Summary
Exagamglogene Autotemcel has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Beta Thalassemia would be drug repurposing rather than first-in-human development. This does not mean it is approved for Beta Thalassemia. ClinicalTrials.gov lists 4 registered trials linking Exagamglogene Autotemcel to Beta Thalassemia: 2 are currently recruiting; 1 is active or not yet recruiting; 1 has completed. The most advanced is Phase 3 (NCT04208529). It plans or enrolled 160 participants. Registration activity spans 2018 to 2022. No published literature item is linked to Exagamglogene Autotemcel and Beta Thalassemia in the OSMF database yet, so the record rests on registry entries alone. Registry entries describe intent to study, not outcomes.
The RepurpOS disease-intelligence file for Beta Thalassemia ranks 50 candidate therapeutics from Open Targets, ChEMBL, DGIdb and PubMed; only a minority carry direct clinical evidence, and this page covers one of those.
Evidence table
| Evidence tier | A · Strong |
|---|---|
| Evidence score | 37.5 (trials 17.5, literature 0.0, tier 15, approved bonus 5.0) |
| Registered trials | 4 total: 2 recruiting, 1 active / not yet recruiting, 1 completed, 0 other |
| Linked publications | 0 |
| Agent type | Drug (Small molecule) |
| Development stage (any indication) | Approved |
| Mechanism / class | Not recorded |
| Data sources | DGIdb |
| Linked via biomarker / target | BCL11A |
How the evidence score is calculated
- Each registered trial scores by status (recruiting / active / enrolling 3, completed 2.5, not yet recruiting 2, unknown 1, terminated / withdrawn / suspended 0.5) plus a phase bonus (phase 3-4 +2, phase 2 +1, phase 1 +0.5). The trial component is capped at 30.
- Each literature item scores by design (Cochrane review 5, meta-analysis 4, systematic review 3, RCT 3, clinical trial publication 2, curated reference 1.5, other PubMed record 1). The literature component is capped at 30.
- The existing evidence tier adds 15 (A / Strong), 10 (B / Moderate), 5 (C / Preliminary) or 0 (D / Anecdotal).
- Agents approved for any indication (max clinical phase 4) add 5, because an approved agent has an established safety profile that lowers the barrier to repurposing trials.
- The score ranks what has been studied, not what works. It does not read effect sizes or directions of effect.
Registered clinical trials
| NCT ID | Title | Status | Phase | Enrolment |
|---|---|---|---|---|
| NCT05356195 | Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT) 2022 | active, not recruiting | Phase 3 | 16 |
| NCT04208529 | A Long-term Follow-up Study in Participants Who Received CTX001 2021 | enrolling by invitation | Phase 3 | 160 |
| NCT05477563 | Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease 2022 | recruiting | Phase 3 | 26 |
| NCT03655678 | A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia 2018 | completed | PHASE2, PHASE3 | 59 |
Published literature
No publication is linked to this pair yet.
Mechanism and notes
No mechanism of action is recorded for this pair in the source databases.
OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target BCL11A. The tier describes how well the drug-disease link is documented, not how well the drug works.
Frequently asked questions
Is Exagamglogene Autotemcel approved for Beta Thalassemia?
Exagamglogene Autotemcel has reached the approved stage (maximum clinical phase 4) for at least one indication, so its use in Beta Thalassemia would be drug repurposing rather than first-in-human development. This does not mean it is approved for Beta Thalassemia. Approval status for the specific indication should always be confirmed with the relevant regulator and prescribing information.
Is Exagamglogene Autotemcel in clinical trials for Beta Thalassemia?
ClinicalTrials.gov lists 4 registered trials linking Exagamglogene Autotemcel to Beta Thalassemia: 2 are currently recruiting; 1 is active or not yet recruiting; 1 has completed. The most advanced is Phase 3 (NCT04208529). It plans or enrolled 160 participants. Registration activity spans 2018 to 2022.
What does the evidence show for Exagamglogene Autotemcel in Beta Thalassemia?
Exagamglogene Autotemcel has 1 completed registered trial for Beta Thalassemia but no linked publication, which usually means results are unpublished, pending, or not yet matched to this record. OSMF's existing evidence tier for this pair is A (Strong), derived from the strength of the disease association recorded in DGIdb, in this case via the biomarker or target BCL11A. The tier describes how well the drug-disease link is documented, not how well the drug works.
Cite this page
Data: ClinicalTrials.gov, PubMed, Open Targets, ChEMBL, DGIdb and the OSMF therapeutic agent database. Last updated 2026-07-18. Page built 2026-10-07.