Werner syndrome results from biallelic mutations in the WRN gene on chromosome 8, leading to accelerated aging symptoms. Researchers at Sumitomo Pharma Co. Ltd. have reported the development and characterization of WRN-108, a splice-switching antisense oligonucleotide (ASO) designed to induce exon 27 skipping in WRN transcripts carrying the c.3139-1G>C mutation.
Hematopoietic progenitor kinase 1 (HPK1) is a serine/threonine kinase primarily expressed in hematopoietic cells that functions as a negative regulator of immune cell signaling. Loss or inactivation of HPK1 enhances T-cell proliferation, cytokine secretion and overall antitumor immune responses.
Stipple Bio Inc. raised $100 million in a series A financing to support progression of the company’s lead drug, STP-100, an antibody-drug conjugate that binds an undisclosed target, and the company’s Pointillist discovery platform. The round was co-led by RA Capital, a16z Bio+Health and Nextech Invest. Existing investors, including Emerson Collective Investments (managed by Yosemite), GV (Google Ventures), Lola Capital Partners, GordonMD Global Investments and others, also participated in the round.
Chengdu Kanghong Pharmaceutical Co. Ltd. has disclosed new compounds targeting apolipoprotein A (ApoA; LPA) and acting as lipoprotein(a)-lowering agents that are potentially useful for the treatment of cardiovascular disorders.
Emory University has divulged new prostaglandin EP2 receptor (PTGER2) antagonists potentially useful for the treatment of cancer, neuropathic pain, hypertension, inflammatory bowel disease, Alzheimer’s disease, atherosclerosis, asthma and glomerulonephritis, among others.
Suzhou Spring-Sea Bio-Pharmaceuticals Co. Ltd. has discovered new prodrugs of glucagon-like peptide 1 (GLP-1) polypeptides acting as glucagon-like peptide 1 receptor (GLP-1R) agonists reported to be useful for the treatment of diabetes, obesity, overweight, chronic kidney disease, metabolic dysfunction-associated steatotic liver disease, neurodegeneration, diabetic nephropathy and cardiovascular disorders, among others.
Vertex Pharmaceuticals Inc. has patented new macrocyclic sulfonamide orexin OX2 receptor agonists potentially useful for the treatment of amyotrophic lateral sclerosis, obesity, hypertension, retinopathy, multiple sclerosis, narcolepsy, hypersomnia and Parkinson's disease, among others.
Acadia Pharmaceuticals Inc. has reported new G protein-coupled receptor GPR88 agonists that are potentially useful for the treatment of tardive dyskinesia.
Liver receptor homolog-1 (LRH-1) is a nuclear receptor that promotes the transcription of genes encoding key steroidogenic enzymes, thereby facilitating de novo androgen biosynthesis within the prostate tumor microenvironment. Researchers from Qilu Pharmaceutical Co. Ltd. reported the discovery and preclinical characterization of a series of novel LRH-1 antagonists.
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