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항목
CGTCurated CoreFDAApproved
lovotibeglogene autotemcel (Lyfgenia, lovo-cel, bb1111)
bluebird bio·βA-T87Q-globin (lentiviral)
2 trials
Overview
Program
Lyfgenia (lovotibeglogene autotemcel)
Overview
Company
bluebird bio
Overview
Modality
CGT
Overview
Target
βA-T87Q-globin (lentiviral)
Overview
Indication
Sickle cell disease with recurrent vaso-occlusive events
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
Gene addition of anti-sickling globin vs CRISPR HbF induction
Positioning
Known limitation
bypassing such limitations
Positioning
Development positioning
Lentiviral alternative to Casgevy in SCD
Technology
Vector
Lentiviral vector
MoA
Mechanism
Lovotibeglogene autotemcel adds functional β-globin gene via lentiviral vector to autologous CD34+ cells, producing anti-sickling HbAT87Q in sickle cell disease.
MoA
Biomarker
HbAT87Q, VOC, TCD velocities.
PK/PD
Animal (cat.)
Unknown
PK/PD
Experiment
pd
Toxicology
Major finding
Hematologic malignancy risk (boxed warning); conditioning toxicities
Clinical
Safety signal
Hematologic malignancy risk (boxed warning); conditioning toxicities
Clinical
Result source
ClinicalTrials.gov NCT02140554
Clinical
Program phase
APPROVED
Clinical
Trial activity
1 recruiting · 1 completed
Clinical
Trial ref
NCT02140554