Companies are deploying siRNA and antisense oligonucleotides to tackle dangerously high cholesterol driven by genetics, betting that a wider population will benefit.
How much Lp(a) circulates in a person’s bloodstream depends on their genes — diet and exercise have no impact. Targeting this molecule, therefore, could benefit a minority of patients who are born with elevated Lp(a), some of whom are otherwise healthy and with no other known cardiovascular risks. “This is like a genetic disease,” says Sekar Kathiresan, co-founder and CEO of Verve Therapeutics, who has published widely on the genetics of cardiovascular disease. Several development programs aimed at lowering Lp(a) particles are underway (Table 1).
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Sheridan, C. RNA drugs lower lipoprotein(a) and genetically driven cholesterol.
Nat Biotechnol (2022). https://doi.org/10.1038/s41587-022-01396-x
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DOI: https://doi.org/10.1038/s41587-022-01396-x
