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항목
CGTCurated CoreFDAApproved
tisagenlecleucel (Kymriah, tisa-cel)
Novartis·CD19
48 trials
Overview
Program
Kymriah (tisagenlecleucel)
Overview
Company
Novartis
Overview
Modality
CGT
Overview
Target
CD19
Overview
Indication
ALL, DLBCL, FL
Overview
Phase
APPROVED
Overview
Status
APPROVED
Overview
Content status
Curated Core
Overview
Data Confidence
Data Confidence · High
Overview
Development Signal
Development Signal · Established
Overview
Approval status
FDA approved
Positioning
Key differentiator
4-1BB persistence profile
Positioning
Known limitation
downregulation of naïve T-cell-associated genes (SELL and CD28)
Technology
Vector
Lentivirus
MoA
Mechanism
Autologous T cells transduced with lentiviral vector encoding CD19-specific CAR with 4-1BB and CD3ζ signaling domains. Engages CD19+ B cells leading to proliferation and cytotoxicity.
MoA
Biomarker
CD19; measurable residual disease in ALL.
PK/PD
Species
NHP, CAR T transgene persistence, B-cell aplasia
PK/PD
Animal (cat.)
NHP, In vitro
PK/PD
Experiment
pharmacodynamic
Toxicology
Species
NHP, Mouse
Toxicology
Animal (cat.)
Human
Toxicology
Major finding
CRS and neurological events; hypogammaglobulinemia from B-cell aplasia.
Toxicology
CRS
CRS ~58–79% in ALL; Grade ≥3 CRS managed with tocilizumab per protocol
Clinical
Safety signal
CRS and neurological events; hypogammaglobulinemia from B-cell aplasia.
Clinical
Reported OS
0
Clinical
Result source
ClinicalTrials.gov NCT04225676
Clinical
Program phase
APPROVED
Clinical
Trial activity
No active/completed counts
Clinical
Trial ref
NCT04225676