Annexon Inc. has identified boronic acid derivatives acting as complement C1s subcomponent inhibitors reported to be useful for the treatment of neurodegeneration, inflammatory, eye, metabolic and autoimmune diseases.
The role of the enzyme γ-secretase in neuronal cholesterol metabolism could have a beneficial effect on the synapse that has not yet been explored in Alzheimer’s disease (AD). On Aug. 4, 2023, scientists at Stanford University School of Medicine and the Howard Hughes Medical Institute published a study online in Neuron that linked cholesterol levels in the brain to synaptic dysfunction in AD.
The composition of the skull bone is unique and plays a direct role in influencing brain health through small channels in the bone and immune cell expression pathways, reported researchers based at the Helmholtz Center and Ludwig-Maximilians University Munich.
Researchers from Oregon Health and Science University presented data from a study that aimed to identify and validate potential new therapeutic targets for the treatment of amyotrophic lateral sclerosis (ALS). Systems immunology analysis utilizing mass cytometry (CyTOF) was used to assess α5 integrin expression in 78,293 single cells, including 21,250 CD45+/CD11b+ myeloid cells from CNS of superoxide dismutase-1 (SOD) G93A mouse model of ALS (SOD1G93A).
Chugai Pharmaceutical Co. Ltd. has divulged nuclear factor erythroid 2-related factor 2 (NFE2-related factor 2; NRF2) activators reported to be useful for the treatment of neurodegenerative, pulmonary and renal diseases.
A research team from Leipzig University, the Max Planck Institute and Heidelberg University, all in Germany, devised a new segmentation algorithm for stroke lesions that improves upon previous methods. They used machine-learning models to effect CT image segmentation in the early stages of acute stroke. The research team published their findings on the fully convolutional graph network in July in the Journal of Medical Imaging.
Biohaven Therapeutics Ltd. has patented new pyrazolo[1,5-a]pyridin-2,3-yl amides acting as potassium voltage-gated channel subfamily KQT member 2/3 (KCNQ2/3) activators.
Satellos Bioscience Inc. has received orphan drug designation and rare pediatric disease designation from the FDA for SAT-3153 for the potential treatment of Duchenne muscular dystrophy (DMD). The first-in-class oral small-molecule therapeutic is designed to restore the innate muscle regeneration process independent of dystrophin and regardless of exon mutation status.
A Design Therapeutics Inc. patent details conjugates consisting of DNA-binding moiety capable of noncovalently binding to a nucleotide repeat sequence linked to protein binding moiety through oligomeric backbone linker.