RNA Therapy Makes CAR-T in the Body a Reality

Orbital's circular RNA reprograms CAR-T cells in vivo, cutting costs and widening access for autoimmune and cancer care.

25 Jul 2025

Orbital Therapeutics has unveiled preclinical data suggesting its circular RNA technology could enable the creation of CAR-T cells directly inside the body, a step that could reduce costs and broaden access to the therapy.

The experimental treatment, OTX-201, uses lipid nanoparticles to deliver RNA into immune cells, reprogramming them in vivo. This avoids the standard procedure of extracting, engineering and reinfusing cells, a process that can take weeks and requires specialised facilities. In primate studies disclosed in July, OTX-201 eliminated disease-driving immune cells.

The company expects to begin clinical trials in the first half of 2026. "This may be the dawn of a new era for RNA medicines," an Orbital executive said during a presentation. Analysts noted that, if effective in humans, the method could move CAR-T therapies into community settings and open the door to wider use in autoimmune disorders.

Momentum is also evident across the industry. Thermo Fisher Scientific recently announced a partnership with Ethris, a biotech focused on stabilised mRNA, to combine manufacturing capacity with non-immunogenic RNA platforms. The deal is aimed at speeding development of in vivo RNA medicines.

Remaining questions include how to ensure safe, temporary expression of engineered cells, as well as how regulators will evaluate these new approaches. Long-term safety data are not yet available.

Even so, investor and industry interest is strong. Advocates argue that Orbital's strategy could reshape cell therapy by lowering manufacturing hurdles and accelerating treatment delivery. If successful, the approach could challenge existing business models and redefine the role of RNA-based therapeutics in oncology and beyond.