A groundbreaking Phase 1 clinical trial has demonstrated the remarkable efficacy of CTX310, a CRISPR-Cas9-based therapy, in significantly reducing cholesterol and triglyceride levels. The study, conducted at the Cleveland Clinic, revealed that a single infusion of the drug led to a 52.5% decrease in LDL cholesterol and a 47.8% reduction in triglycerides one year post-administration. Targeting Lipid Metabolism Disorders The trial enrolled 15 patients suffering from lipid metabolism disorders that proved resistant to standard medical treatments. These participants received doses of CTX310 ranging from 0.1 to 0.8 mg/kg of body weight. To prepare their bodies for the gene-editing procedure, researchers administered corticosteroids and antihistamines. Mechanism of Action CTX310 functions by targeting and inactivating the ANGPTL3 gene within the liver. This gene plays a crucial role in regulating blood fat levels. By silencing the ANGPTL3 gene, the therapy effectively lowers the concentrations of LDL cholesterol and triglycerides. Both of these lipid components are well-established risk factors for cardiovascular disease.

Background:Dyslipidemia, distinctly characterized by hypertriglyceridemia and impaired high-density lipoprotein cholesterol (HDL-C) metabolism in...

Background:Dyslipidemia, distinctly characterized by hypertriglyceridemia and impaired high-density lipoprotein cholesterol (HDL-C) metabolism in...

Background:Dyslipidemia, distinctly characterized by hypertriglyceridemia and impaired high-density lipoprotein cholesterol (HDL-C) metabolism in...

Feline infectious peritonitis virus (FIPV) poses a significant threat to cats worldwide. Besides the limited development of antiviral drugs that mainly target virus proteins, concerns about resistance...

Explore curated forecasts with evidence, scenario odds, and clear timelines.

In a phase 1 basket trial, the small interfering RNA zodasiran, targeting ANGPTL3, lowered triglycerides in patients with severe hypertriglyceridemia and lowered both triglycerides and low-density lip...

Mammoth Biosciences reveals monkey data for CRISPR therapy targeting APOC3 gene to lower triglycerides, with CEO Trevor Martin planning clinical trials in 2026.

Researchers at the Medical University of South Carolina have developed a groundbreaking approach to drug discovery using human stem cell models to identify potential new therapies. Their screening process revealed a class of compounds that significantly reduce cholesterol and triglyceride levels by targeting a different pathway than traditional treatments. Scientists have discovered a potential new treatment for familial hypercholesterolemia, inspired by signs seen in the Mona Lisa.

Mammoth Biosciences reveals monkey data for CRISPR therapy targeting APOC3 gene to lower triglycerides, with CEO Trevor Martin planning clinical trials in 2026.

Autoimmunity's Hidden Trigger: Antibodies Against a Lipid Carrier A recent commentary in Clinical Chemistry highlights a novel autoimmune mechanism behind severe hypertriglyceridemia. The condition, often linked to metabolic disorders, can in rare cases be driven by autoantibodies targeting GPIHBP1, a protein crucial for transporting lipoprotein lipase. This enzyme is essential for breaking down triglyceride-rich lipoproteins in the bloodstream. The commentary synthesizes data from several cohort studies, revealing that the prevalence of these autoantibodies ranges from approximately 1% to 15% in highly selected patient groups with unexplained or severe hypertriglyceridemia.

Residual #CVD risk can persist in some patients despite intensive LDL-C reduction, suggesting the need to address other lipoprotein abnormalities. Could emerging triglyceride-lowering therapies…

A new drug can slash triglyceride levels nearly in half by targeting a genetic driver of high fat levels in the bloodstream, researchers said. The injectable drug, olezarsen, lowered triglyceride levels by 49% at the 50 milligram (mg) dose and by 53% at…

Inclusions of α-Synuclein (αSyn) characterize multiple age-related neurodegenerative diseases, including Parkinson’s disease (PD) and Multiple System Atrophy (MSA). While interactions between αSyn and...

Neuroinflammation is central to Alzheimer’s disease pathogenesis. Stephenson et al. establish that neutral lipid accumulation is necessary for microglial activation and that microglia expressing an Alzheimer’s risk gene hijack triglyceride biosynthesis to acquire a basal immune activated state. Targeting triglyceride metabolism can modulate immune reactivity and disease phenotypes.

Plozasiran, an investigational siRNA targeting hepatic apoC-III, reduces triglyceride-rich lipoproteins (TRLs). The impact of plozasiran on lipoprotei…

Hirano et al. develop a heart failure diagnosis of triglyceride deposit cardiomyovasculopathy based on defective lipolysis and show long-term survival and patient recovery after tricaprin treatment.