Regardless of whether a patient has a rare genetic disorder or a common and debilitating condition, the next era of medicine seeks to treat disease at its root cause and at precisely the location in the body where it is needed. Many medicines today, whether small molecule drugs or large-molecule injections, treat symptoms rather than causes, affect undiseased cells in the body and include risk of dangerous side effects.

Disease can only truly be addressed if we know how it is caused and if we have the right tools. For the first time in history, we have enabling technologies that help us 1) understand disease at a genetic level and 2) build the appropriate tools we need to treat a particular disease at its root cause.

Within the next decade, the application of AI for RNA therapies will drive a paradigm shift toward expediting the development of programmable medicine for numerous diseases.

Machine learning allows us to identify the best biological solutions for even the most difficult-to-cure diseases by sifting through a massive amount of data at a scale that no human would ever be able to process manually. RNA therapies have extraordinary potential to cure a wide range of diseases by fixing the underlying genetic causes of disease. Until now, only a fraction of this potential has been explored. Using a simple analogy, DNA-based gene therapies, which induce permanent genetic changes, can be thought of as “hardware reformats”. Conversely, RNA editing is more akin to a “software update” and doesn’t carry with it the same risk of unintended genetic effects. Also, RNA editing does not require large and non-human proteins like CRISPR-Cas that DNA editing systems rely on, and such non-human proteins may be recognized by the immune system as foreign, triggering immune system reactions.

We started Shape Therapeutics with the vision of repairing the genetic causes of disease. In our pursuit of this vision, we are leveraging AI to build the next generation of programmable medicines.

Our platforms use interpretable and generative machine learning methods to identify which RNA medicines are most likely to be effective, safe, and easy to manufacture. The application of AI + RNA technologies enables: discovery and specific design of disruptive new programmable RNA medicines, identification of better ways to deliver those medicines to a diseased cell or tissue, and design of a manufacturing cell line to produce such medicines at any scale required. It’s fair to say, here at ShapeTX, we view AI as the connective tissue that permeates all of our technology innovation.

We’re making our technologies available to pharma innovators so they can develop new and better RNA medicines for as many patients as possible. ShapeTX is enabling programmable RNA medicine by discovering a new way of treating disease, building innovative approaches and making them available to the world.