Arrhythmogenic cardiomyopathy (ACM) is a devastating inherited disorder characterized by massive cardiomyocyte loss, fibrofatty infiltration and ventricular arrhythmias, among others. Most known genetic causes of ACM involve the gene PKP2, which encodes plakophilin-2. An unmet medical need exists regarding therapies that correct this PKP2 deficiency.
Heart disease caused by damage to blood vessels is the leading cause of death worldwide. Arteries become clogged with fats and cholesterol when certain proteins in the body, known as lipoproteins, combine with and transport fats in the blood to cells. Scientists have long believed that the LDL receptor molecule was responsible for the intracellular transport of LDL. But given that some individuals lacking the LDL receptor still have high levels of LDL, questions remain about the mechanism.
Despite advances in diagnosis and treatment, cardiovascular disease remains the leading cause of death worldwide. Single-nucleotide polymorphisms (SNPs) in microRNAs are known to play important roles in acute coronary syndrome (ACS). MicroRNA 146a (miR-146a) is significantly upregulated in human atherosclerotic plaques and its circulating levels are elevated in patients with ACS, and was thus the focus of a recent study.
Lianbio Co. Ltd. gained its first Asian approval in Macau for Camzyos (mavacamten/Myokardia Inc.) for treating adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). The company in-licensed mavacamten rights from Myokardia, now a wholly owned subsidiary of Bristol Myers Squibb Co., in August 2020 for developing and commercializing mavacamten in mainland China, Hong Kong, Macau, Taiwan, Thailand and Singapore. Mavacamten was granted breakthrough therapy designation in China in February 2022 for patients with oHCM.
Acesion Pharma ApS has announced promising data from preclinical studies with AP-31969, its second-generation oral SK channel inhibitor developed for chronic oral maintenance treatment to prevent atrial fibrillation (AF) recurrence. Acesion aims to develop a novel drug for AF that solves the risk of proarrhythmia associated with existing drugs.
Nicoya Therapeutics (Shanghai) Co. Ltd. has reported the identification of compounds acting as dual endothelin ETA receptor and angiotensin AT1 receptor antagonists.
Rocket Pharmaceuticals Inc. has received IND clearance from the FDA for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM). RP-A601 offers the potential for a one-time, curative alternative to medical therapy, implantable cardioverter defibrillators and ablations.
By analyzing a cohort of adolescents that developed myocarditis or pericarditis after vaccination against SARS-CoV-2 vaccination, researchers from Yale University School of Medicine were able to pinpoint the underlying mechanism as an overly active innate immune response to the vaccine that led to broad activation of T cells and natural killer (NK) cells. Myocarditis “has been seen in other vaccine contexts, though is most common after viral infection,” Carrie Lucas told reporters at a press conference announcing the findings.
Jiangsu Chia Tai Tianqing Pharmaceutical Group Co. Ltd. and Medshine Discovery Inc. have jointly patented pyrimidine tricyclic derivative soluble guanylate cyclase (sGC) activators. They are reported to be useful for the treatment of heart failure and hypertension.
Research at Vasa Therapeutics Sp zoo has led to the identification of diazabicyclooctane derivatives acting as matrix metalloproteinase (MMP) inhibitors and thus reported to be useful for the treatment of heart failure.
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