검색으로 10,000+ 프로그램 중 최대 5개를 골라 PK/PD · 독성 · 임상을 나란히 비교합니다. · 다음 갱신 D-14 · 마지막 7월 13일
현재 선택: 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 | voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl) Spark Therapeutics / Roche·RPE65 (AAV2) ORR null |
|---|---|
Overview Program | Luxturna (voretigene neparvovec) |
Overview Company | Spark Therapeutics / Roche |
Overview Modality | CGT |
Overview Target | RPE65 (AAV2) |
Overview Indication | Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations |
Overview Phase | APPROVED |
Overview Status | APPROVED |
MoA Mechanism | AAV2-mediated RPE65 expression restores visual cycle isomerohydrolase activity in RPE |
MoA Biomarker | MLMT score, full-field light sensitivity, BCVA |
PK/PD Species | NHP, Despite these findings, and no systemic toxicity was identified., MLMT, FST |
PK/PD Animal (cat.) | NHP |
PK/PD Experiment | biodistribution |
Toxicology Species | NHP, Dog, Despite these findings, and no systemic toxicity was identified. |
Toxicology Animal (cat.) | NHP, Dog |
Toxicology Major finding | Procedure-related ocular AEs; inflammation manageable with steroids |
Clinical PFS | Sustained functional vision gains |
Clinical Result source | Phase 3 / FDA label |
Clinical Data Tier / Score | A · 54 |
Clinical Program phase | APPROVED |
Clinical Dose / schedule | One-time per eye (sequential) |
Clinical Clinical sync | 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: 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 | voretigene neparvovec (Luxturna, AAV2-hRPE65v2, voretigene neparvovec-rzyl) Spark Therapeutics / Roche·RPE65 (AAV2) ORR null |
|---|---|
Overview Program | Luxturna (voretigene neparvovec) |
Overview Company | Spark Therapeutics / Roche |
Overview Modality | CGT |
Overview Target | RPE65 (AAV2) |
Overview Indication | Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations |
Overview Phase | APPROVED |
Overview Status | APPROVED |
MoA Mechanism | AAV2-mediated RPE65 expression restores visual cycle isomerohydrolase activity in RPE |
MoA Biomarker | MLMT score, full-field light sensitivity, BCVA |
PK/PD Species | NHP, Despite these findings, and no systemic toxicity was identified., MLMT, FST |
PK/PD Animal (cat.) | NHP |
PK/PD Experiment | biodistribution |
Toxicology Species | NHP, Dog, Despite these findings, and no systemic toxicity was identified. |
Toxicology Animal (cat.) | NHP, Dog |
Toxicology Major finding | Procedure-related ocular AEs; inflammation manageable with steroids |
Clinical PFS | Sustained functional vision gains |
Clinical Result source | Phase 3 / FDA label |
Clinical Data Tier / Score | A · 54 |
Clinical Program phase | APPROVED |
Clinical Dose / schedule | One-time per eye (sequential) |
Clinical Clinical sync | 2026년 7월 12일 (1일 전) |
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