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 | voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl) Spark Therapeutics / Roche·RPE65 (AAV2) ORR null | delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl) Sarepta Therapeutics·Micro-dystrophin (AAVrh74) ORR null |
|---|---|---|
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 |
MoA Mechanism | AAV2-mediated RPE65 expression restores visual cycle isomerohydrolase activity in RPE | AAVrh74 delivers truncated dystrophin designed to restore membrane stability |
MoA Biomarker | MLMT score, full-field light sensitivity, BCVA | Micro-dystrophin IHC/%, CK, NSAA |
PK/PD Species | NHP, Despite these findings, and no systemic toxicity was identified., MLMT, FST | Mouse |
PK/PD Animal (cat.) | NHP | Mouse |
PK/PD Experiment | biodistribution | pd |
Toxicology Species | NHP, Dog, Despite these findings, and no systemic toxicity was identified. | Mouse |
Toxicology Animal (cat.) | NHP, Dog | — |
Toxicology Major finding | Procedure-related ocular AEs; inflammation manageable with steroids | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical PFS | Sustained functional vision gains | — |
Clinical Result source | Phase 3 / FDA label | FDA label / EMBARK |
Clinical Data Tier / Score | C · 54 | S · 56 |
Clinical Program phase | APPROVED | APPROVED |
Clinical Dose / schedule | One-time per eye (sequential) | 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 | voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl) Spark Therapeutics / Roche·RPE65 (AAV2) ORR null | delandistrogene moxeparvovec (Elevidys, SRP-9001, delandistrogene moxeparvovec-rokl) Sarepta Therapeutics·Micro-dystrophin (AAVrh74) ORR null |
|---|---|---|
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 |
MoA Mechanism | AAV2-mediated RPE65 expression restores visual cycle isomerohydrolase activity in RPE | AAVrh74 delivers truncated dystrophin designed to restore membrane stability |
MoA Biomarker | MLMT score, full-field light sensitivity, BCVA | Micro-dystrophin IHC/%, CK, NSAA |
PK/PD Species | NHP, Despite these findings, and no systemic toxicity was identified., MLMT, FST | Mouse |
PK/PD Animal (cat.) | NHP | Mouse |
PK/PD Experiment | biodistribution | pd |
Toxicology Species | NHP, Dog, Despite these findings, and no systemic toxicity was identified. | Mouse |
Toxicology Animal (cat.) | NHP, Dog | — |
Toxicology Major finding | Procedure-related ocular AEs; inflammation manageable with steroids | Hepatotoxicity; immune-mediated myositis/myocarditis signals |
Clinical PFS | Sustained functional vision gains | — |
Clinical Result source | Phase 3 / FDA label | FDA label / EMBARK |
Clinical Data Tier / Score | C · 54 | S · 56 |
Clinical Program phase | APPROVED | APPROVED |
Clinical Dose / schedule | One-time per eye (sequential) | One-time |
Clinical Clinical sync | 2026년 7월 12일 (1일 전) | 2026년 7월 12일 (1일 전) |
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