Orphan drug status for PRGN-3006 UltraCAR-T to treat AML

March 6, 2020 0 By FM

Precigen announced that the US FDA has granted orphan drug designation (ODD) to PRGN-3006, a first-in-class investigational therapy using the company’s non-viral UltraCAR-T therapeutic platform for patients with relapsed or refractory acute myeloid leukaemia (AML). 

PRGN-3006 utilises Precigen’s UltraCAR-T therapeutic platform, which eliminates ex vivo expansion, reduces manufacturing time and provides the ability to administer CAR-T therapy to patients only one day after non-viral gene transfer at the cancer centre. 

PRGN-3006 UltraCAR-T is a multigenic CAR-T cell treatment utilising advanced non-viral gene delivery system to co-express a chimeric antigen receptor, membrane-bound interleukin-15 (mbIL15), and a kill switch for better precision and control in targeting relapsed or refractory AML and higher risk MDS.