Our Approach & Strategy
Reshaping The Future Of
RNA-Based Medicines 
We are developing the leading circular RNA technology platform (oRNA®) and unprecedented lipid nanoparticle (LNP) delivery solutions – significantly expanding the therapeutic possibilities of circular RNA and pioneering the next chapter of safe and effective in vivo therapies.
Shape Matters
oRNA® is formed by self-circularization from a linear RNA precursor. This ease of production is the basis of the unmatched advantages over traditional linear mRNA therapies, which include:
- Simplified production: no caps, no tails, no modified nucleotides
- Simple purification, leading to lower risk of adverse immune reactions
- Longer RNA half-life for increased durability and stability
- Increased protein expression from our groundbreaking discoveries in IRES function
- Ease in working with RNAs in the larger size paradigm
- Easier, more straightforward formulation into lipid nanoparticles
Each segment of the oRNA® sequence is designed for a specific effect: driving circularization, encoding the therapeutic protein, and promoting increased protein expression.
Through our high throughput screening FoRCE™ platform (Formulated oRNA Cell-based Evaluation), we can evaluate thousands of oRNA variants in LNPs in a variety of cell types to identify which oRNA sequences have the ideal performance. We have led the world in adapting powerful expression elements normally found in viruses into our oRNA constructs to increase potency by orders of magnitude.
Closing The Loop On Delivery
Orna has pioneered the creation of novel LNP delivery solutions that are simple, scalable and reach parts of the body other than the liver – the holy grail of RNA delivery. Our approach includes two delivery platforms: panCAR™, which targets multiple lineages of the immune system at once, and SiTu Editing in the Marrow (STEM)™, which targets stem cells in the bone marrow.
These delivery technologies create particles that traffic to their respective sites by passive targeting rather than active targeting that uses ligands attached to the LNP surface and complicates the manufacturing process and the patient’s reaction to the therapy.
Once the oRNA® payload is released, the genetic instructions encoded in it get to work producing high amounts of therapeutic protein.
