FT819

Autologous CAR T-cell therapy has emerged as a revolutionary and potentially curative therapy for patients with certain hematologic malignancies, including refractory cancers. More recently, in a ground-breaking academic clinical study published in Nature Medicine in September 2022, an investigational autologous CD19-targeted CAR T-cell therapy was administered to patients with systemic lupus erythematosus (SLE). Rapid B-cell depletion and elimination of auto-antibody production was observed following infusion of therapy, and all patients achieved clinical remission with significant improvement in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) score. Naïve B-cell reconstitution occurred after an average time of 110 days of CAR T-cell infusion.

While most researchers and clinical investigators continue to focus on the development of autologous or donor-derived CAR T-cell therapies, we are pioneering the development of off-the-shelf CAR T-cell product candidates created from clonal master engineered iPSC lines. 

FT819 is our first investigational, off-the-shelf, iPSC-derived CAR T-cell product candidate, which incorporates a novel 1XX CAR targeting CD19 inserted into the T-cell receptor alpha constant locus. FT819 exhibits potent B-cell killing in a dose-dependent manner comparable to autologous primary CAR-T cells in an in vitro cytotoxicity assay using SLE peripheral blood mononuclear cells. Given that the targeting and rapid depletion of B cells is a common mechanism of action for the successful treatment of B cell-mediated autoimmune diseases, we have initially focused clinical development of FT819 for the treatment of moderate to severe SLE and plan to expand clinical development to certain other areas of autoimmunity.