Doxorubucin is an effective therapeutic in cancer treatment, but it can cause cardiotoxicity and damage the mitochondrial machinery. A new patient-derived and mitochondria-rich human induced pluripotent stem cell derived-cardiomyocyte (hiPSC-CM) model protected by dexrazoxane was developed.
Monte Rosa Therapeutics Inc. has gained IND clearance from the FDA for MRT-8102, a NEK7-directed molecular glue degrader being developed to treat inflammatory conditions linked to NLRP3, IL-1β and IL-6 dysregulation.
Rednvia Co. Ltd. has disclosed alkaline phosphatase tissue-nonspecific isozyme (TNAP) inhibitors reported to be useful for the treatment of ectopic calcification.
Shanghai Circode Biomed Co. Ltd. has obtained IND clearance from the FDA for HM-2002 for ischemic heart disease. It previously received IND clearance in China in January this year.
Atherosclerotic cardiovascular disease (ASCVD) remains a leading cause of mortality worldwide despite advances in managing classic risk factors and therapies lowering LDL-cholesterol. Inhibiting angiopoietin-like protein 4 (ANGPTL4), a key regulator of triglyceride metabolism, is emerging as a potential strategy to treat atherogenic dyslipidemia and lower ASCVD risk.
Frontera Therapeutics Inc. has developed FT-017, an adenovirus-associated vector(AAV)-based gene therapy that carries a human MYBPC3 optimized codon, for the treatment of hypertrophic cardiomyopathy (HCM).
Repair Biotechnologies Inc.’s REP-0003 has been awarded orphan drug designation by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH).
One of the main goals in the prevention of cardiovascular disorders is to maintain low-density lipoprotein cholesterol (LDL-C) at consistently low levels to ensure long-term cardiovascular protection. Investigators at Verve Therapeutics Inc. reported preclinical data on VERVE-102, a GalNAc base editing strategy designed to sustainably inactivate the PCSK9 gene and lower LDL-C in familial hypercholesterolemia.
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