JCXH-213

Two new startups have begun testing their in vivo CAR-T therapies for autoimmune disease in people. China-based Immorna recently treated a systemic sclerosis patient with an in vivo CAR-T therapy, and the patient’s peripheral B cells were undetectable at two weeks. It’s an early signal suggesting that the therapy removed disease-causing immune cells, but far more data are needed to show whether the treatment improves the patient’s disease long-term. Massachusetts-based Orna Therapeutics, which Eli Lilly is buying, also started recruiting for a healthy volunteer study in Australia, according to a US clinical trials database. They are the latest biotechs to test the up-and-coming modality, after Shenzhen-based MagicRNA reported results in five lupus patients last year. At least one other biotech from China, Therorna, is also studying in vivo CAR-T for autoimmune conditions in the clinic, according to ClinicalTrials.gov, along with several Chinese academic institutions. Large US pharma companies, including Eli Lilly and Bristol Myers Squibb, have bought biotechs working on mRNA-based in vivo CAR-T treatments for autoimmune conditions, but have provided few public updates on their programs. Last year, Bristol Myers bought Orbital Therapeutics for $1.5 billion. Then in February, Lilly said it was planning to buy Orna for up to $2.4 billion. Also last year, AbbVie bought Capstan as part of a $2.1 billion deal. In 2025, Capstan started a Phase 1 study in Australia for healthy volunteers as well as patients with rheumatoid arthritis and systemic lupus erythematosus. Meanwhile, Orna began a healthy volunteer study of a CD19-targeted in vivo CAR-T therapy in March. Both trials are running in Australia. Immorna said last week that the first patient received “low doses” of its mRNA-based in vivo CAR-T called JCXH‑213, which targets CD19. The patient’s “peripheral B-cell counts dropped to undetectable levels” and remained undetectable through a two-week treatment period. It appears the patient received several doses over the treatment period, but Immorna did not provide specifics. A biopsy showed B cell depletion in lymph node tissue and the patient did not experience cytokine release syndrome or liver toxicity, Immorna reported. More data are needed to see whether the treatment was effective. In MagicRNA’s case, patients saw some improvement in their lupus disease activity, but did not achieve the level of disease clearance and “immune reset” that has been seen with conventional CAR-T therapy over a three-month period. Immorna is based in Research Triangle Park in North Carolina and Hangzhou, according to its website.