타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 자동 수집 갱신 권장 · 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | exagamglogene autotemcel (Casgevy, exa-cel, CTX001) Vertex / CRISPR Therapeutics·BCL11A enhancer (CRISPR/Cas9) → Hb… 9 trials |
|---|---|
Overview Program | Casgevy (exagamglogene autotemcel) |
Overview Company | Vertex / CRISPR Therapeutics |
Overview Modality | CGT |
Overview Target | BCL11A enhancer (CRISPR/Cas9) → HbF |
Overview Indication | Sickle cell disease with recurrent VOCs; transfusion-dependent β-thalassemia |
Overview Phase | APPROVED |
Overview Status | APPROVED |
Overview Content status | Curated Core |
Overview Data Confidence | Editorial scope · Curated Core |
Overview Development Signal | Development Signal · Established |
Overview Approval status | FDA approved |
Positioning Key differentiator | First approved CRISPR medicine; HbF induction vs lentiviral gene addition |
Positioning Known limitation | bypassing such limitations |
Technology Vector | Electroporation of Cas9 RNP (no integrating viral vector for edit) |
MoA Mechanism | Ex vivo CRISPR/Cas9 editing of autologous CD34+ cells to disrupt BCL11A erythroid enhancer, reactivating fetal hemoglobin (HbF) in sickle cell disease. |
MoA Biomarker | HbF fraction, VOC rate, hospitalization days. |
Toxicology Major finding | Conditioning-related cytopenias, mucositis, infection risk |
Clinical Safety signal | Conditioning-related cytopenias, mucositis, infection risk |
Clinical Program phase | APPROVED |
Clinical Trial activity | 5 recruiting · 2 completed |
타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 자동 수집 갱신 권장 · 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | exagamglogene autotemcel (Casgevy, exa-cel, CTX001) Vertex / CRISPR Therapeutics·BCL11A enhancer (CRISPR/Cas9) → Hb… 9 trials |
|---|---|
Overview Program | Casgevy (exagamglogene autotemcel) |
Overview Company | Vertex / CRISPR Therapeutics |
Overview Modality | CGT |
Overview Target | BCL11A enhancer (CRISPR/Cas9) → HbF |
Overview Indication | Sickle cell disease with recurrent VOCs; transfusion-dependent β-thalassemia |
Overview Phase | APPROVED |
Overview Status | APPROVED |
Overview Content status | Curated Core |
Overview Data Confidence | Editorial scope · Curated Core |
Overview Development Signal | Development Signal · Established |
Overview Approval status | FDA approved |
Positioning Key differentiator | First approved CRISPR medicine; HbF induction vs lentiviral gene addition |
Positioning Known limitation | bypassing such limitations |
Technology Vector | Electroporation of Cas9 RNP (no integrating viral vector for edit) |
MoA Mechanism | Ex vivo CRISPR/Cas9 editing of autologous CD34+ cells to disrupt BCL11A erythroid enhancer, reactivating fetal hemoglobin (HbF) in sickle cell disease. |
MoA Biomarker | HbF fraction, VOC rate, hospitalization days. |
Toxicology Major finding | Conditioning-related cytopenias, mucositis, infection risk |
Clinical Safety signal | Conditioning-related cytopenias, mucositis, infection risk |
Clinical Program phase | APPROVED |
Clinical Trial activity | 5 recruiting · 2 completed |
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