New DNA therapy cuts bad cholesterol by nearly 50%
Updated
Updated · ScienceDaily · May 1
New DNA therapy cuts bad cholesterol by nearly 50%
6 articles · Updated · ScienceDaily · May 1
University of Barcelona and University of Oregon researchers said HpE12 lowered plasma PCSK9 by 50% and cholesterol by 47% three days after one injection in transgenic mice.
In HepG2 liver cells, the DNA hairpin reduced PCSK9 RNA by 74% and protein by 87%, increasing LDL receptor levels so cells removed more LDL cholesterol from blood.
Targeting PCSK9 could offer a cheaper, stable alternative to statins and some newer drugs, potentially avoiding statin-linked muscle side effects if further studies confirm the findings.
An oral pill or a DNA injection: which new therapy will win the race to replace cholesterol statins?
This therapy silences a key gene. What are the unknown long-term risks of this new cholesterol treatment?
CTX310 Phase 1 Trial Shows 49% LDL and 55% Triglyceride Reduction with Single CRISPR Infusion
Overview
CRISPR Therapeutics' CTX310, an in vivo gene-editing therapy targeting the ANGPTL3 gene, showed remarkable results in a Phase 1 trial with 15 participants. A single infusion led to a durable reduction of 49% in LDL cholesterol and 55% in triglycerides by Day 60, driven by a 73% decrease in ANGPTL3 protein at Day 30. This gene editing boosts enzymes that break down harmful lipids independently of traditional pathways, benefiting even patients on other treatments. The therapy demonstrated a favorable safety profile with no serious adverse events. While promising, long-term safety monitoring and strategies for equitable access remain essential as CTX310 advances through clinical development.