타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 자동 수집 갱신 권장 · 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | atidarsagene autotemcel (Lenmeldy, OTL-200, Libmeldy, arsa-cel) Orchard Therapeutics (Kyowa Kirin)·ARSA (arylsulfatase A) gene 3 trials |
|---|---|
Overview Program | Lenmeldy (atidarsagene autotemcel) |
Overview Company | Orchard Therapeutics (Kyowa Kirin) |
Overview Modality | CGT |
Overview Target | ARSA (arylsulfatase A) gene |
Overview Indication | Pre-symptomatic and early-symptomatic metachromatic leukodystrophy |
Overview Phase | APPROVED |
Overview Status | APPROVED |
Overview Content status | Curated Core |
Overview Data Confidence | Editorial scope · Curated Core |
Overview Development Signal | Development Signal · Established |
Overview Approval status | FDA approved |
Positioning Key differentiator | Transduced microglia-like cells cross into the CNS and cross-correct neighboring cells — enzyme replacement cannot reach that compartment. |
Positioning Known limitation | Late-symptomatic disease has irreversible demyelination — timing, not biology, is the limit. |
Positioning Development positioning | First and only disease-modifying option |
Technology Vector | Lentiviral vector encoding human ARSA |
MoA Mechanism | Autologous CD34+ cells carrying a functional ARSA transgene engraft and differentiate; ARSA-secreting myeloid progeny including CNS microglia restore sulfatide degradation and halt demyelination. |
MoA Biomarker | ARSA enzyme activity in peripheral blood mononuclear cells, CSF sulfatide, vector copy number. |
Toxicology Major finding | Busulfan conditioning toxicity — cytopenia, mucositis, infection, and fertility risk. Anti-ARSA antibodies and theoretical insertional oncogenesis require long-term follow-up. |
Toxicology CRS | N/A |
Clinical Safety signal | Busulfan conditioning toxicity — cytopenia, mucositis, infection, and fertility risk. Anti-ARSA antibodies and theoretical insertional oncogenesis require long-term follow-up. |
Clinical Program phase | APPROVED |
Clinical Trial activity | 1 recruiting · 2 completed |
타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 자동 수집 갱신 권장 · 9월 2일
현재 선택: 1개 · 임상 갱신 필요 1개
프로그램 상세에서 관심 등록 후 2개 이상 모으면 여기서 한 번에 비교할 수 있습니다.
표가 넓으면 좌우로 스크롤하세요. 핵심 비교 모드에서는 중요 항목만 표시됩니다.
| 항목 | atidarsagene autotemcel (Lenmeldy, OTL-200, Libmeldy, arsa-cel) Orchard Therapeutics (Kyowa Kirin)·ARSA (arylsulfatase A) gene 3 trials |
|---|---|
Overview Program | Lenmeldy (atidarsagene autotemcel) |
Overview Company | Orchard Therapeutics (Kyowa Kirin) |
Overview Modality | CGT |
Overview Target | ARSA (arylsulfatase A) gene |
Overview Indication | Pre-symptomatic and early-symptomatic metachromatic leukodystrophy |
Overview Phase | APPROVED |
Overview Status | APPROVED |
Overview Content status | Curated Core |
Overview Data Confidence | Editorial scope · Curated Core |
Overview Development Signal | Development Signal · Established |
Overview Approval status | FDA approved |
Positioning Key differentiator | Transduced microglia-like cells cross into the CNS and cross-correct neighboring cells — enzyme replacement cannot reach that compartment. |
Positioning Known limitation | Late-symptomatic disease has irreversible demyelination — timing, not biology, is the limit. |
Positioning Development positioning | First and only disease-modifying option |
Technology Vector | Lentiviral vector encoding human ARSA |
MoA Mechanism | Autologous CD34+ cells carrying a functional ARSA transgene engraft and differentiate; ARSA-secreting myeloid progeny including CNS microglia restore sulfatide degradation and halt demyelination. |
MoA Biomarker | ARSA enzyme activity in peripheral blood mononuclear cells, CSF sulfatide, vector copy number. |
Toxicology Major finding | Busulfan conditioning toxicity — cytopenia, mucositis, infection, and fertility risk. Anti-ARSA antibodies and theoretical insertional oncogenesis require long-term follow-up. |
Toxicology CRS | N/A |
Clinical Safety signal | Busulfan conditioning toxicity — cytopenia, mucositis, infection, and fertility risk. Anti-ARSA antibodies and theoretical insertional oncogenesis require long-term follow-up. |
Clinical Program phase | APPROVED |
Clinical Trial activity | 1 recruiting · 2 completed |
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