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.
Inhibiting the sodium-hydrogen exchanger 1 (NHE1), which simultaneously imports Na+ and exports H+ in cardiac cells, shows therapeutic potential against heart failure.
Nuevocor Pte. Ltd. has closed a $45 million series B, enabling it to move lead gene therapy NVC-001 into the clinic in the treatment of an inherited form of cardiomyopathy.
Chronic hypertension in pulmonary arteries leads to their remodeling, making them increasingly resistant and potentially progressing to right heart failure and premature death. Despite advances in vasodilator therapies, the prognosis of individuals with pulmonary arterial hypertension has not improved substantially in the past 20 years.
Mammoth Biosciences Inc. has nominated its first clinical development candidate – MB-111 – a potential one-time treatment for patients with very high triglycerides, including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia. IND-enabling studies are on track to begin this year.
Researchers have identified glutathione peroxidase 4 (GPX4) as a therapeutic target in abdominal aortic aneurysm, a vascular disease characterized by permanent and focal dilatation of the abdominal aorta, with a mortality rate of up to 85% in case of rupture.
A recent study by researchers from Texas A&M University presented a new vaccine designed to target the ligand-binding domain of the serotonin 2A receptor (5-HT2AR), which resides in the second extracellular loop (EL2) and was previously identified as the key region for receptor activation. The new candidate, called EL2-5HTVac, was shown to provide a long-lasting and selective therapeutic approach to avoid increased bleeding risk complications.
Rona Therapeutics Co. Ltd. has obtained IND clearance from China′s National Medical Products Administration (NMPA) for RN-1871, a small interfering RNA (siRNA) drug targeting angiotensinogen (AGT) for hypertension.
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