CAR T cells are patient-derived, genetically engineered immune cells. They are “living drugs” and constitute a milestone in modern medicine. Equipping T cells, a key cell type of the immune system, with a “chimeric antigen receptor” (CAR) enables them to specifically recognize and attack cancer cells.

CAR T cell therapy has demonstrated its potential by curing patients with otherwise untreatable blood cancers. But it still fails for most patients, often due to T cell intrinsic dysfunction. To address their current limitations and to make CAR T cells intrinsically stronger, scientists at the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences and the Medical University of Vienna have developed a new method for systematic discovery of genetic boosters of CAR T cell function.

The new study, published in Nature, introduces CELLFIE, a CAR T cell engineering and high-content CRISPR screening platform, enabling researchers to systematically modify CAR T cells and evaluate their therapeutic potential.

Oligonucleotide therapies — engineered strands of DNA or RNA — are transforming modern medicine. These cutting-edge treatments bring a new level of precision in combating disease by targeting specific genes to be silenced, activated or edited. “Nucleotide therapeutics allow us to design predictable outcomes by modifying sequences to address almost any condition,” says Peter Guterstam, product manager at biotechnology company Cytiva.

Due to an influx of research in recent years, many nucleotide-based drug candidates, including genetic therapies and vaccines for cancer and viral infections, are now in advanced clinical trial stages. “The development timeline is much quicker than we are used to,” notes Guterstam.

Significant challenges arise during development of RNA and DNA based therapies. From mRNA vaccines to gene editing, scientists are refining delivery methods, optimizing synthesis, and tackling scaling hurdles.