Our first wave of in vivo gene editing programs aims to provide one-time treatments that could permanently reduce the burden of heart disease
Extending the frontier of gene editing with in vivo approaches
We are building on the expertise we have gained through our ex vivo programs to advance our portfolio of in vivo programs across both rare and common diseases. We believe that delivering gene editing cargo either systemically or into specific target organs in the body has the potential to address a variety of diseases.
Non-viral and viral approaches to in vivo delivery
Currently, several methods exist to deliver DNA or RNA to cells inside the body, which we can adapt to deliver gene editing components. These methods fall into two broad categories: non-viral and viral. We are developing therapeutic programs based on technologies in both these areas.
Non-viral: Our efforts into non-viral delivery methods have focused on lipid nanoparticles (LNPs), which predominantly target the liver. We can encapsulate messenger RNA (mRNA) encoding Cas9 and guide RNA into LNPs that are designed to shuttle these components to the liver.
Viral: For other organ systems, including the muscle, lung and central nervous system, we have emphasized viral delivery, primarily using adeno-associated viral (AAV) vectors. These vectors are designed to deliver DNA encoding for Cas9 and guide RNAs into specific tissues of the body.
Our first in vivo programs target validated genes for reducing the risk of heart disease. These programs use lipid nanoparticles (LNPs) to deliver Cas9 mRNA and gRNA to the liver with the goal of reducing expression of genes that have a known association with cardiovascular disease.
To learn more about the full range of investigational therapies we are developing, visit our pipeline page.
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