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타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-4 · 마지막 9월 2일

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프로그램 선택

API CSV14행 · 1개 프로그램
OverviewToxicologyClinical

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항목
CGTStandard DatabasestalePhase 2
CRISPR_SCD001 (CRISPR_SCD001)
Mark Walters, MD·Sickle Cell Disease
1 trials
Overview
Program
CRISPR_SCD001
Overview
Company
Mark Walters, MD
Overview
Modality
CGT
Overview
Target
Sickle Cell Disease
Overview
Indication
Sickle Cell Disease
Overview
Phase
PHASE_2
Overview
Status
RECRUITING
Overview
Content status
Standard Database
Overview
Data Confidence
Data Confidence · Medium
Overview
Development Signal
Development Signal · Emerging
Overview
Approval status
Investigational
Toxicology
Major finding
To pursue gene therapy or not? Is it feasible after graft failure in allogeneic hematopoietic cell transplant recipients.. Two autologous hematopoietic stem cell (HSC)-based gene therapies (GTs) are now commercially available for severe sickle cell disease and transfusion-dependent β-thalassemia. However, the safety and efficacy of a subsequent autologous HSC-based GT after graft failure wit…
Clinical
Safety signal
To pursue gene therapy or not? Is it feasible after graft failure in allogeneic hematopoietic cell transplant recipients.. Two autologous hematopoietic stem cell (HSC)-based gene therapies (GTs) are now commercially available for severe sickle cell disease and transfusion-dependent β-thalassemia. However, the safety and efficacy of a subsequent autologous HSC-based GT after graft failure wit…
Clinical
Program phase
PHASE_2