Our DNA is the basis of the complex and elegant system that creates life, and the emergence of tools to manipulate and alter DNA are opening new frontiers of medicine. As a result, gene therapies have the power to address a wide range of human diseases.
Some of the tools being deployed as DNA therapies, such as CRISPR/Cas9, have made a strong impact on the public conscience. However, other powerful techniques and modalities are joining the forefront. We have witnessed the potential of RNA technology to fight infectious diseases in the form of COVID-19 vaccines, but this represents just a fraction of RNA’s potential for therapeutic opportunities.
Unlike DNA editing, which makes permanent changes to the DNA contained within the nucleus of our cells, RNA editing corrects the faulty instruction without changing the underlying blueprint, DNA.
At ShapeTX, we seek to leverage this modality as a safer, less complex, and more efficient treatment for most genetic disorders. We have the ability to pair an optimal payload with a particular disease based on its specific genetic cause and phenotype. We think of this approach as accessing our RNA toolbox for selection of the appropriate tool that best addresses a given genetic disease. Our RNA tools include: RNAfix®, which uses a guide RNA to redirect a native human enzyme (ADAR) for precise and efficient editing of RNA implicated in genetic diseases; RNAskipTM, which targets premature stop codons using engineered tRNA; and RNAswapTM, a next-generation gene replacement technology.
We believe RNA will be the foundation for the next era of medicine. At ShapeTX, we are investing in payload, delivery, and manufacturing innovations, and harnessing AI capabilities to ensure that we are exploring and contextualizing the full scope of biological information available to us, as we recognize that such innovation will be critical to developing safe and effective medicines at an accessible cost, for all patients in need.