Other genome engineering technologies rely on systems derived from other species, but the human immune system doesn’t take kindly to foreign organisms (whole or in part). Our Splicing-Directed Repair™ technology avoids these risks by using natural, pre-existing systems in human cells to repair mutations.
The potential of our platform is unbounded. And so is the parameter space we need to search to build therapeutic programs. By using the latest in synthetic biology tools informed by RNA biology, we can rapidly search this space with our proprietary pooled and arrayed screening methods.
Our platform uniquely combines safety and breadth. Here are some of the best uses of our platform.
We can replace multiple kilobases of a target gene and address most of the mutational spectrum with a single therapeutic design.
Many genes require precisely-controlled levels to function correctly and safely. Our technology replicates the natural levels and location of target genes.
How do we take decades of anecdotal knowledge in RNA processing and repurpose it to make the most powerful way to repair RNA? We combine the latest into high-throughput screening, automation, long-read sequencing, and computation to compare the activity of thousands of therapeutics at once.