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EDIT-301 · 2 trials · 5 indications
| Arm | Type | Description |
|---|---|---|
| EDIT-301 | EXPERIMENTAL | EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion. |
| Name | Type | Description |
|---|---|---|
| EDIT-301 | GENETIC | Administered by intravenous infusion after myeloablative conditioning with busulfan. |
Key Inclusion Criteria: Diagnosis of Transfusion Dependent B-Thalassemia as defined by: * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE) based on historical data in medical records, and * History of at least 100 mL/kg/year or 1...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
EDIT-301 is an investigational gene therapy being developed for transfusion dependent beta thalassemia and sickle cell disease. It is designed to modify a patient's own hematopoietic stem cells to address the underlying genetic cause of these hemoglobin disorders. The therapy is currently in Phase 1 clinical development.
EDIT-301 uses the gene-editing enzyme AsCas12a to make a specific edit in a patient's hematopoietic stem cells. By targeting this enzyme to a particular genetic region, the therapy aims to increase fetal hemoglobin production, which can compensate for defective adult hemoglobin in sickle cell disease and beta thalassemia.
EDIT-301 is being developed by Editas Medicine, Inc., a biotechnology company traded on the NASDAQ under the ticker symbol EDIT. The company is conducting clinical trials to evaluate the safety and efficacy of this gene therapy in patients with severe sickle cell disease and transfusion-dependent beta thalassemia.
EDIT-301 is in Phase 1 clinical development. It is an investigational therapy and has not been approved by regulatory authorities. The drug has received FDA designations including orphan drug, rare pediatric disease, and regenerative medicine advanced therapy (RMAT) designations.
EDIT-301 is being studied in two Phase 1 clinical trials. The RUBY trial (NCT04853576) evaluates the therapy in patients with severe sickle cell disease, while a second trial (NCT05444894) is studying it in adults with transfusion-dependent beta thalassemia. Both trials are active but not recruiting participants.
EDIT-301 is a gene editing therapy specifically designed for sickle cell disease and beta thalassemia. It uses the AsCas12a enzyme to edit a patient's own stem cells ex vivo. The therapy is distinct from other gene therapies in its use of this particular enzyme and its approach to reactivating fetal hemoglobin.