Two RNA Startups Begin Testing In Vivo CAR-T Therapies for Autoimmune Disease

Two biotechnology companies have entered clinical trials testing revolutionary in vivo CAR-T therapies for autoimmune diseases, marking a significant milestone in personalized medicine. China-based Immorna recently treated a patient with systemic sclerosis using their RNA-based approach, joining another startup in this competitive therapeutic space.

Traditional CAR-T therapy requires extracting a patient's immune cells, genetically modifying them in laboratories, and reinfusing them—a complex, expensive process. In vivo CAR-T therapy aims to reprogram immune cells directly inside the body using RNA or other delivery methods, potentially making these treatments more accessible and affordable.

This development is particularly important for autoimmune disease patients, who often face limited treatment options and progressive symptoms. Systemic sclerosis, the condition being treated by Immorna, is a rare autoimmune disease causing skin and organ hardening with poor long-term outcomes.

The clinical entry of these RNA-based approaches represents a convergence of several cutting-edge technologies: CAR-T cell engineering, RNA therapeutics, and targeted drug delivery. If successful, these therapies could transform treatment paradigms for multiple autoimmune conditions including rheumatoid arthritis, lupus, and multiple sclerosis.

However, significant challenges remain. In vivo delivery must achieve precise targeting while avoiding off-target effects. The technology is still experimental, and safety profiles need extensive evaluation. Additionally, the article provides limited details about trial design, patient selection criteria, and preliminary results, making comprehensive assessment difficult at this early stage.