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타깃 · 모달리티 · 임상 근거 · 비임상 맥락으로 최대 5개 프로그램을 나란히 비교합니다. · 다음 갱신 D-6 · 마지막 9월 2일

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프로그램 선택

API CSV14행 · 1개 프로그램
OverviewToxicologyClinical

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항목
CGTStandard DatabasestalePhase 3
LentiGlobin BB305 Drug Product (LentiGlobin BB305 Drug Product)
Genetix Biotherapeutics Inc.·Beta-Thalassemia Major
7 trials
Overview
Program
LentiGlobin BB305 Drug Product
Overview
Company
Genetix Biotherapeutics Inc.
Overview
Modality
CGT
Overview
Target
Beta-Thalassemia Major
Overview
Indication
Beta-Thalassemia
Overview
Phase
PHASE_3
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
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.. Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer of a marked β-globin (β A-T87Q ) gene could substitute for long-term red-cell…
Clinical
Safety signal
Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.. Donor availability and transplantation-related risks limit the broad use of allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β-thalassemia. After previously establishing that lentiviral transfer of a marked β-globin (β A-T87Q ) gene could substitute for long-term red-cell…
Clinical
Program phase
PHASE_3