Your Daily CRISPR & Gene Editing Briefing

CTX310 Demonstrates Durable Lipid Lowering for One Year in Phase 1a Trial

• CRISPR Therapeutics announced one-year follow-up data from its Phase 1a clinical trial for CTX310, an investigational in vivo CRISPR-Cas9 gene editing therapy targeting ANGPTL3, demonstrating sustained reductions in ANGPTL3, LDL cholesterol, and triglycerides. A single infusion at the highest dose (0.8 mg/kg) led to mean reductions of 79% in ANGPTL3, 48% in triglycerides, and 53% in LDL cholesterol at one year, with no new treatment-related serious adverse events. This durable effect from a single treatment is a significant step towards addressing the lifelong adherence challenges associated with daily medications for high cardiovascular risk patients.

  HcpliveClevelandclinicGlobenewswire

• An NIH-funded research team has identified and engineered Al3Cas12f, a naturally occurring enzyme small enough to fit into adeno-associated virus (AAV) vectors for targeted delivery inside the human body. This enhanced CRISPR system dramatically improved gene-editing performance in human cells, addressing a major limitation where larger gene-editing proteins restrict clinical applications to cells modified outside the body. This breakthrough could enable broader in vivo clinical use of CRISPR technology.

  Eatg