구조모아 (StructureMoa)항암 chemical structure spider web
방문Sarepta Therapeutics
분석
큐레이션 브리핑
AAVrh74 micro-dystrophin DMD 유전자치료. 발현 vs 기능(NSAA) 논쟁·간독성·근염 신호가 핵심.
경쟁 포지션: First approved AAV gene therapy for DMD in the US
기전. Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
Micro-dystrophin (AAVrh74)
Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations
US
Approved
elevidys · delandistrogene moxeparvovec · Elevidys · delandistrogene moxeparvovec-rokl · SRP-9001
2026-09-10
Data Confidence · High
Development Signal · Established
AAV gene therapy
AAVrh74
Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs
구조화 데이터
Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs
Acute liver injury, myocarditis, immune myositis
AAVrh74
Micro-dystrophin (AAVrh74) · Micro-dystrophin expression, NSAA motor function.
Acute liver injury, myocarditis, immune myositis
First approved AAV gene therapy for DMD in the US
Ambulatory DMD patients meeting AAVrh74 Ab and age criteria
Single IV infusion
One-time
Acute liver injury, myocarditis, immune myositis
First approved AAV gene therapy for DMD in the US
Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients.
Immune-Mediated Myocardial Injury and Myocarditis After Gene Replacement Therapy: A Systematic Review and Meta-Analysis
Micro-dystrophin expression, NSAA motor function.
구조화 데이터
non-human primates
pd
Micro-dystrophin Western blot; functional motor scores
구조화 데이터
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
FDA accelerated approval (DMD)
APPROVAL · 2023년 6월
Label expansion (broader ages)
APPROVAL · 2024년 6월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.
Sarepta Therapeutics
분석
큐레이션 브리핑
AAVrh74 micro-dystrophin DMD 유전자치료. 발현 vs 기능(NSAA) 논쟁·간독성·근염 신호가 핵심.
경쟁 포지션: First approved AAV gene therapy for DMD in the US
기전. Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
Micro-dystrophin (AAVrh74)
Duchenne muscular dystrophy (DMD) — ambulatory / label-expanded populations
US
Approved
elevidys · delandistrogene moxeparvovec · Elevidys · delandistrogene moxeparvovec-rokl · SRP-9001
2026-09-10
Data Confidence · High
Development Signal · Established
AAV gene therapy
AAVrh74
Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs
구조화 데이터
Muscle-directed AAV micro-dystrophin vs exon-skipping ASOs
Acute liver injury, myocarditis, immune myositis
AAVrh74
Micro-dystrophin (AAVrh74) · Micro-dystrophin expression, NSAA motor function.
Acute liver injury, myocarditis, immune myositis
First approved AAV gene therapy for DMD in the US
Ambulatory DMD patients meeting AAVrh74 Ab and age criteria
Single IV infusion
One-time
Acute liver injury, myocarditis, immune myositis
First approved AAV gene therapy for DMD in the US
Delandistrogene moxeparvovec (AAVrh74) delivers micro-dystrophin transgene to skeletal/cardiac muscle in Duchenne muscular dystrophy ambulatory patients.
Immune-Mediated Myocardial Injury and Myocarditis After Gene Replacement Therapy: A Systematic Review and Meta-Analysis
Micro-dystrophin expression, NSAA motor function.
구조화 데이터
non-human primates
pd
Micro-dystrophin Western blot; functional motor scores
구조화 데이터
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
FDA accelerated approval (DMD)
APPROVAL · 2023년 6월
Label expansion (broader ages)
APPROVAL · 2024년 6월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.