A preclinical study presented at the 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT), held in Seville Oct. 7-10, showed a new epigenetic editing technology that enables durable gene silencing using ELXRs, short for Epigenetic Long-Term X-Repressors. With this approach, scientists at Scribe Therapeutics Inc. successfully inhibited the expression of the PCSK9 gene, a key regulator of cholesterol metabolism, in human cells, mice and nonhuman primates.
When the levels of LDL cholesterol are elevated, there is increased risk for atherosclerotic cardiovascular disease. Lowering these levels contributes to decreasing the risk of major adverse cardiovascular events. EDIT-401 is a CRISPR-based LDL cholesterol-lowering gene-editing approach developed by Editas Medicine Inc.
The FDA has cleared AD-NP1, a drug developed by University of California, Los Angeles (UCLA) for heart tissue regeneration, to enter clinical trials. The monoclonal antibody blocks the production of ENPP1 protein, which was found to interfere with healing after a heart attack.
Astrazeneca AB has patented new proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitors reported to be useful for the treatment of cardiovascular disorders.
Hypertension is a frequent chronic condition commonly affecting the elderly, but its incidence is increasing among younger individuals. The estimates predict that 1.28 billion individuals aged 30-79 are currently living with hypertension worldwide. Despite the availability of effective treatments, monotherapy often leads to resistance and the management of adverse effects remains cumbersome.
At the recent European Respiratory Society meeting, researchers from 35Pharma Inc. presented data on HS-235, an activin receptor inhibitor designed to neutralize activins and growth differentiation factors (GDFs). Disorders such as heart failure (HF) and pulmonary hypertension (PH) are associated with dysregulated activin and GDFs without neutralizing bone morphogenetic proteins such as BMP-9 and BMP-10, which play key roles in lymphatic and vascular homeostasis.
The University of California has synthesized proprotein convertase subtilisin/kexin-type 9 (PCSK9) inhibitors reported to be useful for the treatment of cancer, atherosclerosis and stroke.
Many patients who survive myocardial infarction go on to experience heart failure. Researchers at Qilu Hospital of Shandong University found that bone morphogenetic protein 1 (BMP1) was upregulated in patients after myocardial infarction, as well as in a mouse model of the same condition.
CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials in China with SYH-2070 injection, a double-stranded small interfering RNA (siRNA) drug for the treatment of hypertriglyceridemia or mixed hyperlipidemia.
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