Myasthenia gravis (MG) is an antibody-mediated chronic neuromuscular disorder leading to fluctuating weakness and early muscle fatigue, with limited treatment options available. In MG, such as other autoimmune diseases, the main pathogenic autoantibody involved is the immunoglobulin G (IgG) isotype.
Seraxis Inc. has obtained IND clearance from the FDA for a phase I/II study of its novel islet replacement therapy SR-02 for type 1 diabetes. SR-02 is comprised of allogeneic pancreatic endocrine cell clusters that, when implanted to the omentum, form a functional endocrine pancreas outside of the native pancreas.
In recent years, immune checkpoint inhibitors have had great success in the treatment of advanced cancers, but often, they are only effective in a minority of patients. Researchers from Weill Cornell Medicine have discovered that the activation of the pentose phosphate pathway (PPP) in combination with standard immune checkpoint inhibitors resulted in enhanced antitumor efficacy both in vitro and in vivo.
A new study helps explain the role of genetic variation in shaping gene regulation in the Indonesian archipelago, one of the most diverse regions in the world. “This study is the only study of splicing from Southeast Asian populations. There is basically no data from this part of the world,” study author Irene Gallego Romero told BioWorld. For drug discovery, most of the people that have historically participated in clinical trials are of European ancestry, and scientists are just beginning to study African populations to better understand genetic differences in these populations, said Romero, a population geneticist and biological anthropologist at the University of Melbourne.
David Baker, Demis Hassabis and John Jumper share the 2024 Nobel Prize in Chemistry for their contributions to the science of protein structure. David Baker was awarded half the prize “for computational protein design,” according to the Royal Swedish Academy of Sciences. Hassabis and Jumper shared the other half “for protein structure prediction.”
Researchers at McGill University and Neurasic Therapeutics Inc. have described substituted thiophene fused cyclohexanone derivatives acting as acid-sensing ion channel 1 (ASIC1) blockers reported to be useful for the treatment of pain, particularly neuropathic and inflammatory pain.
Primemupharma Co. Ltd. has divulged compounds comprising a novel polyester linker acting as antioxidants reported to be useful for the treatment of hearing loss and Meniere’s disease.
Shanghai Helioson Pharmaceutical Co. Ltd. has identified molecular glue degraders acting as DNA-binding protein Ikaros (IKZF1) and/or zinc finger protein Aiolos (IKZF3) degradation inducers reported to be useful for the treatment of cancer, autoimmune disease and inflammatory disorders.
Insilico Medicine Inc. has synthesized NLRP3 inflammasome inhibitors reported to be useful for the treatment of obesity, autoimmune disease and inflammatory disorders.
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