검색으로 10,000+ 프로그램 중 최대 5개를 골라 PK/PD · 독성 · 임상을 나란히 비교합니다. · 다음 갱신 D-13 · 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 | onasemnogene abeparvovec (Zolgensma, AVXS-101, onasemnogene abeparvovec-xioi) Novartis·SMN1 (AAV9) ORR null·t½ Vector genome persists; SMN expression durable in responders |
|---|---|
Overview Program | Zolgensma (onasemnogene abeparvovec) |
Overview Company | Novartis |
Overview Modality | CGT |
Overview Target | SMN1 (AAV9) |
Overview Indication | Spinal muscular atrophy (SMA) Type 1–3, pediatric |
Overview Phase | APPROVED |
Overview Status | APPROVED |
MoA Mechanism | AAV9 delivers SMN1 cDNA to motor neurons and other tissues → restores SMN protein → improves motor neuron survival |
MoA Biomarker | CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation |
PK/PD Half-life | Vector genome persists; SMN expression durable in responders |
PK/PD Species | Mouse, SMN protein, motor scores |
PK/PD Animal (cat.) | Mouse |
PK/PD Experiment | Pharmacokinetic |
Toxicology Species | NHP, Mouse |
Toxicology Animal (cat.) | Mouse, NHP |
Toxicology Major finding | Dose-dependent hepatotoxicity; transient liver enzyme elevations in clinic |
Toxicology CRS | N/A (not CAR-T) |
Clinical PFS | Motor milestone attainment (sitting) in STR1VE/START |
Clinical Result source | START / STR1VE / FDA label |
Clinical Data Tier / Score | A · 67 |
Clinical Program phase | APPROVED |
Clinical Dose / schedule | One-time |
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-13 · 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 | onasemnogene abeparvovec (Zolgensma, AVXS-101, onasemnogene abeparvovec-xioi) Novartis·SMN1 (AAV9) ORR null·t½ Vector genome persists; SMN expression durable in responders |
|---|---|
Overview Program | Zolgensma (onasemnogene abeparvovec) |
Overview Company | Novartis |
Overview Modality | CGT |
Overview Target | SMN1 (AAV9) |
Overview Indication | Spinal muscular atrophy (SMA) Type 1–3, pediatric |
Overview Phase | APPROVED |
Overview Status | APPROVED |
MoA Mechanism | AAV9 delivers SMN1 cDNA to motor neurons and other tissues → restores SMN protein → improves motor neuron survival |
MoA Biomarker | CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation |
PK/PD Half-life | Vector genome persists; SMN expression durable in responders |
PK/PD Species | Mouse, SMN protein, motor scores |
PK/PD Animal (cat.) | Mouse |
PK/PD Experiment | Pharmacokinetic |
Toxicology Species | NHP, Mouse |
Toxicology Animal (cat.) | Mouse, NHP |
Toxicology Major finding | Dose-dependent hepatotoxicity; transient liver enzyme elevations in clinic |
Toxicology CRS | N/A (not CAR-T) |
Clinical PFS | Motor milestone attainment (sitting) in STR1VE/START |
Clinical Result source | START / STR1VE / FDA label |
Clinical Data Tier / Score | A · 67 |
Clinical Program phase | APPROVED |
Clinical Dose / schedule | One-time |
Clinical Clinical sync | 2026년 7월 12일 (1일 전) |
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