Generating gametes from nonreproductive tissues could help overcome infertility. Previous studies have successfully transformed stem cells into viable oocytes through cellular reprogramming. Scientists at Oregon Health & Science University (OHSU) developed a method to derive them from skin cells via somatic cell nuclear transfer (SCNT), unlocking a mechanism that blends mitosis and meiosis. Now, the researchers have taken another step forward by generating fertilizable eggs from human skin cells. Read More
In pulmonary hypertension (PH), upregulated signaling by ActRIIA ligands, such as myostatin, activin A and GDF11, activates SMAD2/3 mediated pathways that drive pulmonary vascular remodeling and right ventricular dysfunction. Therapeutic blockade of these ligands using ligand traps such as sotatercept improves vascular remodeling and ameliorates PH pathology. Read More
Faeth Therapeutics Inc. has launched a new R&D initiative aimed at preserving neurocognitive function in children with tyrosinemia type 1 (TT1). Read More
Convelo Therapeutics Inc. has presented data on their 3-β-hydroxysteroid-Δ8,Δ7-isomerase (EBP) inhibitor CVL-1001 as a remyelinating compound for treating multiple sclerosis. Read More
Mari Serebrov, BioWorld’s regulatory editor, shares how the publication’s 35-year commitment to fact-based, expert-driven journalism helps readers navigate complexity and make informed decisions. Read More
The FDA has granted orphan drug designation to FRF-001, the FOXG1 Research Foundation’s lead gene therapy candidate for the treatment of FOXG1 syndrome. This follows the FDA’s earlier award of rare pediatric disease designation to the investigational therapy. Read More
CSPC Pharmaceutical Group Ltd. has obtained clearance from China’s National Medical Products Administration (NMPA) to conduct clinical trials in China with SYH-2070 injection, a double-stranded small interfering RNA (siRNA) drug for the treatment of hypertriglyceridemia or mixed hyperlipidemia. Read More
Shandong Quanzhong Biomedical Technology Co. Ltd. has disclosed receptor-interacting serine/threonine-protein kinase 1 (RIPK1; RIP-1) inhibitors reported to be useful for the treatment of atherosclerosis, obesity, cancer, osteoarthritis, inflammatory bowel disease, fibrosis, psoriasis and Alzheimer’s disease. Read More
The University of California has synthesized α-synuclein (SNCA) and/or amyloid-β protein and/or microtubule-associated protein tau (PHF-tau; MAPT) propagation inhibitors reported to be useful for diagnosis and treatment of multiple system atrophy, Parkinson’s disease and Alzheimer’s disease. Read More
Many patients who survive myocardial infarction go on to experience heart failure. Researchers at Qilu Hospital of Shandong University found that bone morphogenetic protein 1 (BMP1) was upregulated in patients after myocardial infarction, as well as in a mouse model of the same condition. Read More
Forx Therapeutics AG has identified Werner syndrome ATP-dependent helicase (WRN; RECQ3; RECQL2) inhibitors reported to be useful for the treatment of cancer. Read More
Zai Lab (Shanghai) Co. Ltd. and Zai Lab (US) LLC have divulged spirocyclic compounds acting as transcriptional enhancer factor (TEAD) inhibitors reported to be useful for the treatment of cancer. Read More
Ovarian cancer remains unresponsive to immune checkpoint inhibitors due to its ability to suppress cytotoxicity from immune cells that are infiltrating the tumor, where the O-glycosylation pathway may be disrupted. Precision Biologics Inc. is developing PB-vcMMAE-5, an antibody-drug conjugate composed of the monoclonal antibody PB-223, which targets tumor-specific truncated core-2 O-glycans, conjugated to microtubule inhibitor MMAE and with a drug-to-antibody ratio of 3.92. Read More
Contineum Therapeutics Inc. has described lysophosphatidic acid receptor 1 (LPAR1; EDG2) antagonists reported to be useful for the treatment of cancer, idiopathic pulmonary fibrosis, renal fibrosis, nonalcoholic or metabolic dysfunction-associated steatohepatitis (NASH/MASH) and scleroderma. Read More
