The FDA IND clearance supports the initiation of RiboX's Phase 1 clinical trial evaluating targeted LNP-delivered circular RNA-based in vivo CAR-T therapy for autoimmune cytopenias.

RXIM002 represents the world's first circular RNA-based in vivo CAR-T therapy achieving FDA IND clearance. RiboX expands its clinical-stage pipeline through its proprietary targeted LNP delivery platform.

RiboX Therapeutics Ltd. ("RiboX"), a global clinical-stage biotechnology company pioneering fully engineered circular RNA (circRNA) therapeutics, announced that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND) application for RXIM002. RXIM002 is the world's first circular RNA-based in vivo CAR-T therapy delivered by a targeted lipid nanoparticle (tLNP). This marks an important milestone for the field as circRNA-based in vivo CAR-T officially advancing into clinical development.

RXIM002 is an investigational product employing tLNP encapsulated circRNA that encodes a CD19‑targeting chimeric antigen receptor (CAR) for the treatment of autoimmune cytopenias. By delivering circRNA to T cells in vivo, RXIM002 enables durable CAR expression and generating functional CAR-T cells directly within the patient's body. This "in situ generation" approach fundamentally bypasses the complex, costly and time-consuming ex vivo manufacturing processes required by conventional CAR-T therapies, expanding patient access. In autoimmune diseases, deep B cell depletion driven by the in vivo generated CAR-T cells has the potential to induce immune reset and durable drug-free remission.

Prior to the IND submission, RXIM002 was evaluated in investigator-initiated trials (IITs) in China in autoimmune disease patients. Follow-up of these patients are currently ongoing, with some patients exceeding six months. RiboX submitted complete IIT data as part of the IND package to the FDA, encompassing safety and early efficacy results from all treated patients. Based on the integrity and robustness of this full dataset, the agency permitted an accelerated dose-titration scheme and granted subcutaneous formulation as part of clinical development, paving the way for potentially outpatient administration that could fundamentally transform the CAR-T treatment paradigm.

To the company's knowledge, RXIM002 is the world's first tLNP-circRNA in vivo CAR therapy that achieved FDA IND clearance. This milestone validates RiboX's proprietary tLNP platform and further reinforces the company's leadership in precision RNA therapeutics.

"The FDA IND clearance for RXIM002 is a pivotal milestone for RiboX and for the field of circRNA medicines," said Weiyi Zhang, Ph.D., Chief Executive Officer of RiboX Therapeutics. "As the first-in-class circRNA in vivo CAR-T therapy to enter the clinic, RXIM002 exemplifies our ability to translate robust pre-clinical results and rigorous early clinical evidence into an accelerated development path. This achievement brings us closer to delivering a transformative treatment option for patients living with debilitating autoimmune diseases. We are committed to advancing clinical research of RXIM002 across multiple regions worldwide to address the significant unmet needs."