There is still no effective vaccine or cure for HIV. Scientists are considering options ranging from longer-term antiretroviral therapy (ART) that space out injections by several years to long-lasting pre-exposure prophylaxis (PrEP) that acts as a vaccine while immunization is achieved. What else can be done? The “Innovations in HIV virology: Translating discoveries into novel therapies” symposium in basic science at the 13th IAS Conference on HIV Science (IAS 2025), which took place from July 13 to 17, 2025, in Kigali, Rwanda, showcased some of the new ideas that the scientific community are developing. Read More
Fragile X syndrome (FXS), the most common inherited cause of intellectual disability and autism, is caused by silencing of the Fmr1 gene, leading to a lack of the FMRP protein, which regulates protein synthesis in neurons. One key pathway affected by FMRP loss is the metabotropic glutamate receptor 5 (mGluR5) signaling pathway, where activation of mGluR5 leads to excessive translation of several proteins involved in synaptic plasticity.
Rhabdomyosarcoma is the most frequent cancer affecting soft tissues in children, and existing therapies often fail to stop relapse or prolong survival. Researchers in Germany and the U.K. have developed a potential new therapy based on natural killer cells expressing a chimeric antigen receptor against epidermal growth factor receptor (EGFR-CAR). Read More
SK Bioscience Ltd. announced that the company has submitted an investigational new drug (IND) application to the South Korean Ministry of Food and Drug Safety. Read More
In a recent study published in Nature Genetics, a team of scientists used CRISPR-Cas9 gene editing to systematically analyze genetic weaknesses in uveal melanoma cells and comprehensively map monogenic and digenic dependencies. Read More
In drug development, replacing hydrogen atoms with deuterium may help improve the pharmacokinetic profile without altering pharmacodynamic activity. Read More
Gasherbrum Bio Inc. has patented new heterocyclic glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of type 2 diabetes, among others. Read More
Researchers have identified bi-allelic variants in the POPDC2 gene as the cause of a rare inherited cardiac syndrome characterized by sinus node dysfunction, atrioventricular (AV) conduction defects and hypertrophic cardiomyopathy. Read More
Splicing is a process necessary for the correct synthesis of proteins and an essential step in gene expression. An impaired minor splicing may lead to aberrant pre-mRNA transcripts and exon skipping, leading to premature stop codons and truncated synthesized proteins, resulting in severe consequences. Read More
Deciphera Pharmaceuticals LLC has described new dual serine/threonine-protein kinase B-Raf (BRAF) and/or RAF proto-oncogene serine/threonine-protein kinase (RAF1; c-Raf) and microtubule destabilizers (tubulin polymerization inhibitors) potentially useful for the treatment of cancer. Read More
Compounds acting as histone acetyltransferase KAT6A (MOZ; MYST-3) and KAT7 (HBO-1; MYST-2) inhibitors are reported in an Ideaya Biosciences Inc. patent as potentially useful for the treatment of cancer. Read More
Lung cancer is the most frequent cause of cancer-related death in both men and women worldwide. Current treatments can fail because of variable responses in different types of patients, drug resistance, poor tumor penetration and systemic toxicity, prompting the continuing search for better therapeutics. Read More
Work at Ensem Therapeutics Inc. has led to the discovery of anilino-pyrazole derivatives acting as cannabinoid CB2 receptor agonists. As such, they are reported to be useful for the treatment of anxiety disorders, autoimmune disease, depression, neurodegeneration, neuroinflammation, osteoarthritis, chronic pain and substance abuse and dependence, among others. Read More
Semmelweis University and University of Debrecen have jointly identified isochroman compounds reported to be useful for the treatment of bacterial infections. Read More
SHEN-211 is a selective 3-chymotrypsin-like protease (3CLpro) inhibitor that can protect against SARS-CoV-2. In previous work, SHEN-211 demonstrated high efficacy in inhibiting 3CLpro (IC50=24 nM) and exhibited broad-spectrum antiviral properties. Read More
Malignant gliomas, which include glioblastomas, are the most frequent primary brain cancer and are associated with extremely poor prognosis. They nearly always recur after treatment, rapidly leading to death. Read More
Additional early-stage research and drug discovery news in brief, from: Aspect Biosystems, Enveric Biosciences, Canary Cure, Precision Biosciences. Read More
