구조모아 (StructureMoa)항암 chemical structure spider web
방문분석
큐레이션 브리핑
AAV2 RPE65 망막 유전자치료. 최초 FDA 승인 in vivo AAV. MLMT·BCVA·수술적 망막하 주입 리스크.
경쟁 포지션: Only approved RPE65 gene therapy
기전. Voretigene neparvovec uses AAV2 to deliver functional RPE65 to retinal pigment epithelium, restoring the visual cycle and improving light sensitivity in RPE65-mutant IRD.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
RPE65 (AAV2)
Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations
US, EU
Approved
luxturna · voretigene neparvovec · Luxturna · voretigene neparvovec-rzyl · AAV2-hRPE65v2
2026-09-10
Data Confidence · High
Development Signal · Established
AAV gene therapy
AAV2
Surgical subretinal delivery; landmark ophthalmology gene therapy
구조화 데이터
Surgical subretinal delivery; landmark ophthalmology gene therapy
bypasses defective phototransduction pathways, showing promise for patients regardless of their genetic profile
AAV2
RPE65 (AAV2) · MLMT, FST, BCVA.
Intraocular inflammation, cataract, retinal tear
Only approved RPE65 gene therapy
Patients with viable retinal cells and biallelic RPE65 mutations
Subretinal injection (periocular surgery)
One-time per eye (sequential)
Intraocular inflammation, cataract, retinal tear
Only approved RPE65 gene therapy
Voretigene neparvovec uses AAV2 to deliver functional RPE65 to retinal pigment epithelium, restoring the visual cycle and improving light sensitivity in RPE65-mutant IRD.
payload expression
MLMT, FST, BCVA.
bypasses defective phototransduction pathways, showing promise for patients regardless of their genetic profile
구조화 데이터
nonhuman primate
biodistribution
MLMT score improvement
구조화 데이터
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
FDA approval (RPE65 IRD)
APPROVAL · 2017년 12월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.
분석
큐레이션 브리핑
AAV2 RPE65 망막 유전자치료. 최초 FDA 승인 in vivo AAV. MLMT·BCVA·수술적 망막하 주입 리스크.
경쟁 포지션: Only approved RPE65 gene therapy
기전. Voretigene neparvovec uses AAV2 to deliver functional RPE65 to retinal pigment epithelium, restoring the visual cycle and improving light sensitivity in RPE65-mutant IRD.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
RPE65 (AAV2)
Inherited retinal dystrophy due to confirmed biallelic RPE65 mutations
US, EU
Approved
luxturna · voretigene neparvovec · Luxturna · voretigene neparvovec-rzyl · AAV2-hRPE65v2
2026-09-10
Data Confidence · High
Development Signal · Established
AAV gene therapy
AAV2
Surgical subretinal delivery; landmark ophthalmology gene therapy
구조화 데이터
Surgical subretinal delivery; landmark ophthalmology gene therapy
bypasses defective phototransduction pathways, showing promise for patients regardless of their genetic profile
AAV2
RPE65 (AAV2) · MLMT, FST, BCVA.
Intraocular inflammation, cataract, retinal tear
Only approved RPE65 gene therapy
Patients with viable retinal cells and biallelic RPE65 mutations
Subretinal injection (periocular surgery)
One-time per eye (sequential)
Intraocular inflammation, cataract, retinal tear
Only approved RPE65 gene therapy
Voretigene neparvovec uses AAV2 to deliver functional RPE65 to retinal pigment epithelium, restoring the visual cycle and improving light sensitivity in RPE65-mutant IRD.
payload expression
MLMT, FST, BCVA.
bypasses defective phototransduction pathways, showing promise for patients regardless of their genetic profile
구조화 데이터
nonhuman primate
biodistribution
MLMT score improvement
구조화 데이터
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
FDA approval (RPE65 IRD)
APPROVAL · 2017년 12월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.