Pluvia AS has synthesized pharmacological chaperones able to stabilize phenylalanine hydroxylase (PAH) reported to be useful for the treatment of hyperphenylalaninemia, particularly phenylketonuria (PKU).

National Institute of Biological Sciences has disclosed pyrazole urea derivatives acting as inhibitor of nuclear factor κB kinase subunit ε (IKBKE; IKK-i) and/or TANK-binding kinase 1 (TBK1; NAK; T2K) inhibitors reported to be useful for the treatment of cancer, diabetes, obesity and inflammatory disorders.

Previous studies revealed a positive correlation between O-GlcNAcylation and tumor growth via the stabilization of target proteins, with O-GlcNAcylation transferase (OGT) being the only enzyme capable of catalyzing the addition of O-GlcNAcylation to these proteins. Scientists at Sun Yat-Sen University and affiliated organizations investigated the role of OGT in the progression and drug resistance of multiple myeloma.

The Ca2+ stored in the cellular endoplasmic reticulum (ER) plays a crucial role in protein folding and lipid transfer, and its impairment leads to cellular ER stress. When chronic cellular ER stress occurs in the liver, it triggers the pathogenesis of metabolic dysfunction-associated steatotic liver disease (MASLD) and metabolic dysfunction-associated steatohepatitis (MASH). Previous reports found that NACHT and WD repeat domain containing 1 (NWD1) localized in the ER and mitochondria in neural stem/progenitor cells, but the significance of NWD1 outside the brain is not well known.