Research shows that individuals with schizophrenia have abnormal gene expression patterns in their brains compared with people without the condition. In a study published in PLOS Biology on Jan. 24, 2023, the authors reported that many genes in brain tissue from the dorsolateral prefrontal cortex, a region involved in cognition, have expression patterns that follow a 12-hour cycle. The investigators found that these patterns were largely lost in people with schizophrenia. Read More
In a study published in the online edition of Proceedings of the National Academy of Sciences on Jan. 23, 2023, a team of scientists from Frederick National Laboratory for Cancer Research and the University of California, San Francisco (UCSF) described how neurofibromin 1 (NF1) missense mutations act in a dominant negative manner through dimerization with wild-type neurofibromin. Read More
Sickle cell disease (SCD) is autosomal recessive disorder caused by mutations in the β-globin gene, and induction of fetal γ-globin is considered an established therapeutic strategy for the treatment of this disease. A research team led by scientists at Kyorin Pharmaceutical Co. Ltd. has discovered RK-701, a small-molecule inhibitor of G9a and G9a-like protein (GLP) as a potential therapeutic agent for SCD. Read More
The FDA has cleared Tango Therapeutics Inc.'s IND application for TNG-462, a next-generation methylthioadenosine-cooperative (MTA) inhibitor of protein arginine methyl transferase 5 (PRMT5) for the treatment of cancers with methylthioadenosine phosphorylase (MTAP) deletion. Read More
Researchers from Meiji Seika Pharma Co. Ltd. and affiliated organizations reported the discovery and preclinical evaluation of a novel PD-1 agonist antibody, HM-266, being developed for the treatment of various inflammatory disorders, including autoimmune diseases. Read More
Amyotrophic lateral sclerosis (ALS) is the most frequent adult-onset motor neuron disease, and it is pathologically related with frontotemporal dementia (FTD). Genetic studies have identified C9ORF72 as a major genetic cause of ALS/FTD. Further genetic analyses and validation studies have identified some other genes associated with ALS risk, highlighting among them the NUP50 gene, which encodes nuclear pore complex protein Nup50. Read More
Korea University Research and Business Foundation (KURBF) has disclosed peptides acting as apoptosis inducers reported to be useful for the treatment of cancer. Read More
Medshine Discovery Inc. has synthesized proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moieties covalently linked to bromodomain-containing protein 4 (BRD4; HUNK1) targeting moieties through a linker reported to be useful for the treatment of cancer. Read More
Otosclerosis affects about 0.3% of population and it is among the most common cause of conductive hearing loss. Otosclerosis is highly familial, with positive family history reported in about 50% to 60% of cases. The disease is characterized by pathologic remodeling of the bone encasing the inner ear (otic capsule). Read More
UCB SA has identified imidazotriazine derivatives acting as IL-17 modulators reported to be useful for the treatment of autoimmune and inflammatory disorders. Read More
Toray Industries Inc. has divulged aminocyclohexane derivatives acting as κ-opioid receptor agonists reported to be useful for the treatment of pain. Read More
Inmune Bio Inc., together with a collaborator at University of California, Irvine School of Medicine, has shown targeting soluble TNF (sTNF) using a dominant-negative TNF (DN-TNF) biologic significantly decreased muscle damage in a murine mdx model of Duchenne muscular dystrophy (DMD) and showed a statistically significant increase in muscle regeneration. Read More
Dana-Farber Cancer Institute Inc. has described small-molecule GTPase KRAS (G12D mutant) inhibitors reported to be useful for the treatment of cancer. Read More
