Drug intelligence / Profile preview

PACE CART19

Development stage
Phase 1
Lead developer
University of Pennsylvania
Modality
CRISPR-Cas9 → CRISPR Systems → Programmable Nucleases → Gene Editing → Gene Therapies, CAR-T Cells → Engineered T Cells → Adoptive Cell Transfer → Cell Therapies
Administration
Intravenous
01

Overview

PACE CART19 is an investigational, universal allogeneic CAR-T cell therapy developed by the University of Pennsylvania. It consists of pre-manufactured T cells from healthy donors that are genetically engineered using CRISPR to eliminate endogenous T-cell receptor (TCR), HLA class I and II molecules, and to express a chimeric antigen receptor (CAR) targeting CD19. The gene edits include knockout of beta-2 microglobulin (B2M), CIITA, and the TCR alpha chain to reduce immunogenicity and prevent graft-versus-host disease. Upon administration, these edited CAR-T cells recognize and bind to CD19-expressing tumor cells, leading to targeted cytotoxicity against malignant B-cells. The primary indications explored were relapsed or refractory CD19+ leukemias and lymphomas[1][2][4].

Other names
allogeneic CRISPR-edited anti-CD19 CAR T cells PACE CART19
02

Targets

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