Our Science

People and patients drive our science

RaNA is dedicated to advancing the treatment paradigm by spearheading the discovery of revolutionary RNA-targeted medicines that selectively upregulate gene expression within cells in the body. Our approach has broad therapeutic potential, opening up a vast number of hard-to-treat disease targets with significant need

Transcriptional Activation

Transcription is the first step of gene expression when DNA is copied into messenger RNA (mRNA). During this process, RaNA’s oligonucleotides, are designed to block long non-coding RNA (lncRNA) from recruiting complexes known as chromatin modifiers, which can suppress the expression of certain genes.

What’s so important about IncRNA?

  • Strong emerging evidence that lncRNA is associated with disease1
  • Vast majority of the genome is transcribed as lncRNA2,3
  • lncRNA selectively modulates gene expression

By blocking the interaction between lncRNA and chromatin modifiers, transcription is activated, resulting in gene upregulation or expression. The result? The production of proteins is increased, in turn treating or preventing disease.

 See this approach in action in Spinal Muscular Atrophy.

Post-Transcriptional Activation

During post-transcription – after DNA is copied into mRNA but before translation – mRNA decay occurs naturally within our cells. Half-lives of mRNA limit the amount of therapeutic protein available in our bodies.

Half Life Distribution

In patients with certain diseases, this presents an opportunity. RaNA’s oligonucleotides are designed to stabilize the structure of mRNA, increasing its half-life, thus increasing protein levels, which are important in treating and preventing disease.

See this approach in action in Friedreich’s Ataxia.

  1. Genome-wide identification of polycomb-associated RNAs by RIP-seq. Mol Cell. 2010 Dec 22;40(6):939-53
  2. Modular regulatory principles of large non-coding RNAs. Nature. 2012 Feb 15;482(7385):339-46
  3. Long non-coding RNAs: insights into functions. Nat Rev Genet. 2009 Mar;10(3):155-9

We are looking for partners to help us realize the full potential of our science.