Phenylketonuria (PKU) is an inborn error of metabolism caused by heritable phenylalanine hydroxylase gene mutations that result in decreased metabolism of phenylalanine (Phe) causing brain damage. The most severe phenotype termed PKU occurs when untreated individuals achieve plasma Phe concentrations of >1200 microM/L, which are neurotoxic.
A study from Weill Cornell Medicine and The Jackson Laboratory has described the epigenetic mark SARS-CoV-2 left on immune system stem cells in the most severe cases of COVID-19 early in the pandemic, before the development of vaccines. In their work published in Cell on Aug. 18, 2023, the researchers presented a new methodology to analyze the epigenetic changes in monocytes and circulating hematopoietic stem and progenitor cells (HSPCs) that give rise to monocytes. That allowed corresponding author Steven Josefowicz and his colleagues to see if there were already changes induced by COVID-19 before HSPCs differentiated into monocytes.
Gasherbrum Bio Inc. has described glucagon-like peptide 1 receptor (GLP1R) agonists reported to be useful for the treatment of type 2 diabetes, dyslipidemia, hyperglycemia, hypertension, obesity, stroke, myocardial infarction and gout, among others.
Redx Pharma plc has identified Rho kinase 1 (ROCK 1; p160-ROCK) and/or rho kinase 2 (ROCK 2; ROCKα) inhibitors reported to be useful for the treatment of cancer, fibrosis, autoimmune, inflammatory and neurological disorders.
Silexon Intelligent Technology Co. Ltd. has synthesized heterocyclic compounds acting as mitogen-activated protein kinase kinase kinase kinase 1 (MAP4K1; HPK1; MEKKK1) inhibitors reported to be useful for the treatment of cancer and viral infections.
Shanghai Haihe Biopharma Co. Ltd. has disclosed phosphonic acid derivatives and their prodrugs reported to be useful for the treatment of cancer and infections.
Methylthioadenosine phosphorylase (MTAP) is an enzyme ubiquitously expressed in all tissues. MTAP homozygous deletion occurs in 10-15% of different cancers such as leukemia, lymphoma, lung, brain, breast and others.
17-β-hydroxysteroid dehydrogenase type 10 (17-β-HSD10) catalyzes the turnover of steroids and neurosteroids, among other substrates, and is also a structural component of RNase P. 17-β-HSD10 is involved in the development of several pathologies, and considered a potential drug target for Alzheimer’s disease and some hormone-dependent cancers. A few 17-β-HSD10 inhibitors with either pyrazole, steroids, or benzothiazolylurea structure have been previously developed.
Methyltransferase-like 3 (METTL3) regulates mRNA translation and contributes to the most frequent modification to mRNA, the situation of a methyl group on the N6-position of A (m6A) during transcription.
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