Hundreds of millions of people around the world have received mRNA vaccines for COVID-19, providing them highly effective protection from developing serious illness from SARS-CoV-2 infection. This marks the dawn of a revolutionary new technology that has been years in development, and is poised to transform the treatment of not only viral infections but also many other diseases. At the Icahn Genomics Institute (IGI), we are developing new RNA therapeutics and nanomedicines for a broad range of diseases, including vaccines to prevent different viral infections, as well as cancer vaccines and adjuvants that direct a patient’s immune system to eliminate malignant cells and put cancers in long-term remission.
Beyond vaccines, scientists at the IGI are utilizing RNA for other types of therapeutics. This includes the development of short interfering RNA (siRNA) for the treatment of genetic diseases such as the porphyrias, and antisense oligonucleotides to alter the exon usage of specific genes involved in cancers, and treatments aimed at disorders of the central nervous system, such epilepsy.
A critical factor for the current and future success of RNA therapeutics is delivery. Getting mRNAs and oligonucleotides into cells of the body requires efficient delivery vehicles and modifications. Extensive work is ongoing in the institute to develop new nanoparticle formulations for more efficient and targeted delivery of RNAs and small molecules to desired cell types and tissues. This is enabling diverse applications that include non-invasive immunoimaging, reprogramming immune cells to promote tumor immunity, and modulation of inflammatory responses.