Exagamglogene autotemcel
ApprovedCTX001
Aliases: Casgevyexa-cel
Source: Company Disclosure·Trials: 4
Modality
Gene Editing
MOA
CFTR Modulator
Target
BCL11A
Pathway
RNA Splicing
SCDThalassemia
Development Pipeline
Preclinical
~Dec 2013
→ ~Mar 2015
Phase 1
~Jun 2015
→ ~Sep 2016
Phase 2
~Dec 2016
→ ~Mar 2018
Phase 3
~Jun 2018
→ ~Sep 2019
NDA/BLA
~Dec 2019
→ ~Mar 2021
Approved
Jun 2021
→ Sep 2027
ApprovedCurrent
NCT03876010
947 pts·Thalassemia
2023-03→2027-09·Terminated
NCT06650518
1,986 pts·Thalassemia
2021-06→2027-02·Terminated
NCT08376011
831 pts·SCD
2024-10→2027-04·Completed
+1 more trial
4,816 total pts2 indications
CompletedCurrentUpcoming
Catalysts (4)
2027-02-0810mo awayPh3 Readout· Thalassemia
2027-04-251.1y awayPh3 Readout· SCD
2027-06-241.2y awayPh3 Readout· Thalassemia
2027-09-181.5y awayPh3 Readout· Thalassemia
Trial Timeline
Q3Q42022Q2Q3Q42023Q2Q3Q42024Q2Q3Q42025Q2Q3Q42026Q2Q3Q42027Q2Q3Q4
Approved
Termina…
Approved
Termina…
Approved
Termina…
Approved
Complet…
Catalysts
Ph3 Readout
2027-02-08 · 10mo away
Thalassemia
Ph3 Readout
2027-04-25 · 1.1y away
SCD
Ph3 Readout
2027-06-24 · 1.2y away
Thalassemia
Ph3 Readout
2027-09-18 · 1.5y away
Thalassemia
CompletedTerminated|StartCompletionToday
Trials (4)
| NCT | Phase | Indication | Status | N | EP |
|---|---|---|---|---|---|
| NCT03876010 | Approved | Thalassemia | Terminated | 947 | CR |
| NCT06650518 | Approved | Thalassemia | Terminated | 1986 | ACR20 |
| NCT08376011 | Approved | SCD | Completed | 831 | HAM-D |
| NCT03037434 | Approved | Thalassemia | Terminated | 1052 | SeizFreq |