Base Editing Applications Tackling disease, one letter at a time
Base editors are designed to rewrite just a single “letter” with high efficiency and precision and thereby intervene at the most basic level to potentially treat a wide range of diseases. Single base changes are a versatile way of precisely changing the genome, enabling many different possible editing strategies:
Correct a single point mutation to restore the gene’s proper function
Change a base into a protective variant so the gene may prevent or modify the risk of disease
Turn on a gene that may prevent or modify a disease. This is done by editing a regulatory element responsible for gene expression
Turn off a gene that contributes to a disease. This is done by editing a regulatory element for gene expression
Edit multiple sites at the same time, combining different types of edits to help treat a disease
Leveraging clinically validated delivery modalities
Base editors can be delivered into cells using ex vivo or in vivo methods. Ex vivo means that cells are removed from the body, edited, and then returned to the body. In vivo means the base editor is delivered directly into organs of the body, such as the liver, to make edits within the target cells.
Our delivery strategy is to establish a suite of clinically validated technologies, including electroporation and nonviral and viral delivery modalities. We understand that to address the unique characteristics of each disease, we may need different technologies to deliver medicines to the relevant target organs.
We are using electroporation for delivery to blood cells and immune cells for ex vivo delivery.
Nonviral lipid nanoparticles
We are using LNPs for direct in vivo delivery to the liver initially. Our high-throughput, in vivo lipid nanoparticle screening platform may also allow us to identify lipid nanoparticles capable of carrying base editors to other organs in the body, such as the blood cells, immune cells, CNS...
We can also use nonpathogenic viruses, such as adeno-associated viruses, to transport genetic code to target cells. We are using viral vectors for direct in vivo delivery to the eye and potentially other organs in the future.
“Many existing gene editing approaches are like ‘scissors’ that cut the genome. Base editors are like ‘pencils’ that enable erasing and rewriting one letter of the genome at a time.”