What if nature has already shown us how to reverse genetic disease?

Human cells are continuously replacing and joining RNA sequences through a process called RNA splicing. We repurpose this ubiquitous and powerful phenomenon to fix genetic mutations using our Splicing-Directed Repair™ technology.

A human approach to a human problem

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.

Our Splicing-Directed Repair™ platform is the most broadly-applicable and inherently-safe way to repair genetic mutations.

Repair any mutation type
Inherently-safe: no immunogenic proteins
Retain native gene expression levels
Address thousands of mutations with individual designs
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Editing RNA with RNA

Our Splicing-Directed Repair™ platform is composed entirely of RNA molecules. By avoiding the use of proteins, we reduce the risk of adaptive immune response.

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RNA repair vs. RNA editing

Most diseases are caused by mutations of varied size and type that are distributed across human genes. Our Splicing-Directed RepairTM platform supports the replacement of mutations spread across multiple exons with a single therapeutic design.

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Retain native gene expression patterning

The timing, location, and level of gene expression is critical to the efficacy and safety of any gene therapy. But these intricacies are lost among most gene therapies, resulting in consequences that are sometimes devastating for patients. Our Splicing-Directed RepairTM platform retains native gene expression patterning that will improve outcomes for patients and reduce translational risk.
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A discovery engine to rapidly traverse a new world of RNA therapeutics

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.

Best Uses of Our Platform

Our platform uniquely combines safety and breadth. Here are some of the best uses of our platform.

Repair thousands of mutations with a single therapeutic

We can replace multiple kilobases of a target gene and address most of the mutational spectrum with a single therapeutic design.

Goldilocks genes

Many genes require precisely-controlled levels to function correctly and safely. Our technology replicates the natural levels and location of target genes.

Non-immunogenic and redosable
With the right delivery vector, our technology can be redosed indefinitely without inducing adaptive immune response.
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Platform Discovery Engine

Synthetic biology meets RNA

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.

Work with a team of committed scientists to maximize the impact of Splicing-Directed Repair™