구조모아 (StructureMoa)항암 chemical structure spider web
방문Orchard Therapeutics (Kyowa Kirin)
분석
큐레이션 브리핑
경쟁 포지션: No approved alternative; HSCT alone does not prevent late-infantile progression.
기전. Autologous CD34+ cells carrying a functional ARSA transgene engraft and differentiate; ARSA-secreting myeloid progeny including CNS microglia restore sulfatide degradation and halt demyelination.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
ARSA (arylsulfatase A) gene
Pre-symptomatic and early-symptomatic metachromatic leukodystrophy
US, EU
Approved
lenmeldy · atidarsagene autotemcel · Lenmeldy · Libmeldy · OTL-200 · arsa-cel
2026-09-10
Data Confidence · High
Development Signal · Established
Ex vivo autologous hematopoietic stem cell gene therapy
Lentiviral vector encoding human ARSA
CD34+ cells transduced ex vivo, then reinfused after myeloablative conditioning
Autologous CD34+ collection → transduction → cryopreserved single-dose product
Transduced microglia-like cells cross into the CNS and cross-correct neighboring cells — enzyme replacement cannot reach that compartment.
구조화 데이터
Transduced microglia-like cells cross into the CNS and cross-correct neighboring cells — enzyme replacement cannot reach that compartment.
Late-symptomatic disease has irreversible demyelination — timing, not biology, is the limit.
Lentiviral vector encoding human ARSA
ARSA (arylsulfatase A) gene · ARSA enzyme activity in peripheral blood mononuclear cells, CSF sulfatide, vector copy number.
Conditioning-related cytopenia, infection, and insertional oncogenesis surveillance
No approved alternative; HSCT alone does not prevent late-infantile progression.
Children with pre-symptomatic late-infantile or juvenile MLD, or early-symptomatic juvenile MLD
First and only disease-modifying option
Single IV infusion after busulfan conditioning
One-time
Conditioning-related cytopenia, infection, and insertional oncogenesis surveillance
No approved alternative; HSCT alone does not prevent late-infantile progression.
Autologous CD34+ cells carrying a functional ARSA transgene engraft and differentiate; ARSA-secreting myeloid progeny including CNS microglia restore sulfatide degradation and halt demyelination.
ARSA enzyme activity in peripheral blood mononuclear cells, CSF sulfatide, vector copy number.
Late-symptomatic disease has irreversible demyelination — timing, not biology, is the limit.
구조화 데이터
Human
Durable engraftment; ARSA activity restored within months of infusion
ARSA activity, GMFM motor scores, DQ/IQ trajectory
구조화 데이터
차트 로드 중…
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
EU approval as Libmeldy
APPROVAL · 2020년 12월
FDA approval as Lenmeldy
APPROVAL · 2024년 3월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.
Orchard Therapeutics (Kyowa Kirin)
분석
큐레이션 브리핑
경쟁 포지션: No approved alternative; HSCT alone does not prevent late-infantile progression.
기전. Autologous CD34+ cells carrying a functional ARSA transgene engraft and differentiate; ARSA-secreting myeloid progeny including CNS microglia restore sulfatide degradation and halt demyelination.
우선 정리한 핵심 프로그램입니다. 공개 출처가 있는 필드만 표시합니다.
구조화 데이터
ARSA (arylsulfatase A) gene
Pre-symptomatic and early-symptomatic metachromatic leukodystrophy
US, EU
Approved
lenmeldy · atidarsagene autotemcel · Lenmeldy · Libmeldy · OTL-200 · arsa-cel
2026-09-10
Data Confidence · High
Development Signal · Established
Ex vivo autologous hematopoietic stem cell gene therapy
Lentiviral vector encoding human ARSA
CD34+ cells transduced ex vivo, then reinfused after myeloablative conditioning
Autologous CD34+ collection → transduction → cryopreserved single-dose product
Transduced microglia-like cells cross into the CNS and cross-correct neighboring cells — enzyme replacement cannot reach that compartment.
구조화 데이터
Transduced microglia-like cells cross into the CNS and cross-correct neighboring cells — enzyme replacement cannot reach that compartment.
Late-symptomatic disease has irreversible demyelination — timing, not biology, is the limit.
Lentiviral vector encoding human ARSA
ARSA (arylsulfatase A) gene · ARSA enzyme activity in peripheral blood mononuclear cells, CSF sulfatide, vector copy number.
Conditioning-related cytopenia, infection, and insertional oncogenesis surveillance
No approved alternative; HSCT alone does not prevent late-infantile progression.
Children with pre-symptomatic late-infantile or juvenile MLD, or early-symptomatic juvenile MLD
First and only disease-modifying option
Single IV infusion after busulfan conditioning
One-time
Conditioning-related cytopenia, infection, and insertional oncogenesis surveillance
No approved alternative; HSCT alone does not prevent late-infantile progression.
Autologous CD34+ cells carrying a functional ARSA transgene engraft and differentiate; ARSA-secreting myeloid progeny including CNS microglia restore sulfatide degradation and halt demyelination.
ARSA enzyme activity in peripheral blood mononuclear cells, CSF sulfatide, vector copy number.
Late-symptomatic disease has irreversible demyelination — timing, not biology, is the limit.
구조화 데이터
Human
Durable engraftment; ARSA activity restored within months of infusion
ARSA activity, GMFM motor scores, DQ/IQ trajectory
구조화 데이터
차트 로드 중…
수집된 임상 필드(phase, status, endpoint 등) 기반 자동 요약입니다. 효능 수치는 선택한 등록 결과이며 전적응증 값이 아닙니다.
EU approval as Libmeldy
APPROVAL · 2020년 12월
FDA approval as Lenmeldy
APPROVAL · 2024년 3월
바이오정보모아는 연구·교육용 과학·의약품 개발 정보입니다. 진료, 진단, 치료 지침, 규제 자문이 아닙니다.