Definition

A preparation of autologous T lymphocytes that have been genetically modified to express a chimeric antigen receptor (CAR) specific for glypican-3 (GPC3) and glutamic-oxaloacetic transaminase 2 (GOT2) transgene, with potential immunostimulating and antineoplastic activities. Upon administration, autologous anti-GPC3 CAR T cells BOXR1030 specifically targets and binds to GPC3-expressing tumor cells, resulting in tumor cell lysis. GPC3, a heparan sulfate proteoglycan and a member of the glypican family, is overexpressed on certain tumor cell types while minimally expressed on normal, healthy cells; GPC3 plays an important role in cellular proliferation and differentiation. The inclusion of GOT2, a mitochondrial enzyme that plays an important role in cellular metabolism, may improve T-cell metabolic function and anti-tumor activity.