For two decades, lowering LDL cholesterol meant statins—daily pills that work for most but leave roughly one in five patients with muscle pain or liver enzyme changes severe enough to quit. On May 1, researchers at the University of Barcelona and Oregon Health & Science University published results on a short, engineered DNA molecule that silences the gene controlling how the liver clears LDL, cutting cholesterol by nearly 50 percent in mice after a single injection.
The molecule, HpE12, targets PCSK9—the same gene that Repatha, Praluent, and Leqvio go after, but upstream: it blocks the gene before any protein is made. The PCSK9 field, though, has not stood still. Eli Lilly paid $1.3 billion in July 2025 for Verve Therapeutics, whose base-editing drug cut LDL by 53 percent in a Phase 1b human trial. Merck's once-daily oral PCSK9 inhibitor, enlicitide, hit 57 percent LDL reduction in three Phase 3 trials, and Merck published its manufacturing method in Science on May 7. A third-generation injectable, Lerochol, won FDA approval in December 2025. HpE12 has not entered human trials. Its claimed advantages are cost and stability: the researchers say synthetic DNA strands are cheaper to synthesize than antibodies or RNA-based therapies and are not immunogenic. Those claims haven't been tested at clinical scale.
