Exagamglogene autotemcel
Trade name: Casgevy
Cell therapyapprovedOrphan Drug FDA
Approved
Dec 8, 2023
Clinical trial activity
8 trials · 3 clinical orgs · 1 marketing orgs
Phase 1
3
Phase 2
8
Phase 3
5
Phase 4
0
Earliest trial started Sep 27, 2018 (NCT03655678)
Timeline
Indications
Mechanism of action
- B-cell lymphoma/leukemia 11ABLOCKER
blocker - BCL11A
- B-cell lymphoma/leukemia 11AGENE EDITING NEGATIVE MODULATOR
B-cell lymphoma/leukemia 11A gene editing negative modulator
Autologous CD34+ cell enriched population with a CRISPR/Cas9 mediated DNA double-strand break at the enhancer region of the BCL11A. Reduced BCL11A expression results in an increase in gamma-globin expression and foetal haemoglobin (HbF) protein production.
Approval history
- approvedDec 8, 2023
Chemistry & pharmacology
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
Oral
No
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL5095474 ↗
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT06308159 | Phase 1/Phase 2 (Phase 2) | May 11, 2024 | Government of China |
| NCT05951205 | Phase 3 | Jan 31, 2024 | Vertex |
| NCT05477563 | Phase 3 | Aug 2, 2022 | CRISPR Therapeutics, Vertex |
| NCT05356195 | Phase 3 | May 3, 2022 | CRISPR Therapeutics, Vertex |
| NCT05329649 | Phase 3 | May 2, 2022 | CRISPR Therapeutics, Vertex |
| NCT04208529 | Phase 3 | Jan 20, 2021 | CRISPR Therapeutics, Vertex |
| NCT03745287 | Phase 1/Phase 2 (Phase 2) | Nov 27, 2018 | CRISPR Therapeutics, Vertex |
| NCT03655678 | Phase 1/Phase 2 (Phase 2) | Sep 27, 2018 | CRISPR Therapeutics, Vertex |
Showing 8 of 8 trials
Page 1 / 1
Organizations
Research & Development (3)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Vertex | For profit | 7 | 6 | 2 | 2018 |
| CRISPR Therapeutics | For profit | 6 | 1 | 2 | 2018 |
| Government of China | Government | 1 | 1 | 1 | 2024 |
3 organizations
Page 1 / 1
Marketing (1)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| AbbVie | For profit | NDA | Dec 8, 2023 |