January 9, 2023

Ex Vivo to In Vivo: the Journey of Cell Therapy

What is the Next Step for CAR-T?

There are 6 currently approved ex-vivo CAR-T therapies. While CAR-Ts have the potential to be highly effective they come with a challenging manufacturing process that makes them widely inaccessible to patients. Looking to the future, in vivo CAR-Ts have the potential to eliminate this cumbersome manufacturing process and improve patient outcomes.

A handful of innovative biotech companies are utilizing different drug delivery methods to kick-start their in-vivo CAR-T technologies. One company, in particular, Capstan Therapeutics, is focused on merging mRNA technology as seen in the COVID-19 vaccines with lipid nanoparticle delivery.

CAR-T cells are normally produced outside of the body, complicating the manufacturing process. In vivo cell therapy modifies immune cells within the patient’s body with the help of technologies like mRNA, lipid nanoparticles, viral vectors, and gene editing.

Several investments and collaborations in 2021 highlighted the growing interest in the advancement of in vivo engineering. Moderna established a collaboration with gene editing company Metagenomi to combine CRISPR-based systems with mRNA and LNP technologies to develop in vivo gene editing therapeutics. Abintus Bio entered into an agreement with Memorial Sloan Kettering Cancer Center to use its signaling technology for in vivo CAR-X products. Sanofi acquired Tidal Therapeutics, a company utilizing proprietary nanoparticles to deliver mRNA to immune cells. This is only the tip of the iceberg. Several other biotechs have been operating in this space.