RiboX Wins FDA IND Clearance for circRNA CAR-T Therapy
RiboX Therapeutics has received U.S. Food and Drug Administration (FDA) clearance for the Investigational New Drug (IND) application for RXIM002, an investigational circular RNA (circRNA)-based in vivo CAR-T therapy.
The treatment uses a targeted lipid nanoparticle (tLNP) to deliver circRNA encoding a CD19-targeting chimeric antigen receptor (CAR) directly to T cells inside the body.
RXIM002 is being developed for autoimmune cytopenias and is designed to generate functional CAR-T cells in vivo.
This approach could reduce the need for the complex and costly ex vivo manufacturing process used in conventional CAR-T therapies.
By targeting disease-associated B cells, the treatment aims to support immune reset and potentially achieve longer-lasting remission.
Before submitting the US IND, RiboX evaluated RXIM002 in investigator-initiated trials in China involving patients with autoimmune diseases.
Follow-up remains ongoing, with some participants monitored for more than six months. Safety and early efficacy data from these studies were included in the FDA submission.
The clearance also supports an accelerated dose-titration approach and development of a subcutaneous formulation, which could enable more convenient treatment.
RiboX is advancing RXIM002 through the Phase 1 POPULUS-1 study, initially targeting patients with relapsed or refractory immune thrombocytopenia (ITP).
The trial will evaluate safety, pharmacokinetics, pharmacodynamics and early efficacy.
