People with the rare inherited metabolic disorder Gaucher disease have a deficiency in the lipid-digesting glucocerebrosidase enzyme, which causes the accumulation of harmful levels of glucolipids in various organs. The enzyme has a very short half-life, which rules out enzyme replacement as an effective therapy, and as things stand, there are few treatments for this and other lysosomal storage diseases (LSDs). Now, researchers have discovered two small molecules that enhance the activity of glucocerebrosidase in cellular models of LSD, pointing to a potential new approach to treating these diseases. Read More
HIV-1 infects lymphocytes and macrophages, gradually destroying the immune system. Multiple treatment combinations suppress the viral load to undetectable levels, but their long-term use leads to adverse effects. Allosteric inhibition of HIV-1 integrase has emerged as a source for new treatment strategies. Read More
The inappropriate use of antibiotics over long periods of time has led to increasing bacterial drug resistance. Quinolones are among the most effective and widely used antibacterials, and there are ongoing efforts to develop new quinolone-based drugs able to overcome emerging bacterial drug resistance. Read More
Immorna Biotherapeutics Inc. has received a grant from the Bill & Melinda Gates Foundation to support the clinical development of JCXH-108, a monovalent respiratory syncytial virus (RSV) vaccine based on Immorna’s proprietary mRNA and ready-to-use (RTU)-lipid nanoparticle (LNP) technologies. Read More
Purdue Research Foundation has discovered compounds for inhibiting protein aggregation potentially useful for the treatment of wild-type transthyretin amyloidosis, type 2 diabetes, amyotrophic lateral sclerosis and more. Read More
Gilead Sciences Inc. recently disclosed details on the work that led to the discovery of elunonavir (GS-1156), an unboosted HIV protease inhibitor currently in phase I studies. Read More
From no hope to viable treatments, BioWorld is there to cover the breakthroughs in medicine. Listen to Randy Osborne explain why the BioWorld team is ‘always on the lookout for what's next.’ Read More
Researchers from Tanta University and affiliated organizations reported the discovery and preclinical characterization of novel pazopanib analogues as potential anticancer agents. Read More
Invenra Inc.’s bispecific antibody, INV-724, developed for the treatment of neuroblastoma, has been awarded orphan drug and rare pediatric disease designations by the FDA. Read More
Several recent Janssen Pharmaceutica NV patents describe new NLRP3 inflammasome inhibitors reported to be useful for the treatment of traumatic brain injury, multiple sclerosis, amyotrophic lateral sclerosis, dementia, Huntington’s, Parkinson’s, Alzheimer’s and motor neuron diseases. Read More
Recent studies have shown that up-regulation of peroxisome proliferator-activated receptor γ coactivator-1α (PGC-1α) was able to induce fetal hemoglobin synthesis in human primary erythroblasts. Read More
Lucy Therapeutics Inc. has disclosed new mitochondrial F1F0 ATPase hydrolase inhibitors potentially useful for the treatment of cancer, acute coronary syndrome, ischemia, neurodegeneration and Parkinson's disease. Read More
Zymedi Co. Ltd. has patented compounds acting as lysyl tRNA synthetase (KARS) inhibitors and reported to be useful for the treatment of cancer metastasis, inflammation, fibrosis and cardiovascular disorders. Read More
