Approval Probability
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Also known as Exagamglogene autotemcel, Exa-cel
CTX001 · 6 trials · 10 indications
A VOC is a condition of SCD characterized by vaso-occlusion presenting as recurrent pain episodes. The percentage of participants who remain VOC free after achieving VF12 were data reported in the outcome measure.
Neutrophil engraftment is defined as the first day of 3 consecutive measurements of absolute neutrophil count (ANC)≥500/μL on 3 different days, within 42 days after exa-cel infusion without the use of unmodified CD34+ cells after reaching the nadir, defined as ANC \<500/µL.
Neutrophil engraftment is defined as the first day of 3 consecutive measurements of absolute neutrophil count (ANC)≥500/μL on 3 different days, without use of the unmodified CD34+ cells after reaching the nadir, defined as ANC\<500/μL. Time to neutrophil engraftment was calculated by the neutrophil engraftment date subtract exa-cel infusion date +1.
Platelet engraftment is defined as the first day of 3 consecutive measurements of unsupported (no platelet transfusions for the last 7 days) platelet ≥50,000/μL on 3 different days after Exa-cel infusion. For participants discharged early day 7 after the last platelet transfusion will be the day of platelet engraftment, as long as 3 subsequent and consecutive unsupported measurements on 3 different days are \>50,000/μL. For participants who have been discharged prior to platelet engraftment, it is recommended to collect blood every 2 to 3 days to obtain an accurate assessment of platelet engraftment. Time to platelet engraftment was defined as first of 3 consecutive measurements on 3 different days with platelet ≥50 × 109/L without a platelet transfusion for 7 consecutive days.
The transplant-related mortality is defined as death related to Busulfan and/or exa-cel infusion. The number and proportion of TRM participants who have died within 100 days, or with at least 100 days post exa-cel infusion.
The transplant-related mortality is defined as death related to Busulfan and/or exa-cel infusion. The number and proportion of TRM within 12 months will be summarized for participants who have died within 12 months, or with at least 12 months post exa-cel infusion.
| 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. |
| Exa-cel | EXPERIMENTAL | Exa-cel (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants received a single infusion of exa-cel through a central venous catheter on Day 1. |
| Name | Type | Description |
|---|---|---|
| CTX001 | BIOLOGICAL | Administered by intravenous (IV) infusion following myeloablative conditioning with busulfan |
| Exa-cel | BIOLOGICAL | Administered by 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...
Top 6 of 8 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Agios Pharmaceuticals, Inc. | AGIO | 5 | PHASE3 | Mitapivat, AG-348 |
| 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 |
| Regeneron Pharmaceuticals, Inc. | REGN | 1 | PHASE2 | REGN7999 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
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.