When a group of British scientists studied which proteins might be in the wrong place of the cell in amyotrophic lateral sclerosis (ALS) patients, they found hundreds of them mislocalized. Other studies had shown that TDP-43 protein was mislocalized. But it was not known that the phenomenon was widespread, and affected mRNA as well as proteins. “Our study revealed that these mislocalized proteins were heavily involved in RNA binding functions and exhibited high binding affinities to RNAs,” Rickie Patani told BioWorld. Read More
The identification of new targets in diseases of the central nervous system (CNS) such as Alzheimer’s and Parkinson’s – conditions which continue to have significant unmet needs – has taken a small step forward as one company, Violet Therapeutics Inc., plans to put $10.6 million in seed funding toward building out a pipeline based on technologies that elucidate the way cells interact amongst one another. Read More
Cessation Therapeutics Inc. has obtained IND clearance from the FDA to initiate a first-in-human trial of CSX-1004 for the prevention of fentanyl overdose. The trial in healthy volunteers is expected to begin next month. Read More
Researchers from Chongqing Medical University have reported discovering a novel cyclin-dependent kinase 7 (CDK7) inhibitor, N76-1, which they are investigating as a potential new candidate for the treatment of triple-negative breast cancer (TNBC). Read More
Researchers at Association Française Contre Les Myopathies, Consiglio Nazionale delle Ricerche, and Università degli Studi di Roma La Sapienza have disclosed peptidomimetics derived from leucyl-tRNA synthetase. Read More
Researchers from China Pharmaceutical University have disclosed the discovery and preclinical evaluation of a new receptor-interacting serine/threonine-protein kinase 2 (RIPK2) inhibitor, Zharp2-1, as a potential therapeutic candidate for the treatment of inflammatory bowel disease (IBD). Read More
A team of scientists led by The Wistar Institute has been awarded a 5-year National Cancer Institute (NCI) Program Project Grant valued at more than $12 million to explore the role of Epstein-Barr virus (EBV) in epithelial cancers. The project, which brings together scientists from The Wistar Institute and Harvard University, will focus entirely on the EBV-epithelial cancer link and look at metabolic and epigenetic vulnerabilities simultaneously. Read More
Shanghai Haiyan Pharmaceutical Technology Co. Ltd. and Yangtze River Pharmaceutical Group have synthesized macrophage colony-stimulating factor 1 receptor (CSF-1R; CD115; c-Fms) inhibitors reported to be useful for the treatment of cancer. Read More
An mRNA vaccine candidate that acts in the liver by recruiting memory T cells could be the key against malaria, according to a study in mice that demonstrated its efficacy by including a natural killer T (NKT) cell agonist. Read More
Krystal Biotech Inc. has received FDA acceptance of its IND application for lead oncology drug candidate KB-707 for the treatment of locally advanced or metastatic solid tumor malignancies. KB-707 is a modified herpes simplex virus 1 (HSV-1) vector designed to deliver genes encoding both human IL-12 and IL-2 to the tumor microenvironment and promote systemic immune-mediated tumor clearance. Read More
Astellas Pharma Inc. has divulged substituted quinolone derivates acting as acetylcholine receptor cluster inducers reported to be useful for the treatment of neuromuscular disorders. Read More
Researchers from Arizona State University presented preclinical data for the novel dual specificity tyrosine phosphorylation kinase 1a (DYRK1a) inhibitor DYR-533, being developed for the treatment of tauopathies, including Alzheimer’s disease (AD). Read More
