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 | voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl) Spark Therapeutics / Roche·RPE65 (AAV2) 7 trials | delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl) Sarepta Therapeutics·Micro-dystrophin (AAVrh74) 12 trials |
|---|---|---|
Overview Program | Luxturna (voretigene neparvovec) | Elevidys (delandistrogene moxeparvovec) |
Overview Company | Spark Therapeutics / Roche | Sarepta Therapeutics |
Overview Modality | CGT | CGT |
Overview Target | RPE65 (AAV2) | Micro-dystrophin (AAVrh74) |
Overview Indication | Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations | Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations |
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 | Surgical subretinal delivery; landmark ophthalmology gene therapy | Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs |
Positioning Known limitation | bypasses defective phototransduction pathways, showing promise for patients regardless of their genetic profile | Acute liver injury, myocarditis, immune myositis |
Technology Vector | AAV2 | AAVrh74 |
MoA Mechanism | Voretigene neparvovec uses AAV2 to deliver functional RPE65 to retinal pigment epithelium, restoring the visual cycle and improving light sensitivity in RPE65-mutant IRD. | Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients. |
MoA Biomarker | MLMT, FST, BCVA. | Micro-dystrophin expression, NSAA motor function. |
Toxicology Species | NHP, Dog | — |
Toxicology Animal (cat.) | NHP, Dog | — |
Toxicology Major finding | Procedure-related ocular AEs; inflammation manageable with steroids | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Safety signal | Procedure-related ocular AEs; inflammation manageable with steroids | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Program phase | APPROVED | APPROVED |
Clinical Trial activity | 4 recruiting · 3 completed | 4 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 | voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl) Spark Therapeutics / Roche·RPE65 (AAV2) 7 trials | delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl) Sarepta Therapeutics·Micro-dystrophin (AAVrh74) 12 trials |
|---|---|---|
Overview Program | Luxturna (voretigene neparvovec) | Elevidys (delandistrogene moxeparvovec) |
Overview Company | Spark Therapeutics / Roche | Sarepta Therapeutics |
Overview Modality | CGT | CGT |
Overview Target | RPE65 (AAV2) | Micro-dystrophin (AAVrh74) |
Overview Indication | Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations | Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations |
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 | Surgical subretinal delivery; landmark ophthalmology gene therapy | Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs |
Positioning Known limitation | bypasses defective phototransduction pathways, showing promise for patients regardless of their genetic profile | Acute liver injury, myocarditis, immune myositis |
Technology Vector | AAV2 | AAVrh74 |
MoA Mechanism | Voretigene neparvovec uses AAV2 to deliver functional RPE65 to retinal pigment epithelium, restoring the visual cycle and improving light sensitivity in RPE65-mutant IRD. | Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients. |
MoA Biomarker | MLMT, FST, BCVA. | Micro-dystrophin expression, NSAA motor function. |
Toxicology Species | NHP, Dog | — |
Toxicology Animal (cat.) | NHP, Dog | — |
Toxicology Major finding | Procedure-related ocular AEs; inflammation manageable with steroids | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Safety signal | Procedure-related ocular AEs; inflammation manageable with steroids | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical Program phase | APPROVED | APPROVED |
Clinical Trial activity | 4 recruiting · 3 completed | 4 recruiting · 6 completed |
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