One-time IV AAV9 gene therapy delivering a functional SMN1 copy for pediatric SMA. First systemic AAV gene therapy approved for SMA; hepatotoxicity monitoring is central to the label. PFS: Motor milestone attainment (sitting) in STR1VE/START.
SMN1 (AAV9)
Spinal muscular atrophy (SMA) Type 1–3, pediatric
US, EU, JP, KR
Approved
AAV gene therapy
SMN1 cDNA under hybrid CMV enhancer/chicken β-actin promoter
AAV9
Suspension cell culture AAV production
Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators
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
Event-free survival, CHOP-INTEND motor milestones
Acute liver injury / elevated aminotransferases; corticosteroid cover
Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
AAV9 delivers SMN1 cDNA to motor neurons and other tissues → restores SMN protein → improves motor neuron survival
Macrophage Activation
CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation
Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk
차트 로드 중…
임상시험이 많습니다. 임상시험 탭에서 Phase/Status 필터·검색을 사용하세요.
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다.
FDA approval (SMA)
APPROVAL · 2019년 5월
EMA approval
APPROVAL · 2020년 5월
One-time IV AAV9 gene therapy delivering a functional SMN1 copy for pediatric SMA. First systemic AAV gene therapy approved for SMA; hepatotoxicity monitoring is central to the label. PFS: Motor milestone attainment (sitting) in STR1VE/START.
SMN1 (AAV9)
Spinal muscular atrophy (SMA) Type 1–3, pediatric
US, EU, JP, KR
Approved
AAV gene therapy
SMN1 cDNA under hybrid CMV enhancer/chicken β-actin promoter
AAV9
Suspension cell culture AAV production
Single-dose systemic SMN1 replacement vs chronic SMN2 splicing modulators
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
Event-free survival, CHOP-INTEND motor milestones
Acute liver injury / elevated aminotransferases; corticosteroid cover
Only one-time AAV SMN1 gene therapy vs Spinraza / Evrysdi chronic dosing
AAV9 delivers SMN1 cDNA to motor neurons and other tissues → restores SMN protein → improves motor neuron survival
Macrophage Activation
CHOP-INTEND, sitting/walking milestones, survival without permanent ventilation
Pre-existing anti-AAV9 neutralizing antibodies; irreversible hepatotoxicity risk
차트 로드 중…
임상시험이 많습니다. 임상시험 탭에서 Phase/Status 필터·검색을 사용하세요.
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다.
FDA approval (SMA)
APPROVAL · 2019년 5월
EMA approval
APPROVAL · 2020년 5월