구조모아 (StructureMoa)항암 chemical structure spider web
방문분석
큐레이션 브리핑
AAV9 SMN1 유전자치료(onasemnogene). 소아 SMA 1회 IV. Spinraza·Evrysdi와 event-free survival·운동이정표·간독성 모니터링이 비교 핵심.
경쟁 포지션: Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
기전. 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.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
SMN1 (AAV9)
Spinal muscular atrophy (SMA) Type 1–3, pediatric
US, EU, JP, KR
Approved
zolgensma · onasemnogene abeparvovec · Zolgensma · onasemnogene abeparvovec-xioi · AVXS-101
2026-09-10
Data Confidence · High
Development Signal · Established
AAV gene therapy
AAV9
Suspension cell culture AAV production
Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators
구조화 데이터
Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators
Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk
AAV9
SMN1 (AAV9) · CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation
Acute liver injury / elevated aminotransferases; corticosteroid cover
Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
Pediatric patients with bi-allelic SMN1 mutations (label age/weight limits)
Disease-modifying gene therapy (often 1L for eligible infants)
Single intravenous infusion
One-time
Acute liver injury / elevated aminotransferases; corticosteroid cover
Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
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.
Macrophage Activation
CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation
Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk
구조화 데이터
mouse
Vector genome persists; SMN expression durable in responders
SMN protein in CSF/blood; motor milestone attainment
Systemic IV AAV9 with CNS/motor neuron tropism; vector genome persistence
구조화 데이터
임상시험이 많습니다. 임상시험 탭에서 Phase/Status 필터·검색을 사용하세요.
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
FDA approval (SMA)
APPROVAL · 2019년 5월
EMA approval
APPROVAL · 2020년 5월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.
분석
큐레이션 브리핑
AAV9 SMN1 유전자치료(onasemnogene). 소아 SMA 1회 IV. Spinraza·Evrysdi와 event-free survival·운동이정표·간독성 모니터링이 비교 핵심.
경쟁 포지션: Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
기전. 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.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
SMN1 (AAV9)
Spinal muscular atrophy (SMA) Type 1–3, pediatric
US, EU, JP, KR
Approved
zolgensma · onasemnogene abeparvovec · Zolgensma · onasemnogene abeparvovec-xioi · AVXS-101
2026-09-10
Data Confidence · High
Development Signal · Established
AAV gene therapy
AAV9
Suspension cell culture AAV production
Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators
구조화 데이터
Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators
Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk
AAV9
SMN1 (AAV9) · CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation
Acute liver injury / elevated aminotransferases; corticosteroid cover
Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
Pediatric patients with bi-allelic SMN1 mutations (label age/weight limits)
Disease-modifying gene therapy (often 1L for eligible infants)
Single intravenous infusion
One-time
Acute liver injury / elevated aminotransferases; corticosteroid cover
Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
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.
Macrophage Activation
CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation
Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk
구조화 데이터
mouse
Vector genome persists; SMN expression durable in responders
SMN protein in CSF/blood; motor milestone attainment
Systemic IV AAV9 with CNS/motor neuron tropism; vector genome persistence
구조화 데이터
임상시험이 많습니다. 임상시험 탭에서 Phase/Status 필터·검색을 사용하세요.
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
FDA approval (SMA)
APPROVAL · 2019년 5월
EMA approval
APPROVAL · 2020년 5월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.