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Gene-editing treatment permanently lowered cholesterol levels after just one infusion in recent trial
A new base-editing therapy has demonstrated the ability to permanently lower LDL cholesterol levels after a single infusion by silencing the PCSK9 gene. In the highest-dose group, LDL cholesterol fell by an average of 62 percent, with PCSK9 levels dropping by as much as 88 percent, and these reductions appeared within weeks and lasted surprisingly long. Eli Lilly plans to initiate a larger Phase 2 study for this therapy by the end of 2026.
Researchers at the San Raffaele Telethon Institute for Gene Therapy (SR-Tiget) have developed a novel SMArT platform to significantly enhance the precision and safety of CRISPR-Cas9 gene editing in human blood stem cells. This innovative strategy enriches correctly edited cells to near purity while selectively removing those with unintended genomic alterations, addressing a major safety concern for broader clinical application.
Scribe Therapeutics presented preclinical data at ASGCT 2026 on its ELXR CRISPR platform, which is designed to silence genes without permanently cutting or editing DNA. Their lead program, STX-1150, targets the PCSK9 gene for LDL cholesterol regulation, showing sustained lower cholesterol effects for nearly 18 months in non-human primates after a single treatment.
The Bottom Line
Recent advancements highlight a dual focus in gene editing: achieving durable therapeutic effects with single-dose treatments, as seen in the cholesterol-lowering therapy, and simultaneously improving the safety and precision of existing CRISPR tools to expand their clinical applicability.
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