ORN252

ORN252 is an investigational circular RNA (oRNA) therapeutic developed by Orna Therapeutics for the treatment of B-cell malignancies. It utilizes Orna's proprietary oRNA technology to encode a chimeric antigen receptor (CAR) targeting the CD19 protein, which is widely expressed on the surface of B-cell cancers. Unlike conventional CAR-T therapies that require complex ex vivo cell engineering, ORN252 is designed for direct in vivo administration via lipid nanoparticles (LNPs). This approach allows for the transient expression of the anti-CD19 CAR on the patient's own immune cells, potentially offering a more scalable and redosable alternative to cell-based therapies while maintaining high potency and stability due to the circular nature of the RNA. It is currently being evaluated in a Phase 1 study for safety and tolerability in healthy volunteers and patients with B-cell malignancies.

Other names

Anti-CD19 in-vivo CARAnti-CD-19 in-vivo CARAnti-CD 19 in-vivo CARCD19 oRNA-LNPCD-19 oRNA-LNPCD 19 oRNA-LNP