Shape Therapeutics says its research on RNA editing technology could make it easier to repair disease-causing mutations without compromising efficiency.
Prashant Mali, the co-founder of Shape, and his colleagues claim they have engineered a “new kind of guide RNA” that can recruit the cell’s own ADARs (an enzyme naturally found in the human body) to carry out edits at a precise target RNA region.
According to Shape, ADARs have the ability to alter the RNA code and correct G-to-A mutations, which are the cause of multiple genetic disorders including Parkinson’s disease, cystic fibrosis, and alpha-1 antitrypsin deficiency.
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“These new guide RNAs are built as circular rather than linear molecules, which makes them resistant to the cell’s RNA-degrading enzymes,” David Huss chief scientific officer at Shape told us.
“This unique design allows the guide RNAs to persist for longer inside the cell and thereby, increase overall target editing efficiency.”
To prove this concept, Mali and colleagues demonstrated that the RNA editing technology can treat a mouse model of Hurler syndrome (the most severe form of mucopolysaccharidosis type 1) by correcting the disease-causing mutation in RNA.
Shape entered a multi-target collaboration and license agreement with Roche in August 2021 to develop gene therapies for specific targets in the areas of Alzheimer’s disease, rare diseases, and Parkinson’s disease.
Under the terms of the deal, Shape agreed to apply its RNA editing platform RNAfix and potentially use its AAVid technology platform for next-generation tissue-specific adeno-associated viruses (AAVs).
According to Huss, the RNA research “adds another tool to the arsenal of RNA technologies available to treat genetic diseases,” and “the partnership has been a merging of like-minded individuals with true scientific passion to create novel treatment options for neuroscience and rare disease indications.”
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