Deficiencies in interferon-stimulated gene 15 (ISG15), a protein that normally regulates the immune response, causes mild but persistent inflammation. However, its absence also provides an unexpected advantage by increasing resistance to viral infections. Inspired by this condition and using mRNA technology, scientists at Columbia University and the Icahn School of Medicine at Mount Sinai have developed a broad-spectrum antiviral platform. Read More
A new method for accelerating the maturation of neuronal cell models and brain organoids is poised to make it possible to track the etiology of neurodegenerative diseases that develop over decades. The non-invasive technique uses graphene to convert light into electrical cues that prompt neurons to connect and communicate in vitro. Read More
Researchers from the California Institute of Technology and collaborating institutions have developed a novel HIV vaccine candidate, a new germline-targeting Env SOSIP trimer called 3nv.2, that is designed to elicit antibodies targeting three key epitopes on the HIV envelope protein. Read More
Jazz Pharmaceuticals plc and Saniona AB have entered into a global license agreement for Jazz to obtain exclusive worldwide rights to develop and commercialize SAN-2355 for epilepsy and other potential indications. Read More
Nanjing Tian Yin Jian Hua Pharma Tech Co. Ltd. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of amyotrophic lateral sclerosis, multiple sclerosis, psoriasis, rheumatoid arthritis, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH), gout, inflammatory bowel and Alzheimer’s disease, among others. Read More
Rheumatoid arthritis (RA) is a chronic autoimmune disease characterized by the formation of an invasive and proliferative tissue called synovial pannus, consisting of fibroblast-like synoviocytes (FLSs), immune cells and newly formed vessels. Read More
Arc Therapies Inc., a startup from the National Cancer Center Japan, has initiated research of YB328, a newly identified gut microbe, toward clinical application. The company has designated the YB328 strain as ARC-0812 (RUX: Lux) and will proceed with preclinical and clinical trials to explore its role as an immune adjuvant in cancer immunotherapy. Read More
Despite the success of traditional viral-based CAR T-cell therapies against several blood malignancies, their efficacy remains limited against solid tumors. Non-viral engineering of CAR T cells using electroporation or lipid nanoparticle delivery of CAR-encoding mRNA achieves high but transient CAR expression, highlighting the limitations of current preclinical models for evaluating mRNA-based CAR T cells. Read More
Hangzhou Zhongmei Huadong Pharmaceutical Co. Ltd. has synthesized Myt1 kinase (PKMYT1) inhibitors reported to be useful for the treatment of cancer. Read More
Incyte Corp. has identified Mas-related G-protein coupled receptor member X2 (MRGPRX2) antagonists reported to be useful for the treatment of inflammatory disorders. Read More
Loss-of-function mutations in the CTNNB1 gene cause β-catenin deficiency, leading to CTNNB1 syndrome, a rare neurodevelopmental disorder marked by motor and cognitive impairments. Because the disease stems from single-allele mutations that vary widely, a broadly applicable corrective strategy is needed. Since CTNNB1 is dosage-sensitive, therapies must preserve normal regulation, and in some cases may also need to suppress harmful mutant transcripts. Read More
UCB SA has divulged compounds acting as NAD(+) hydrolase SARM1 (SAMD2; MyD88-5) inhibitors reported to be useful for the treatment of inflammation, injury, neurodegeneration, and eye, autoimmune and neurological disorders, among others. Read More
Zhejiang Bolaatu Medical Technology Co. Ltd. has described glucocorticoid receptor (GR) agonists reported to be useful for the treatment of asthma. Read More
Calcific aortic valve disease (CAVD) is the most prevalent acquired valve heart disorder in aging populations. Its most severe form in aortic valve stenosis (AVS), with an average survival of 2-3 years once symptoms appear. The early diagnosis of AVS based on circulating biomarkers is crucial to label high-risk patients before they progress to the severe form. Read More
