In cancer, aberrant signaling pathways can evade therapy; activation of oncogenic PI3K and MAPK signaling has inspired researchers to develop molecularly targeted drugs, such as LP-182 (University of Michigan), a multitargeted kinase inhibitor that researchers have tested in vitro and in vivo for the treatment of myelofibrosis. Read More

Ryvu Therapeutics has outlined its plans over the next 2 years to advance one preclinical program into phase I trials, strengthen its synthetic lethality platform and accelerate its early pipeline. The company is currently leading multiple initiatives in the area of synthetic lethality. Read More

Neuron-targeting of caveolin-1 (Cav-1) using AAV9 delivery and a synapsin-driven Cav-1 engineered construct, SynCav1, showed therapeutic potential in the hSOD1G93A model of familial amyotrophic lateral sclerosis (ALS). Read More

TFChem SAS has described new cyclic glycoaminoacid derivatives reported to be useful for the treatment of dry skin, atopic dermatitis, aging, inflammatory disorders, fibrosis and psoriasis, among other disorders. Read More

Cold Spring Harbor Laboratory, Vichem Chemie Research and The Feinstein Institute for Medical Research have described dual-specificity tyrosine-(Y)-phosphorylation regulated kinase 1A (DYRK1A) inhibitors reported to be useful for the treatment of cancer, Down syndrome, mild cognitive impairment and Alzheimer's disease. Read More

Adiponectin (ADIPOQ) is an adipocyte-derived hormone that promotes insulin sensitivity, inhibits cell death and decreases inflammation. Previous studies identified common variants in the ADIPOQ gene that have been associated with aberrant adiponectin levels, obesity, type 2 diabetes and diabetic kidney disease. In a recent study, the first multigenerational family was identified as harboring a protein-truncating mutation in the ADIPOQ gene, p.Gly93GlufsTer73, that cosegregated with diabetes and end-stage renal disease. Read More

Insusense has divulged pyridine derivatives targeting sortilin (neurotensin NTR3; NT3; Gp95) receptors reported to be useful for the treatment of neurodegenerative disorders, cancer, pain, diabetes mellitus, diabetic retinopathy, cardiovascular disease, hereditary eye conditions and hearing loss. Read More

The University of Michigan, the University of Illinois and the Penn State Research Foundation have presented rifamycin derivatives acting as RNA polymerase (bacterial) inhibitors reported to be useful for the treatment of tuberculosis. Read More

Yangtze River Pharmaceutical Group and Shanghai Haiyan Pharmaceutical Technology have discovered substituted pyrazolo[1,5-a]pyrimidine-7-amine derivatives acting as cyclin-dependent kinase 9 (CDK9) inhibitors reported to be useful for the treatment of cancer. Read More

Two large-scale studies provide new data on genes, inherited variations, and de novo mutations associated with autism spectrum disorder (ASD). Some of them are also associated with other neurological conditions, like developmental delay (DD), or schizophrenia. Read More

Learning and memory are complex processes that involve many genes, cell types and brain circuits. Genetic screening performed by researchers have identified potassium voltage-gated channel subfamily C member 3 (KCNC3) to be involved in the hippocampal learning and memory processes in mice. Read More

RNA interactome capture (RIC) was used by researchers from The Barcelona Institute of Science and Technology and their collaborators to examine the melanoma RNA-binding proteomes (RBPomes) of two melanoma cell lines: nontumoral melanocytic Mel-ST and metastatic SK-Mel-147. Read More