The startup undertaking the research is Verve Therapeutics, and the company has received $58.5 million from investors. This includes a major slice from including Alphabet’s GV venture fund. Other backers are Arch Venture Partners, F-Prime Capital and Biomatics Capital. The aim of the new process is to deploy CRISPR gene editing to spread fortunate DNA variations via injections, in order to trigger small alterations to human DNA.
With these gene variations, researchers have previously determined there are certain genetic mutations which can protect people from heart disease (gene variants like PCSK9, HMGCR, and NPC1L1). Verve scientists are aiming to deploy CRISPR in order to install the variant types into other people who do not possess these specific mutations.
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CRISPR refers to ‘clustered regularly-interspaced short palindromic repeats’, and it is a type of biological cut-and-paste technology where molecular scissors can be used to make changes to DNA. The potential applications of the biotechnology include correcting genetic defects, treating and preventing the spread of diseases and improving crops.
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According to company co-founder Sekar Kathiresan, the initial aim is to use the gene-editing treatment with adults who have actually had a heart attack and want to avoid a second one. This could then be rolled out to other adults, or even used on children before heart disease is presented as a serious risk.
The company aims to use CRISPR nucleases and base editors to edit genes within the adult, which should improve lifelong lipid and metabolic status, lowering the risk of coronary artery disease.
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Looking to the future, Kathiresan states “With our therapy, we could change the treatment paradigm for heart disease from chronic care, daily pills or monthly injections to a one-and-done approach.”
