Suzhou Puhe Biopharma Co. Ltd. has described apelin receptor (APLNR) agonists reported to be useful for the treatment of idiopathic pulmonary fibrosis, diabetes, obesity, heart failure, hypertension, pulmonary hypertension, sarcopenia and scleroderma.
Chong Kun Dang Pharmaceutical Corp. has prepared and tested glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of fibrosis, neurodegeneration, nutrition, endocrine, immunological, inflammatory, psychiatric and renal disorders, among others.
Remedium Bio Inc. has entered into a multitarget research and development collaboration with Eli Lilly & Co. to advance gene therapies for type 2 diabetes and obesity using Remedium’s Prometheus dose-adjustable gene therapy platform.
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.