Eli Lilly and Co. shared insights into an inhibitor targeting ANGPTL3, named solbinsiran. Solbinsiran, a N-acetylgalactosamine (GalNAc) conjugated Dicer-substrate small interfering RNA (DsiRNA), is designed to target ANGPTL3 expression in the liver.
Bitterroot Bio Inc. presented data on an anti-CD47 hybrid protein, BRB-002, tested in a murine model of atherosclerosis. BRB-002 was tested at 2.5 or 10 mg/kg i.p. 3x/week after a high-fat diet. Firstly, BRB-002 was seen to bind CD47 with high affinity in several cell lines, but not in Jurkat CD47-knockout cells.
The outcomes from two investigational CRISPR-based therapies were presented by researchers from Crispr Therapeutics at the recent American Heart Association’s scientific sessions.
While the human epididymis protein 4 (HE4) has been previously identified as a blood biomarker of fibrosis, the relationship HE4 has with other blood biomarkers has not been established.
The generation of in vitro small blood vessels mimicking the alterations of cerebral small vessel disease (CSVD) allowed a British research group to identify a way to seal leaks to treat conditions such as stroke or vascular dementia. The inhibition of matrix metalloproteinases (MMPs), enzymes that participate in the formation of new vessels, restored the normal union of the cells, preventing the permeability of the system.
A recognized link exists between oxidative stress, obesity and atrial fibrillation (AF). NADPH oxidase 2 (NOX2) serves as a significant contributor to reactive oxygen species (ROS) production in the heart, and it is known to be elevated in obese mice.
Ethris GmbH and Heqet Therapeutics srl, a company spun out last year from King’s College London, have entered into a collaboration agreement to harness the potential of non-coding RNA (ncRNA) for heart tissue regeneration following acute myocardial infarction and in heart failure.
The largest genetic analysis of abdominal aortic aneurysm (AAA) carried out to date has identified almost 100 new risk variants linked to the disorder. The study also highlighted a possible therapeutic target for this pathology that, at the moment, has no treatment. AAA affects 4% of people over 65 years of age in the U.S. and causes 41,000 deaths per year. The incidence is three to four times higher in men than in women.
Investigators from Biomarin Pharmaceutical Inc. have presented the first preclinical data for BMN-293, a novel adeno-associated virus (AAV) gene transfer vector carrying the MYBPC3 gene. MYBPC3 mutations can cause hypertrophic cardiomyopathy (HCM), a heart medical condition characterized by an abnormally thick myocardium, which makes it more difficult for the heart to pump blood.
Heterozygous familial hypercholesterolemia (HeFH) is caused by mutations in the LDL receptor gene, resulting in unusually high levels of low-density lipoprotein (LDL-C) in serum. Researchers from Epigenic Therapeutics Co. Ltd. presented the discovery of an epigenetic modulation therapeutic, EPI-001, for HeFH.
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