Drug intelligence / Profile preview

PBLTT52CAR19

Development stage
Phase 1
Lead developer
Great Ormond Street Hospital for Children NHS Trust
Modality
CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies, Gene Therapies
Administration
Intravenous
01

Overview

PBLTT52CAR19 is an allogeneic CRISPR-Cas9 engineered CAR T-cell therapy targeting CD19. It is designed as an "off-the-shelf" alternative to autologous CAR T-cell therapies for treating B-cell malignancies. The therapy involves multiplexed DNA editing of T cell receptor α chain (TRAC) to prevent graft-versus-host disease and removal of CD52 to provide survival advantage in the presence of alemtuzumab. The CAR expression is linked to the DNA editing through incorporation of self-duplicating CRISPR guide RNA expression cassettes within the 3' long terminal repeat of a CAR19 lentiviral vector.

Other names
Allogeneic CRISPR-edited Anti-CD19 CAR T Cells PBLTT52CAR19
02

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