Acute pancreatitis took center stage as Wall Street took heed of phase III data from Ionis Pharmaceuticals Inc. with olezarsen for severe hypertriglyceridemia (sHTG), while another player in the space, Arrowhead Pharmaceuticals Inc., signed a sizeable deal in a separate therapeutic area with Novartis AG.
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.
To strengthen its development efforts, Novo Nordisk A/S will collaborate in a deal that could bring Replicate Bioscience Inc. about $550 million. In return, Novo is getting an exclusive, worldwide license to use Replicate’s self-replicating RNA (srRNA) platform to develop the candidates. The two are aiming at targets in cardiometabolic diseases that include treatments for obesity and type 2 diabetes.
Leal Therapeutics Inc. is taking advantage of new genetic data that suggest products from metabolic pathways are involved in psychiatric and neurodegenerative diseases.
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.
A few years after it was founded with the aim of taking RNA therapies to the next level, Arnatar Therapeutics Inc. emerged from stealth, disclosing a $52 million series A round raised in 2024 as well as U.S. FDA orphan and rare pediatric disease designations for ART-4, an antisense oligonucleotide candidate targeting the root cause of Alagille syndrome.
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