RNA editing holds great promise to rewrite what’s possible for patients. Since the discovery of the first generation of editing technologies, researchers have been working to overcome the inherent complexity of these initial approaches to develop viable new treatments for patients.
Our foundational platform for RNA editing – Oligonucleotide Promoted Editing of RNA (OPERA) – is inspired by the elegance and simplicity of the natural processes that many species, including humans, use to make transient, reversible changes to genetic instructions.
RNA editing enables the careful repair and modulation of disease-causing mutations and protein function — opening new possibilities for diseases where progress with existing technologies has been slow to materialize.
Our unique platform, OPERA, is designed to solve many of the challenges facing current gene therapy and gene editing approaches, including safety and flexibility of delivery mechanisms.
See our ADAR-based editing approach in action and how it offers the potential to advance genetic medicines beyond rare genetic diseases.