The concept of directly editing genes in humans could seem like it exists only in science fiction, but for a couple of years, it’s been science fact.

Base editing is a new technology with the potential to edit genes in the DNA of intact cells, and now a company called Verve Therapeutics has received clearance from the New Zealand Regulatory Authorities to test base editing therapy in humans.

Verve will begin clinical trials for its gene-editing programme for patients with HeFH – a common genetic disease that causes high cholesterol.

Verve is the third biotech to test gene-editing technology; however, unlike Intellia and Editas Medicine, Verve’s program uses base editing, which has been described as using a pencil and eraser to make a change without cutting the DNA. Most previous editing therapies have been ex vivo; however, as Verve’s therapy targets the liver, this isn’t possible.

Verve’s preclinical data showed that their treatment can lower blood LDL cholesterol by 70% and indicated durability for 20 months.

Verve aims to get clearance to begin clinical trials in the UK and US later this year and to have an initial clinical readout from New Zealand next year.

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