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타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-5 · 마지막 9월 2일

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프로그램 선택

API CSV14행 · 1개 프로그램
OverviewToxicologyClinical

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항목
CGTStandard DatabasestalePhase 2
OTL-200 Gene Therapy (OTL-200 Gene Therapy)
Orchard Therapeutics·Lysosomal Storage Disease
3 trials
Overview
Program
OTL-200 Gene Therapy
Overview
Company
Orchard Therapeutics
Overview
Modality
CGT
Overview
Target
Lysosomal Storage Disease
Overview
Indication
Lysosomal Storage Disease; Metachromatic Leukodystrophy
Overview
Phase
PHASE_2
Overview
Status
ACTIVE
Overview
Content status
Standard Database
Overview
Data Confidence
Data Confidence · Medium
Overview
Development Signal
Development Signal · Emerging
Overview
Approval status
Investigational
Toxicology
Major finding
A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel.. To understand the benefit-risk profile for historical and current treatments for MLD. A systematic review was conducted on the effectiveness, safety, and costs of MLD treatments: allogeneic haematopoietic stem cell t…
Clinical
Safety signal
A systematic review of clinical effectiveness and safety for historical and current treatment options for metachromatic leukodystrophy in children, including atidarsagene autotemcel.. To understand the benefit-risk profile for historical and current treatments for MLD. A systematic review was conducted on the effectiveness, safety, and costs of MLD treatments: allogeneic haematopoietic stem cell t…
Clinical
Program phase
PHASE_2