RNA Clinical Discovery

The Clinical Discovery Group within the RNA Bioscience Initiative seeks to develop new RNA therapeutics for disease diagnosis and treatment.

RNAs are informative readouts of disease status: the presence or absence of particular RNAs and isoforms can be specific to particular pathologic conditions, e.g., infection, cancer, and ischemia. Because the transcriptomes of normal and diseased cell types are complex and often poorly characterized, the potential utility of RNAs as biomarkers has not been fully exploited. The advent of new high throughput technologies, especially massively parallel single-cell RNA sequencing, empowers our researchers to fill this void.

An exceptional feature of RNA therapeutics is their ability to target any RNA via complementary sequence, enabling therapies for many diseases. RNA therapies are becoming increasingly common mainstream clinical tools. The RBI recently partnered with Children's Hospital Colorado to develop antisense oligonucleotide therapies for pediatric neurogenetic diseases.

Recent RNA-based vaccines developed by Pfizer and Moderna for SARS-CoV-2 have demonstrated remarkable potency, possibly ushering in a new era of RNA-based immunological tools.

The Denver/Boulder area is a major hub of RNA biotechnology. Establishment of partnerships with local companies is a priority for the RBI Clinical Discovery Group.