Individuals with both sickle cell disease (SCD) and sickle cell trait are at higher risk than others of developing renal medullary cancer (RMC), the rarest and deadliest subtype of kidney cancer. Researchers at MD Anderson Cancer Center have identified the molecular mechanisms behind the increased risk, gaining new insights into antitumor immunity more generally and, potentially, new ways to treat RMC, and possibly other tumors as well.

SCD “has been studied for 30 years, but 95% of the effort [has been] working on the red blood cells … how red blood cells contribute to hypoxia and then reduce oxygen supply,” Chunru Lin told BioWorld. Read More

In their ongoing search for effective small-molecule inhibitors of focal adhesion kinase (FAK), researchers at Beijing Normal University and collaborators started from a promising lead compound that they previously described. Read More

Latus Bio Inc. is developing a new gene therapy, LTS-101, for the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease characterized by deficiency in the tripeptidyl peptidase 1 (TPP1) protein that leads to lysosomal dysfunction and neurodegeneration. Read More

In their efforts to develop next-generation drugs whose structures differ from those of conventional IMiDs, researchers at Fujimoto Pharmaceutical Corp. developed FPFT-2216 through optimization of a lead compound. Read More

Autobahn Therapeutics Inc. has disclosed fatty acid amide (FAAH) cleavable prodrugs acting as sphingosine 1-phosphate S1P1 receptor (S1PR1; EDG1) and/or S1P5 receptor (S1PR5; EDG8) agonists reported to be useful for the treatment of multiple sclerosis, amyotrophic lateral sclerosis, Alzheimer’s disease, Parkinson’s disease, Huntington’s disease and epilepsy. Read More

Researchers have been exploring ways to activate the cGAS/STING pathway in tumors as a way to slow their growth. Read More

Progressive multifocal leukoencephalopathy (PML) is a fatal demyelinating disease of the central nervous system caused by lytic infection of oligodendrocytes by pathogenic strains of the JC polyomavirus (JCV). This opportunistic virus has long persisted as a serious threat during periods of immunosuppression, and currently, there is no effective antiviral treatment available. Read More

Vertex Pharmaceuticals Inc. has synthesized sodium channel protein type 10 subunit α (SCN10A; Nav1.8) blockers reported to be useful for the treatment of pain. Read More

Aquinnah Pharmaceuticals Inc. has identified microtubule-associated protein tau (PHF-tau; MAPT) aggregation inhibitors reported to be useful for the treatment of Alzheimer's disease and frontotemporal dementia. Read More

The third most significant genetic risk factor for late-onset Alzheimer’s disease is a mutation in the clusterin (CLU) gene that disrupts the expression of sCLU, a cytoprotective protein involved in preventing protein aggregation and promoting the clearance of misfolded proteins. Read More

Hinova Pharmaceuticals Inc. has divulged phosphatidylinositol 3-kinase (PI3K) inhibitors reported to be useful for the treatment of cancer. Read More

Enanta Pharmaceuticals Inc. has described 17β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17β-HSD13) inhibitors reported to be useful for the treatment of liver (hepatocellular carcinoma) cancer, cirrhosis, idiopathic pulmonary fibrosis and nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH). Read More

Researchers from the U.S. have published findings regarding the link between low NEIL2 expression and airway inflammation and increased DNA damage. Read More