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 | onasemnogene abeparvovec (Zolgensma, AVXS-101, onasemnogene abeparvovec-xioi) Novartis·SMN1 (AAV9) 28 trials |
|---|---|
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 |
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 | Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators |
Positioning Known limitation | Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk |
Positioning Development positioning | Disease-modifying gene therapy (often 1L for eligible infants) |
Technology Vector | AAV9 |
MoA Mechanism | Onasemnogene abeparvovec is a non-replicating AAV9 vector delivering a functional SMN1 transgene to motor neurons and other tissues, restoring SMN protein production in spinal muscular atrophy. |
MoA Biomarker | CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation |
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 Safety signal | Dose-dependent hepatotoxicity; transient liver enzyme elevations in clinic |
Clinical Program phase | APPROVED |
Clinical Trial activity | No active/completed counts |
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 | onasemnogene abeparvovec (Zolgensma, AVXS-101, onasemnogene abeparvovec-xioi) Novartis·SMN1 (AAV9) 28 trials |
|---|---|
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 |
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 | Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators |
Positioning Known limitation | Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk |
Positioning Development positioning | Disease-modifying gene therapy (often 1L for eligible infants) |
Technology Vector | AAV9 |
MoA Mechanism | Onasemnogene abeparvovec is a non-replicating AAV9 vector delivering a functional SMN1 transgene to motor neurons and other tissues, restoring SMN protein production in spinal muscular atrophy. |
MoA Biomarker | CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation |
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 Safety signal | Dose-dependent hepatotoxicity; transient liver enzyme elevations in clinic |
Clinical Program phase | APPROVED |
Clinical Trial activity | No active/completed counts |
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