Orna’s in situ CAR therapy combines oRNATM technology with proprietary LNPs to create modified immune cells within a patient. This easily redosable format would not require patient lymphodepletion and would allow for reliable dose control, overcoming barriers of existing ex vivo CAR-T therapies.
Orna has achieved non-viral delivery of a large, full-length, dystrophin-encoding RNA in human cells, as well as in vivo delivery of smaller length versions in mouse models. These data are a step on the path to delivering improved gene therapy to patients, such as those with Duchenne Muscular Dystrophy (DMD).
Orna is investigating the suitability its oRNATM technology combined with intramuscularly administered immunotropic LNPs for vaccine applications, including for COVID-19. The oRNATM half-life observed in muscle and immune cells, combined with the intramuscular administration of immunotropic lipids, suggests that oRNA-LNP technology may be beneficially applied to vaccine development.