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API CSV23행 · 1개 프로그램
OverviewTechnologyMoAPK/PDToxicologyClinical

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항목
CGTStandard DatabasestalePhase 2
transduced CD34+ hematopoietic stem cell (transduced CD34+ hematopoietic stem cell)
Genetix Biotherapeutics Inc.·CD3
48 trials
Overview
Program
transduced CD34+ hematopoietic stem cell
Overview
Company
Genetix Biotherapeutics Inc.
Overview
Modality
CGT
Overview
Target
CD3
Overview
Indication
Metachromatic Leukodystrophy; Adrenoleukodystrophy
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
Technology
Vector
lentiviral vector and used to transduce hematopoietic stem and progenitor cells (HSPCs) fr
MoA
Biomarker
CD19CAR-transduced HSC-T cells demonstrated high
PK/PD
Species
Mouse
PK/PD
Animal (cat.)
Mouse
PK/PD
Experiment
Pharmacokinetic
Toxicology
Species
Mouse
Toxicology
Animal (cat.)
Mouse
Toxicology
Major finding
thrombocytopenia was 1 to 7 days
Clinical
Safety signal
thrombocytopenia was 1 to 7 days
Clinical
Result source
ClinicalTrials.gov NCT02140554
Clinical
Program phase
PHASE_2
Clinical
Trial ref
NCT02140554
transduced CD34+ hematopoietic stem cell 비교 | 바이오정보모아