A base-by-base comparison of the genome sequences of 240 species of mammals has pinpointed sites in the human genome where mutations are likely to cause disease. The sites are all perfectly conserved across the mammalian family tree over 100 million years of evolution, indicating they underlie fundamental biological processes that do not tolerate diversity or change very well. Read More
In research presented this week at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, investigators from Life Biosciences Inc. reported using epigenetic reprogramming in a nonarteritic anterior ischemic optic neuropathy (NAION) model in nonhuman primates (NHPs). Read More
Cyclin-dependent kinase 4 and 6 (CDK4 and CDK6) are validated targets for the treatment of ER+/HER2- breast cancer, but resistance to therapy is still a problem in these patients, with >20% of them developing intrinsic resistance and up to 70% developing acquired resistance within 3 years. Read More
Researchers from Athira Pharma Inc. presented preclinical efficacy data for ATH-1105, a positive modulator of hepatocyte growth factor (HGF)/MET, being evaluate for the treatment of amyotrophic lateral sclerosis (ALS). Read More
Merck Sharp & Dohme Corp. researchers have patented targeted activator of cell kill (TACK) compounds acting as Gag polyprotein (HIV-1)/protein Pol dimerization inducers and thus reported to be useful for the treatment of HIV infection. Read More
Mast cell-expressed membrane protein 1 (MCEMP1) is a type II transmembrane protein predominantly expressed in myeloid lineage immune cells, including lung-resident mast cells and alveolar macrophages. MCEMP1 is a critical factor in allergic and inflammatory lung diseases, with higher expression levels correlating with increased disease severity. However, the molecular mechanisms underlying the role of MCEMP1 in the pathogenesis of lung inflammatory diseases remain unclear. Read More
Ocugen Inc.’s OCU-410ST (AAV5-hRORA) has been awarded orphan drug designation by the FDA for the treatment of ABCA4-associated retinopathies, including Stargardt disease, retinitis pigmentosa 19 (RP19) and cone-rod dystrophy 3 (CORD3) diseases. Read More
Generate Biomedicines Inc. and The University of Texas MD Anderson Cancer Center have established a strategic collaboration to jointly discover and codevelop protein therapeutics for up to five oncology targets in advanced cancers, including small-cell and non-small-cell lung cancer. Read More
GSK plc and Ideaya Biosciences Inc. have jointly developed cyclic vinyl sulfone compounds acting as Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors that are reported to be useful for the treatment of cancer. Read More
Therini Bio Inc. has closed a US$36 million series A financing round that will support its work on developing fibrin-targeted therapies for diseases driven by chronic inflammation, including Alzheimer’s disease, multiple sclerosis and retinal diseases such as diabetic macular edema. Read More
Rnaimmune Inc., a nonwholly owned subsidiary of Sirnaomics Ltd., has received clearance from the FDA for its IND application to conduct a phase I trial for RV-1730, a SARS-CoV-2 vaccine booster candidate. Read More
2-Oxo-3-azabicyclo[3.1.0]hexane derivatives acting as ketohexokinase (KHK) inhibitors have been reported in a recent Hua Medicine Ltd. patent as potentially useful for the treatment of diabetes, fibrosis, obesity, neurological, metabolic, gastrointestinal, cardiovascular and renal diseases, among others. Read More
Research at Iteos Belgium SA has led to the discovery of macrocyclic compounds acting as equilibrative nucleoside transporter ENT1 inhibitors and reported to be useful for the treatment of cancer. Read More
Recent findings in murine studies have unveiled a circuit linking the basolateral amygdala to the anterior cingulate cortex (BLA-ACC circuit) as a key for chronic pain-induced depression symptoms; researchers aimed to clarify the mechanisms behind this phenotype. Stimulation of this circuit led to the upregulation of semaphorin 4A (SEMA4A), which was the focus of the study. Read More
A recent Astrazeneca AB patent describes 2,5-diazabicyclo[4.2.0]octanes and octahydrofuro[3,4-b]pyrazines acting as glucagon-like peptide 1 receptor (GLP-1R) agonists potentially useful for the treatment of type 2 diabetes. Read More
Cyclin-dependent kinase 8 (CDK8) is implicated in both transcription and cellular response to environmental stress signals. Due to its association with the complex that sustains cell proliferation and viability, and its role in cancer cell stress response to radiotherapy and chemotherapy, CDK8 is considered a target to watch in cancer therapy. Read More
Researchers from Abbvie Inc. recently presented the discovery and preclinical evaluation of a novel CD19-targeting glucocorticoid receptor modulator (GRM) agonist antibody-drug conjugate (ADC), ABBV-319, being developed for the treatment of B-cell malignancies. Read More
KRAS Q61H mutation has been found in pancreatic ductal adenocarcinoma (PDAC), colorectal cancer (CRC), and non-small-cell lung cancer (NSCLC) among other solid tumors. In healthy cells, RAS proteins switch between ON and OFF states during signal transduction, but in cancer, mutations in RAS genes or regulators install RAS proteins in ON state permanently. Read More
