RNA End-Joining Technology
A potential method of targeting bigger genes
While adeno-associated virus (AAV) vectors are the preferred delivery method for gene therapies, they can only hold genes of a certain size. This limits the genes—and thus diseases—that today’s gene therapies can target.
With our proprietary RNA end-joining platform, we are working to split large genes into multiple AAV capsids and reconstitute them once administered. This technology has the potential to unlock new possibilities for gene therapy in diseases caused by large genes that have historically not been viable targets for gene therapy.
Hear how our proprietary RNA end-joining technology is being explored in diseases caused by genes previously considered too large to address with gene therapy.
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