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CTX001 · 6 trials · 10 indications
| Arm | Type | Description |
|---|---|---|
| CTX001 | EXPERIMENTAL | CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive a single infusion of CTX001 through a central venous catheter. |
| Name | Type | Description |
|---|---|---|
| CTX001 | BIOLOGICAL | Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan |
Key Inclusion Criteria: * Participants with TDT and SCD: * Eligible for autologous stem cell transplant as per investigator's judgment. * Participants with TDT: * Diagnosis of TDT as defined by: * Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hem...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Agios Pharmaceuticals, Inc. | AGIO | 5 | PHASE3 | Mitapivat |
| Bristol-Myers Squibb Company | BMY | 6 | PHASE3 | Luspatercept |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 2 | PHASE3 | Etavopivat A, Etavopivat B, Etavopivat C |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE2 | REGN7999 |
| ICON Plc | ICLR | 1 | PHASE2 | SP-420 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
CTX001 is an investigational therapy being developed for sickle cell disease and transfusion-dependent beta-thalassemia. It is a gene-edited cell therapy that targets the HBB gene. The drug is currently in Phase 3 clinical development for these rare blood disorders.
CTX001 targets the HBB gene, which encodes the beta-globin subunit of hemoglobin. By editing this gene, the therapy aims to address the underlying genetic cause of sickle cell disease and beta-thalassemia. The drug is designed as a one-time treatment for these inherited blood disorders.
CTX001 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. The company is conducting Phase 3 clinical trials to evaluate the safety and efficacy of this investigational therapy for sickle cell disease and beta-thalassemia.
CTX001 is in Phase 3 clinical development. It is an investigational therapy and has not been approved by regulatory authorities. The drug is being studied for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia in multiple ongoing clinical trials.
CTX001 is being evaluated in several clinical trials, including NCT04208529, a long-term follow-up study in participants who received the drug, and NCT05356195, a Phase 3 study in pediatric participants with transfusion-dependent beta-thalassemia. NCT05477563 is a Phase 3 trial in participants with beta-thalassemia and severe sickle cell disease.
CTX001 is also known by the nonproprietary name exagamglogene autotemcel. It is an investigational gene-edited cell therapy being developed by Vertex Pharmaceuticals for sickle cell disease and transfusion-dependent beta-thalassemia. The drug is currently in Phase 3 clinical trials.