The U.S. Food and Drug Administration (FDA) has cleared Precigen’s Investigational New Drug (IND) application for PRGN-3007, a multigenic autologous chimeric antigen receptor (CAR) T-cell therapy.
This decision allows the company to initiate a phase I/Ib clinical trial evaluating PRGN-3007 in advanced receptor tyrosine kinase-like orphan receptor 1-positive (ROR1+) hematologic malignancies and solid tumors. The hematologic arm of the study will target patients with relapsed or refractory chronic lymphocytic leukemia (CLL), mantle cell lymphoma (MCL), acute lymphocytic leukemia (ALL), and diffuse large B-cell lymphoma (DLBCL).
ROR1 is overexpressed in CLL, MCL, ALL, and DLBCL, as well as in various solid tumors. PRGN-3007 uses a single multicistronic transposon plasmid to simultaneously express a CAR targeting ROR1, membrane-bound interleukin–15, a kill switch, and a mechanism for the intrinsic blockade of PD-1 gene expression. According to a company press release, PRGN-3007’s design, “where the blockade of PD-1 expression is intrinsic and localized to UltraCAR-T cells, is aimed at avoiding systemic toxicity and the high cost of checkpoint inhibitors by eliminating the need for combination treatment.”