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Also known as Exagamglogene autotemcel, CTX001
Exa-cel · 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 11 of 12 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 6 | PHASE3 | Etavopivat |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab, OTQ923, ITU512 |
| Agios Pharmaceuticals, Inc. | AGIO | 3 | PHASE3 | Mitapivat, Tebapivat |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, Rilzabrutinib |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Travere Therapeutics, Inc. | TVTX | 1 | PHASE3 | L-Citrulline |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470 |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
Exa-cel is being developed for the treatment of sickle cell disease, a hematological disorder. It is a cell therapy studied in patients with severe sickle cell disease. Development is being led by Vertex Pharmaceuticals Incorporated under ticker VRTX.
Exa-cel is being developed by Vertex Pharmaceuticals Incorporated, which trades under the ticker VRTX. The company is the sponsor of the clinical program evaluating the cell therapy in sickle cell disease.
Exa-cel is in Phase 2 clinical development for sickle cell disease. It is an investigational cell therapy and has not been established as approved. The program includes one completed Phase 2 trial with 63 enrolled participants.
Exa-cel has been studied in the Phase 2 trial NCT03745287, titled A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease. The trial is completed, enrolled 63 participants aged 12 years and older, and ran in the United States, Belgium, Canada, France, Germany, Italy, and the United Kingdom.
Exa-cel is an investigational cell therapy in Phase 2 clinical development for sickle cell disease and is not established as approved. The program has received a PRIORITY_REVIEW FDA designation. Its single Phase 2 trial, NCT03745287, is completed.