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 | delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl) Sarepta Therapeutics·Micro-dystrophin (AAVrh74) 12 trials |
|---|---|
Overview Program | Elevidys (delandistrogene moxeparvovec) |
Overview Company | Sarepta Therapeutics |
Overview Modality | CGT |
Overview Target | Micro-dystrophin (AAVrh74) |
Overview Indication | Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations |
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 | Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs |
Positioning Known limitation | Acute liver injury, myocarditis, immune myositis |
Technology Vector | AAVrh74 |
MoA Mechanism | Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients. |
MoA Biomarker | Micro-dystrophin expression, NSAA motor function. |
Toxicology Major finding | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Safety signal | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Program phase | APPROVED |
Clinical Trial activity | 4 recruiting · 6 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 | delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl) Sarepta Therapeutics·Micro-dystrophin (AAVrh74) 12 trials |
|---|---|
Overview Program | Elevidys (delandistrogene moxeparvovec) |
Overview Company | Sarepta Therapeutics |
Overview Modality | CGT |
Overview Target | Micro-dystrophin (AAVrh74) |
Overview Indication | Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations |
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 | Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs |
Positioning Known limitation | Acute liver injury, myocarditis, immune myositis |
Technology Vector | AAVrh74 |
MoA Mechanism | Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients. |
MoA Biomarker | Micro-dystrophin expression, NSAA motor function. |
Toxicology Major finding | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Safety signal | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Program phase | APPROVED |
Clinical Trial activity | 4 recruiting · 6 completed |
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