타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 자동 수집 갱신 권장 · 9월 2일
현재 선택: 2개 · 임상 갱신 필요 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 | etranacogene dezaparvovec (Hemgenix, AMT-061, CSL222, etranacogene dezaparvovec-drlb) CSL Behring·Factor IX (AAV5 Padua) 7 trials | valoctocogene roxaparvovec (Roctavian, BMN 270, valoctocogene roxaparvovec-rvox) BioMarin·Factor VIII (AAV5) 8 trials |
|---|---|---|
Overview Program | Hemgenix (etranacogene dezaparvovec) | Roctavian (valoctocogene roxaparvovec) |
Overview Company | CSL Behring | BioMarin |
Overview Modality | CGT | CGT |
Overview Target | Factor IX (AAV5 Padua) | Factor VIII (AAV5) |
Overview Indication | Hemophilia B (congenital Factor IX deficiency) in adults | Severe hemophilia A in adults |
Overview Phase | APPROVED | APPROVED |
Overview Status | APPROVED | APPROVED |
Overview Content status | Curated Core | Curated Core |
Overview Data Confidence | Editorial scope · Curated Core | Editorial scope · Curated Core |
Overview Development Signal | Development Signal · Established | Development Signal · Established |
Overview Approval status | FDA approved | FDA approved |
Positioning Key differentiator | High-activity FIX Padua enables durable FIX levels from liver-directed AAV5 | First FDA-approved gene therapy for hemophilia A |
Positioning Known limitation | ALT elevations, infusion reactions; AAV5 NAb screening | bypassing agents (BPAs), recombinant factor VIII (rFVIII), recombinant factor VIII Fc fusion protein (rFVIIIFc), and gene ther |
Technology Vector | AAV5 | AAV5 |
MoA Mechanism | Etranacogene dezaparvovec (AAV5) delivers FIX Padua transgene to hepatocytes for sustained Factor IX expression in hemophilia B. | Valoctocogene roxaparvovec (AAV5-hFVIII) provides liver-directed Factor VIII expression for severe hemophilia A. |
MoA Biomarker | FIX activity (IU/dL), annualized bleeding rate. | FVIII activity, ABR. |
Toxicology Species | NHP, Mouse | — |
Toxicology Major finding | Transient hepatotoxicity / ALT rise; corticosteroid management | Hepatocellular enzyme elevations; steroid protocols |
Clinical Safety signal | Transient hepatotoxicity / ALT rise; corticosteroid management | Hepatocellular enzyme elevations; steroid protocols |
Clinical Program phase | APPROVED | APPROVED |
Clinical Trial activity | 4 recruiting · 2 completed | 1 recruiting · 6 completed |
타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 자동 수집 갱신 권장 · 9월 2일
현재 선택: 2개 · 임상 갱신 필요 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 | etranacogene dezaparvovec (Hemgenix, AMT-061, CSL222, etranacogene dezaparvovec-drlb) CSL Behring·Factor IX (AAV5 Padua) 7 trials | valoctocogene roxaparvovec (Roctavian, BMN 270, valoctocogene roxaparvovec-rvox) BioMarin·Factor VIII (AAV5) 8 trials |
|---|---|---|
Overview Program | Hemgenix (etranacogene dezaparvovec) | Roctavian (valoctocogene roxaparvovec) |
Overview Company | CSL Behring | BioMarin |
Overview Modality | CGT | CGT |
Overview Target | Factor IX (AAV5 Padua) | Factor VIII (AAV5) |
Overview Indication | Hemophilia B (congenital Factor IX deficiency) in adults | Severe hemophilia A in adults |
Overview Phase | APPROVED | APPROVED |
Overview Status | APPROVED | APPROVED |
Overview Content status | Curated Core | Curated Core |
Overview Data Confidence | Editorial scope · Curated Core | Editorial scope · Curated Core |
Overview Development Signal | Development Signal · Established | Development Signal · Established |
Overview Approval status | FDA approved | FDA approved |
Positioning Key differentiator | High-activity FIX Padua enables durable FIX levels from liver-directed AAV5 | First FDA-approved gene therapy for hemophilia A |
Positioning Known limitation | ALT elevations, infusion reactions; AAV5 NAb screening | bypassing agents (BPAs), recombinant factor VIII (rFVIII), recombinant factor VIII Fc fusion protein (rFVIIIFc), and gene ther |
Technology Vector | AAV5 | AAV5 |
MoA Mechanism | Etranacogene dezaparvovec (AAV5) delivers FIX Padua transgene to hepatocytes for sustained Factor IX expression in hemophilia B. | Valoctocogene roxaparvovec (AAV5-hFVIII) provides liver-directed Factor VIII expression for severe hemophilia A. |
MoA Biomarker | FIX activity (IU/dL), annualized bleeding rate. | FVIII activity, ABR. |
Toxicology Species | NHP, Mouse | — |
Toxicology Major finding | Transient hepatotoxicity / ALT rise; corticosteroid management | Hepatocellular enzyme elevations; steroid protocols |
Clinical Safety signal | Transient hepatotoxicity / ALT rise; corticosteroid management | Hepatocellular enzyme elevations; steroid protocols |
Clinical Program phase | APPROVED | APPROVED |
Clinical Trial activity | 4 recruiting · 2 completed | 1 recruiting · 6 completed |
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