Ribox Therapeutics obtained FDA IND clearance for RXIM-002, a circular RNA (circRNA)-based in vivo CAR-T approach delivered by a targeted lipid nanoparticle. The company will initiate a Phase I trial in autoimmune cytopenias, including relapsed or refractory immune thrombocytopenia. This IND move reflects ongoing efforts to translate “in vivo CAR-T” concepts that aim to generate CAR-like activity directly in the patient rather than manufacturing personalized ex vivo products. On gene editing safety, reports emerged of another gene editing death in China—this time a boy treated with Huidagene Therapeutics’ HG-302 for Duchenne muscular dystrophy—after a separate gene editing death in the country earlier. While details of causality were not included in the provided excerpt, the update amplifies scrutiny on clinical safeguards for in vivo and genome editing platforms. Together, the FDA clearance and the safety headlines show the sector’s balancing act: accelerating first-in-human innovation while maintaining rigorous monitoring and risk controls as therapies expand across indications and modalities.