Jennifer Doudna won the Nobel Prize for Chemistry in 2020 for her contribution to the path breaking gene-editing technology CRISPR. The development of the technology has been beautifully captured in Walter Isaacson’s book – The Code Breaker. While gene-editing has raised prospects of curing rare diseases, a new development bodes well for potentially curing diseases which afflicts millions, such as high cholesterol.
“A patient in New Zealand last week became the first person to be injected with a drug that is designed to powerfully and permanently lower cholesterol, ushering in a critical test of Crispr gene-editing technology.
If the drug from Verve Therapeutics works — and that’s still a big open question — it could someday be offered as a one-time shot to address the No. 1 killer in the US: heart disease.
…To bring Crispr to the masses, Verve turned to a newer variation of Crispr called “base editing.” Whereas the original gene-editing tool, Crispr-Cas9, uses a two-part system — “guide RNA” ferries an enzyme called Cas9 to a selected stretch of DNA, which the Cas9 snips out — base editing uses guide RNA alongside a different enzyme capable of changing individual DNA building blocks, or nucleotides.
…Verve plans to aim the drug at the roughly 1 million people in the US with an inherited form of dangerously high cholesterol called familial hypercholesterolemia. Long term, however, the company thinks its therapy could offer a permanent, potentially more effective alternative to the statins that millions of Americans take every day. In that distant and still theoretical future, the average person could get Verve’s one-time shot to keep cholesterol low enough to avoid heart attacks altogether.” In that distant and still theoretical future, the average person could get Verve’s one-time shot to keep cholesterol low enough to avoid heart attacks altogether.
…So far, the approach has worked remarkably well in monkeys. Now, the company will be the first to find out whether the promise of those single-letter edits carries over into humans. The trial is starting in New Zealand with 40 subjects, but Verve hopes to eventually receive permission to enroll people in the UK and the US.
When the initial swath of data from the study comes out next year, scientists and investors alike will go through it with a fine-tooth comb to understand three critical questions: How well does it work to lower cholesterol? Does cholesterol stay low forever? Are there any unexpected safety concerns?
The answers matter not only for Verve but for the millions of people who struggle to keep their cholesterol in check despite the many available drugs on the market.
But they also have broad consequences for the field. The outcome of the study could sway companies like Verve — biotech or big pharma firms that are technology agnostic and simply want to develop the best drug for a particular disease — toward a particular type of gene editor when developing future therapies. It will also raise the question of what other common diseases can be feasibly tackled with current gene-editing approaches. Another nascent effort is using Crispr to engineer stem cells that can be given to people with Type 1 diabetes to try to restore insulin production.”
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