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항목
CGTStandard DatabasestalePhase 2
Lentiviral vector transduced CD34+ cells (Lentiviral vector transduced CD34+ cells)
Genetix Biotherapeutics Inc.·CD3
50 trials·t½ 16.2 h
Overview
Program
Lentiviral vector transduced CD34+ cells
Overview
Company
Genetix Biotherapeutics Inc.
Overview
Modality
CGT
Overview
Target
CD3
Overview
Indication
P47-Phox, Deficiency of
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 in nonhuman primates
MoA
Mechanism
targeted CAR along with a rapamycin-activated cytokine receptor
MoA
Biomarker
CD19, CD33, and CD123-to harbor a CD34-derived hinge region that allow
PK/PD
Half-life
16.2 h
PK/PD
Species
NHP, 2 h
PK/PD
Animal (cat.)
NHP
PK/PD
Experiment
pharmacokinetic
Toxicology
Species
NHP, 2 h
Toxicology
Animal (cat.)
NHP
Toxicology
Major finding
thrombocytopenia were noted but not lymphopenia
Clinical
Safety signal
thrombocytopenia were noted but not lymphopenia
Clinical
Result source
ClinicalTrials.gov NCT02140554
Clinical
Program phase
PHASE_2
Clinical
Trial ref
NCT02140554