검색으로 10,000+ 프로그램 중 최대 5개를 골라 PK/PD · 독성 · 임상을 나란히 비교합니다. · 다음 갱신 D-14 · 마지막 7월 13일
현재 선택: 2개
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… ORR null | lovotibeglogene autotemcel (Lyfgenia, lovo-cel, bb1111) bluebird bio·βA-T87Q-globin (lentiviral) 2 trials |
|---|---|---|
Overview Program | Casgevy (exagamglogene autotemcel) | Lyfgenia (lovotibeglogene autotemcel) |
Overview Company | Vertex / CRISPR Therapeutics | bluebird bio |
Overview Modality | CGT | CGT |
Overview Target | BCL11A enhancer (CRISPR/Cas9) → HbF | βA-T87Q-globin (lentiviral) |
Overview Indication | Sickle cell disease with recurrent VOCs; transfusion-dependent β-thalassemia | Sickle cell disease with recurrent vaso-occlusive events |
Overview Phase | APPROVED | APPROVED |
Overview Status | APPROVED | APPROVED |
MoA Mechanism | Disruption of BCL11A erythroid enhancer → γ-globin / HbF upregulation | LVV integrates anti-sickling β-globin → reduces polymerization of HbS |
MoA Biomarker | HbF%, F-cells, VOC rate, transfusion volume | HbAT87Q%, VOE rate |
PK/PD Species | Rat | — |
PK/PD Animal (cat.) | Rat | Unknown |
PK/PD Experiment | pharmacokinetic | pd |
Toxicology Species | Rat | — |
Toxicology Major finding | Conditioning-related cytopenias, mucositis, infection risk | Hematologic malignancy risk (boxed warning); conditioning toxicities |
Clinical Result source | CLIMB / FDA label | ClinicalTrials.gov NCT02140554 |
Clinical Data Tier / Score | S · 55 | C · 45 |
Clinical Program phase | APPROVED | APPROVED |
Clinical Dose / schedule | One-time | One-time |
Clinical Clinical sync | 2026년 7월 12일 (1일 전) | 2026년 7월 12일 (1일 전) |
검색으로 10,000+ 프로그램 중 최대 5개를 골라 PK/PD · 독성 · 임상을 나란히 비교합니다. · 다음 갱신 D-14 · 마지막 7월 13일
현재 선택: 2개
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… ORR null | lovotibeglogene autotemcel (Lyfgenia, lovo-cel, bb1111) bluebird bio·βA-T87Q-globin (lentiviral) 2 trials |
|---|---|---|
Overview Program | Casgevy (exagamglogene autotemcel) | Lyfgenia (lovotibeglogene autotemcel) |
Overview Company | Vertex / CRISPR Therapeutics | bluebird bio |
Overview Modality | CGT | CGT |
Overview Target | BCL11A enhancer (CRISPR/Cas9) → HbF | βA-T87Q-globin (lentiviral) |
Overview Indication | Sickle cell disease with recurrent VOCs; transfusion-dependent β-thalassemia | Sickle cell disease with recurrent vaso-occlusive events |
Overview Phase | APPROVED | APPROVED |
Overview Status | APPROVED | APPROVED |
MoA Mechanism | Disruption of BCL11A erythroid enhancer → γ-globin / HbF upregulation | LVV integrates anti-sickling β-globin → reduces polymerization of HbS |
MoA Biomarker | HbF%, F-cells, VOC rate, transfusion volume | HbAT87Q%, VOE rate |
PK/PD Species | Rat | — |
PK/PD Animal (cat.) | Rat | Unknown |
PK/PD Experiment | pharmacokinetic | pd |
Toxicology Species | Rat | — |
Toxicology Major finding | Conditioning-related cytopenias, mucositis, infection risk | Hematologic malignancy risk (boxed warning); conditioning toxicities |
Clinical Result source | CLIMB / FDA label | ClinicalTrials.gov NCT02140554 |
Clinical Data Tier / Score | S · 55 | C · 45 |
Clinical Program phase | APPROVED | APPROVED |
Clinical Dose / schedule | One-time | One-time |
Clinical Clinical sync | 2026년 7월 12일 (1일 전) | 2026년 7월 12일 (1일 전) |
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