Regulonix LLC and the U.S. Department of Health and Human Services (HHS) have jointly patented new sodium channel protein type 9 subunit α (SCN9A; Nav1.7) blockers reported to be useful for the treatment of neuropathic pain.
FAST kinase domain-containing protein 5 (FASTKD5) is a mitochondrial protein that is needed for processing mRNA in the primary mitochondrial transcript. Several mutations have been found in other proteins involved in mitochondrial metabolism, but mutations in the FASTKD5 gene have not yet been reported.
Progenra Inc. reports that it has discovered a new class of small-molecule PINK1 activators that restore function in mutant forms of the PINK1 kinase, a key mitochondrial regulator whose dysfunction leads to familial or early-onset Parkinson’s disease (PD).
Qanatpharma AG (QP), Zuse Institute Berlin (ZIB), Enamine Ltd. and Proteros biostructures GmbH have announced the launch of a research collaboration to accelerate the discovery of novel therapeutics targeting cerebral perfusion deficits associated with subarachnoid hemorrhage (SAH).
The U.S. FDA has granted orphan drug designation to Klotho Neurosciences Inc.’s secreted-Klotho (s-KL) promoter, gene and delivery system (KLTO-202 or s-KL-AAV.myo) for the treatment of amyotrophic lateral sclerosis (ALS).
At first blush, the brain’s extracellular matrix (ECM) seems like the opposite of synaptic plasticity. Plasticity is the ability to change; the ECM is stable, to the point that it is often described as a scaffold – something to lend stability. “ECM proteins have some of the longest lifetimes of any protein in the brain,” Anna Molofsky told her audience at the XVII Meeting on Glial Cells in Health and Disease, which is being held in Marseille this week.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged bifunctional epoxide hydrolase 2 (EPHX2; sEH) inhibitors reported to be useful for the treatment of pain, diabetic retinopathy and neurodegeneration. An exemplified compound (Ex 23 pg 78, claim 18) inhibited human sEH activity (IC50=0.5 nM) using Rapidfire mass spectrometry (RFMS) assays (WO 2025125135).
Idorsia Pharmaceuticals Ltd. has synthesized aryl sulfone and sulfanone derivatives acting as orexin OX2 receptor agonists reported to be useful for the treatment of eating disorders, fatigue, Kleine-Levin syndrome, narcolepsy, obesity, pain, and psychiatric and inflammatory disorders, among others.
Neuroinflammation is a common hallmark in several neurodegenerative disorders, including Alzheimer’s and Parkinson’s diseases, among others, where TREM2 (triggering receptor expressed on myeloid cells 2) is a crucial member involved in this biological process and is mainly expressed in microglial cells, being thus an attractive target for diagnostic imaging.