Fanconi Anemia Complementation Group A, a rare inherited disorder that affects bone marrow function and increases the risk of cancer. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities.
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 on the Nasdaq stock exchange.
RP-L102 is currently in Phase 2 clinical development. It is an investigational therapy, meaning it has not yet been approved by the FDA or other regulatory agencies. The ongoing Phase 2 trial is active but not recruiting participants.
RP-L102 is being evaluated in a Phase 2 clinical trial with the identifier NCT04248439, titled 'Gene Therapy for Fanconi Anemia, Complementation Group A'. This single-arm, open-label study is enrolling approximately 5 participants in the United States, with a minimum age of 1 year.
No, RP-L102 is not a monoclonal antibody. Although its modality is listed as such in some databases, RP-L102 is actually a gene therapy product designed to deliver a functional copy of the FANCA gene to patients with Fanconi Anemia Complementation Group A.