Barth syndrome is a rare genetic disorder caused by mutations in the TAZ gene, which encodes an enzyme essential for remodeling cardiolipin, critical for mitochondrial function. A recent study published in Nature identified the enzyme ABHD18 as a candidate deacylase in the cardiolipin biosynthesis pathway and a potential therapeutic target for this syndrome.
Congruence Therapeutics Inc. has closed a $32 million financing round to advance its pipeline of small-molecule correctors for diseases of protein misfolding.
Researchers from Mount Sinai Center for Translational Medicine and Pharmacology at Icahn School of Medicine at Mount Sinai and colleagues have developed a therapeutic humanized antibody that blocks the action of follicle-stimulating hormone (FSH), a pituitary hormone previously thought to only play a role in fertility.
Rallybio Corp. has received an equity milestone payment of $12.5 million from Recursion Pharmaceuticals Inc. triggered by the initiation of additional preclinical studies for REV-102, an investigational oral ENPP1 inhibitor.
Diabetic nephropathy (DN) is a complication of diabetes and a leading cause of end-stage renal disease globally, with a rate of about 40% in patients with diabetes and limited access to therapeutic options.
Editas Medicine Inc. has nominated a lead in vivo development candidate, EDIT-401, a potential one-time therapy designed to significantly reduce LDL cholesterol (LDL-C) levels. The in vivo gene editing medicine is designed to treat hyperlipidemia by directly editing the LDLR gene to increase LDLR protein expression and reduce LDL-C levels.
Medipal Holdings Corp. and JCR Pharmaceuticals Co. Ltd. have signed an exclusive global licensing deal and a co-development and commercialization partnership in Japan for JR-479, an investigational therapy for the lysosomal storage disorder GM2 gangliosidosis.
Replicate Bioscience Inc. and Novo Nordisk A/S have entered into a multiyear research collaboration that will leverage Replicate’s novel self-replicating RNA (srRNA) platform to develop new therapeutic candidates to treat obesity, type 2 diabetes and other cardiometabolic diseases.
The signaling of ephrin B (EphB) has been shown to be involved in the progression of metabolic disorders, among others. In this context, researchers from Texas Tech University Health Sciences Center have developed a pan-EphB inhibitor – STA-013 – for the treatment of metabolic syndrome.
Hinova Pharmaceuticals Inc. has divulged glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, obesity, hepatic steatosis and polycystic ovary syndrome.
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