Novel nicotinamide phosphoribosyltransferase (NAMPT) positive allosteric modulators (N-PAMs) have been discovered and evaluated by University of Illinois and University of Arizona investigators.
Many patients with cancer in advanced stages develop cachexia, a metabolic disorder characterized by systemic weight loss and muscle and adipose tissue wasting.
The EMA has awarded orphan drug designation to GC Biopharma Corp.’s intracerebroventricular enzyme replacement therapy (ERT) candidate, GC-1130A, for mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome type A), developed in collaboration with Novel Pharma Inc.
Using interactions between viral peptides and human proteins as a starting point, researchers from Enyo Pharma Inc., the University of Lyon and other institutions were able to bootstrap themselves to a mitochondria-targeting small molecule that showed activity in a mouse model of nonalcoholic steatohepatitis (NASH) with chronic kidney disease.
Korro Bio Inc. has highlighted new data for KRRO-110, its first development candidate for the potential treatment of α-1 antitrypsin deficiency (AATD), an inherited genetic disorder caused by single nucleotide variants in the SERPINA1 gene.
E-Therapeutics plc has offered a pipeline update, following the nomination of novel target genes, which have yielded promising results in preclinical studies.
Investors bailed on many med-tech companies last year, fearing that the frenzy surrounding GLP-1 agonists would tank companies in the weight-loss, diabetes and orthopedics segments. Their concerns now appear overblown in many instances, with some of the most directly affected businesses reporting a “rising tide” associated with an increased focus on obesity treatment that has lifted their boats rather than sinking them.
Researchers at Myrobalan Therapeutics Inc. and Myrobalan Therapeutics Nanjing Co. Ltd. have divulged macrophage colony-stimulating factor 1 receptor (CSF-1R; CD115; c-Fms) inhibitors reported to be useful for the treatment of cancer, autoimmune diseases, neurodegeneration, obesity, metabolic diseases, and inflammatory disorders.
Announcing two licensing deals with Swiss pharma giant Novartis AG, Shanghai-based Argo Biopharmaceutical Co. Ltd. said on Jan. 7 that it stands to gain up to $4.165 billion for two of its cardiovascular assets combined. Marking the “first significant overseas out-licensing transaction in the RNAi field from a Chinese biotech company,” the deal includes an up-front payment of $185 million from Novartis to Argo.