
One-shot gene-editing therapy reduces LDL by up to 62% in early trial
In a small Phase I study, Verve Therapeutics’ VERVE-102, a one-time mRNA-based gene-editing therapy targeting PCSK9, reduced LDL by up to 62% in the highest-dose group among 35 participants, with no serious safety events aside from a transient mild rise in a liver enzyme; results suggest durable LDL-C lowering, but larger and longer trials are needed for confirmation, and the FDA has granted Fast Track designation.





