This deep dive focuses on Verve Therapeutics - a genome engineering company targeting cholesterol-causing genes to clear arteries and put an end to heart attacks.
In the US alone, one person dies of a heart attack every 36 seconds . By the time, you are finished reading this newsletter, someone will have lost a family member and loved one.
One of the greatest risk factors for a heart attack is high cholesterol levels, which can have a genetic component. Today, we use pills and injections tolower cholesterol levels to reduce the risk of heart attacks, but these drugs often have to be taken life long.
Verve Therapeutics is developing a one-shot solution: using CRISPR to change your DNA and stop the build up of bad cholesterol.
“We’re on the cusp of potentially transforming that model to a one-and-done treatment,” says Sekar Kathiresan, chief executive officer of Verve Tx.
Right now, Verve Tx is targeting people who have already had a heart attack, but if it works to reduce low-density lipoprotein (LDL, e.g. "bad" cholesterol") they may eventually aim to give it to young people who are at a greater risk of having a heart attack as a preventative measure. (Heart attack "vaccine" anyone?! 🤯)
The physician, founder, and CEO behind Verve Tx is Sekar Kathiresan, a cardiologist and geneticist at Harvard. His work lead to the discovery of genetic mutations that caused people to have low levels of cholesterol, offering protection from heart attacks. What if we can replicate these protective mutations in people who weren't necessarily born with them and in so doing turn off cholesterol-raising genes to offer protection against heart attacks.
How CRISPR is used to potentially prevent heart attacks:
The strategy uses CRISPR base editors, which are engineered versions of traditional CRISPR systems that are capable of making specific, localized, and small changes to the DNA without creating DNA breaks (which can pose a safety issue). The base editor is encased within a lipid nanoparticle that is later injected into the body. The nanoparticle travels around and eventually makes its way to the liver (where cholesterol is produced) and delivers the base editor. The base editor protein is designed to target one of two important genes, PCSK9 or ANGPTL3, because modifications in these genes were previously shown to reduce cholesterol levels.
The work showed encouraging signs in non-human primate models, cutting bad cholesterol levels by a whopping 59% and sustaining that effect up to 8 months later. Read the full study in Nature.
When should we expect this therapy to become available?
It'll be years before there is enough evidence of the drug's safety and efficacy toconsider seeking approval from regulators, and even then the challenge with convincing insurers it's worth covering when it will almost certainly be way more expensive than other available options.... So far analysts forecast that Verve's therapy will cost from $50,000 to $200,000 per patient.
Despite the technical and commercial challenges, we think working toward a ♥️ attack free world is worth the investment.
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