Researchers have used cell culture experiments to understand how gene expression was affected in a patient with a rare pain insensitivity syndrome, and have identified a network of hundreds of genes whose expression was changed compared to sex-matched controls. Published online in the journal Brain on May 23, 2023, the research is one step toward translating a rare mutation into medications that could provide benefits for common ailments. Read More
The researchers who enabled patients with spinal cord injuries to walk independently after implanting programmable electrodes below their lesions have now taken things one step further, restoring direct communication from the brain to the spinal cord, enabling the brain rather than an external computer to direct leg movements. Read More
Patients with irritable bowel syndrome (IBS) have chronic abdominal pain as a result of visceral hypersensitivity. Voltage-gated sodium channels control cellular excitability and are involved in the pathophysiology of several functional gastrointestinal disorders. Researchers from Nocion Therapeutics Inc. and the University of Oklahoma recently reported on the preclinical testing of NTX-1175, a sodium-channel blocker, in rat models of acute colonic hypersensitivity to distension. Read More
Researchers from Guilin Medical University (GLMU) reported the discovery and preclinical evaluation of novel indoleamine 2,3-dioxygenase 1 (IDO-1) inhibitors as potential anticancer agents. Synthesis and optimization of a series of chromone-oxime derivatives containing piperazine sulfonamide moieties led to the identification of compound [I] as the lead IDO-1 inhibitor. Read More
Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage. Read More
Recent Perha Pharmaceuticals patents describe imidazolone derivatives acting as inhibitors of dual specificity protein kinase CLK1 and/or CLK4 and/or and dual-specificity tyrosine-phosphorylation-regulated kinase 1A (DYRK1A). As such, they are reported to be useful for the treatment of diabetes, cancer, CDKL5 deficiency disorder, chromosome 22q13 deletion syndrome, Alzheimer’s disease, viral infections, Niemann-Pick disease type C and tauopathies, among others. Read More
Proteolysis targeting chimera (PROTAC) compounds comprising an E3 ubiquitin ligase binding moiety covalently linked to non-receptor tyrosine-protein kinase TYK2-targeting moiety have been reported in a Kymera Therapeutics Inc. patent as potentially useful for the treatment of neurological, inflammatory and endocrine disorders, autoimmune diseases, transplant rejection, graft-vs.-host disease and cancer. Read More
Researchers from Skyline Therapeutics (Shanghai) Co. Ltd. presented preclinical data for the new recombinant AAV vector therapeutic SKG-0106, being developed for the treatment of neovascular (wet) age-related macular degeneration (AMD). Read More
China Pharmaceutical University and Jiangsu Chia Tai Tianqing Pharmaceutical Group Co. Ltd. have jointly developed Wee1-like protein kinase (Wee1) inhibitors reported to be useful for the treatment of cancer. Read More
Research at Simcere Zaiming Pharmaceutical Co. Ltd. has led to the development of polycyclic compounds acting as E3 ubiquitin-protein ligase CBL-B inhibitors and thus reported to be useful for the treatment of cancer and autoimmune disease. Read More
Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the spinal cord, with the consequent muscle atrophy. There is thus a need for new AAV gene therapies for SMA that confer better safety and efficacy. Read More
