RiboX Therapeutics Announces FDA IND Clearance for RXIM002, the first Circular RNA-Based in vivo CAR Therapy for Autoimmune Cytopenias

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 today 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.

About RXIM002 RXIM002 is a first-in-class, tLNP-delivered circRNA therapy encoding an anti-CD19 CAR. It's designed to generate functional CAR T cells in vivo to achieve durable, non-integrating CAR-expression. By inducing profound depletion of pathogenic B cells, RXIM002 promotes immune reset and sustained disease remission, offering a potentially transformative, off-the-shelf solution for patients with B-cell-driven autoimmune diseases.

About POPULUS-1 Study POPULUS-1 is a Phase 1 clinical trial evaluating the safety, pharmacokinetics, pharmacodynamics, and early efficacy of RXIM002 in patients with relapsed or refractory autoimmune cytopenias. The study will initially enroll partici...