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.
Small conductance SK (Ca) channel blockers, particularly SK (Ca) 2.3 (SK3; SKCa3; hKCa3) channel blockers, have been described in an Acesion Pharma ApS patent as potentially useful for the treatment of arrhythmia.
Rejuvenate Bio Inc. has released new preclinical efficacy data for its gene therapy, RJB-0402, in a mouse model of arrhythmogenic cardiomyopathy (ACM), an inherited disease caused by mutations in one of several genes encoding desmosomal proteins.
Just one week after birth, the heart experiences a change in metabolism that helps it meet the high energy demand necessary to fulfill its function. This evolutionary developmental process could have medical advantages.
Evotec SE and Novo Nordisk A/S have announced the launch of Lab En2, a translational drug discovery accelerator that aims to advance early research from academic institutions into novel therapeutics.
Novo Nordisk A/S and Valo Health Inc. have entered into an agreement to discover and develop novel treatments for cardiometabolic diseases. The collaboration will leverage Valo’s Opal Computational Platform, including access to real-world patient data, artificial intelligence (AI)-enabled small-molecule discovery and Biowire human tissue modeling platform designed to speed up the discovery and development process.
Calcineurin inhibitors used in clinics – including immunosuppressants for transplant rejection and autoimmune disease treatment – may cause persistent hypertension. Calcineurin inhibitors elevate sympathetic vasomotor activity by decreasing calcineurin activity and potentiating N-methyl-D-aspartate receptor (NMDAR) activity in the hypothalamus. However, how these inhibitors promote NMDAR activity in the hypothalamic paraventricular nucleus to increase sympathetic vasomotor activity remains unknown.