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.
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.
Researchers from Tanta University and affiliated organizations reported the discovery and preclinical characterization of novel pazopanib analogues as potential anticancer agents.
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.
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.
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.
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.
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.
Researchers at Lawrence Livermore National Laboratory, Leidos Biomedical Research Inc. and Theras Inc. (dba Bridgebio Oncology Therapeutics) have described GTPase KRAS (G12C mutant) inhibitors reported to be useful for the treatment of cancer.
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