A technology that combines transcriptomic data and AI enables a novel approach to drug discovery based on the state of cells, how they behave and which genes they express. The Drugreflector model, developed by scientists at Cellarity Inc., learns from gene expression profiles and predicts which compounds could induce beneficial changes in that cellular state to develop a treatment. Read More
Expedition Medicines, which Flagship Pioneering Inc. has incubated for the past three years, came out of stealth mode with a $50 million commitment from Flagship to support Expedition’s platform technology to discover small molecules that covalently bind to their target. Read More
Myocardial ischemia-reperfusion (MI/R) injury is a major contributor to heart failure. The long noncoding RNA (lncRNA) myocardial infarction-associated transcript (MIAT) has been shown to worsen cardiac damage during ischemia, making it a promising target for RNA interference. Read More
Esophageal cancer is among the most lethal cancers with a high mortality rate worldwide. Chinese researchers have explored the potential link between long intergenic non-protein coding RNA 1354 (LINC01354) and esophageal cancer. Read More
Nippon Shinyaku Co. Ltd. has disclosed compounds inhibiting binding between r(CUG) repeats in DMPK (DM1) and MBNL protein reported to be useful for the treatment of myotonic dystrophy 1. Read More
From Dolly the sheep to mRNA vaccines, BioWorld has been reporting every breakthrough and challenge along the way. Our commitment to accurate, contextualized journalism has helped readers navigate this fast-evolving industry for 35 years. Join us as we celebrate the milestones that shaped biopharma and medtech, then and now. Read More
The enzyme PARP1 helps repair single-stranded DNA breaks, and its inhibition shows antitumor efficacy in ovarian, breast, prostate and pancreatic cancers involving mutations in BRCA1 or BRCA2. However, resistance to PARP1 inhibition is a major problem. Read More
Anti-inflammatory compounds can alleviate many acute and chronic diseases, including autoimmune, cardiovascular and neurodegenerative disorders. However, many such compounds increase risk of numerous adverse events because they inhibit not only cyclooxygenase-2 (COX-2), which induces pathological inflammation, but also COX-1, which is important for renal and gastrointestinal function. Read More
Merck KGaA has reported proteolysis targeting chimera (PROTACs) comprising an E3 ubiquitin ligase-binding moiety covalently linked to mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1)-targeting moiety. They are described as potentially useful for the treatment of cancer. Read More
Boehringer Ingelheim Pharma GmbH & Co. KG has discovered son of sevenless homolog 1 (SOS1) inhibitors potentially useful for the treatment of cancer. Read More
In idiopathic pulmonary fibrosis, the lung tissue thickens and stiffens through proliferation of fibroblasts and invasion by inflammatory cells. One of the drivers of these processes is the enzyme autotaxin, which produces the signaling molecule lysophosphatidic acid. Several inhibitors of autotaxin have been reported, which show varying degrees of clinical potential. Read More
Jiangsu Hengrui Pharmaceuticals Co. Ltd.; Shanghai Hengrui Pharmaceutical Co. Ltd. have synthesized antibody-drug conjugates (ADCs) comprising a claudin 6 (CLDN6)-targeting antibody linked to cytotoxic drug through linker. They are described as potentially useful for the treatment of ovarian cancer. Read More
Solu Therapeutics Inc. has identified heterobifunctional cotinine-containing compounds acting as sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers or sodium channel protein type 9 subunit α (SCN9A; Nav1.7) blockers. They are reported to be useful for the treatment of pain, cough and pruritus. Read More
Positive allosteric modulators (PAMs) of the muscarinic acetylcholine M4 receptor show potential against schizophrenia because they can enhance endogenous acetylcholine signaling, which in turn may mitigate psychotic symptoms as well as improve attention and working memory in individuals with the disorder. However, the efficacy of a promising PAM, CVL-231, has been disappointing in clinical trials. Read More
