Nucleic acid holds the recipes for cooking up any disease-modulating protein, and by extension, potential therapies. But nucleic acid is stubborn and doesn’t like following instructions on where in the body it should make those proteins. Dr. David Giljohann and his team at Exicure have created a technology platform that brings nucleic acid in line, opening the possibility to make proteins in the body where they’re needed most.
Tell us about your research…
We are developing therapeutics for immuno-oncology, inflammatory diseases and genetic disorders based on our proprietary Spherical Nucleic Acid (SNA) technology. The digital drug design of our SNA technology enables rapid drug development across all of these therapeutic areas. The ability to apply our SNA technology locally enables more effective treatments.
Can you explain that to a non-scientist?
Nucleic acids are the building blocks of cells; together, they form the instructions for how our bodies grow and function. In many diseases, wrong instructions are being provided. Nucleic acids can be used to correct or change these instructions and therefore have high therapeutic potential for treating diseases. While scientists have understood this for decades, we’ve struggled to turn this knowledge into better therapeutics because we couldn’t deliver these nucleic acids to specific parts of the body. We’ve discovered that changing the architecture of these nucleic acids by putting them into a 3D spherical arrangement solves this problem.
We’ve discovered that changing the architecture of these nucleic acids by putting them into a 3D spherical arrangement solves this problem.
Now, we can leverage the power of nucleic acid therapeutics by placing them precisely where they need to be, whether that’s the skin for psoriasis, gut for ulcerative colitis, eye for glaucoma, lung for cystic fibrosis, brain for spinal muscular atrophy, or directly into tumors for cancer.
How could it someday impact patient lives?
While the unmet medical need in cancer is obvious, the cumulative need stemming from the 7,000 known “rare” diseases is less well understood.
While the unmet medical need in cancer is obvious, the cumulative need stemming from the 7,000 known “rare” diseases is less well understood. These diseases, which by definition impact fewer than 200,000 Americans each, collectively affect almost 10% of the population.
These diseases, which by definition impact fewer than 200,000 Americans each, collectively affect almost 10% of the population (up to 30 million Americans and 350 million people globally), half of which are children. Sadly, 95% of these diseases have no FDA-approved treatment. Patients need better therapeutics to get to them faster.