A collaboration of scientists from the NIH Brain Initiative consortium has published eight simultaneous studies in Neuron, Cell, Cell Genomics, Cell Reports and Cell Reports Methods, with the results of the Armamentarium project, a new set of gene therapy tools for the research and treatment of human brain disorders. The methodology, based on genetic techniques, RNA detection, genomic enhancers and viral vectors, is designed to access different CNS cell types, neuronal and non-neuronal cells, with common and reproducible protocols now available for any laboratory. Read More
STAT6 plays a central role in regulating Th2-driven immune responses. Recent studies have identified gain-of-function mutations in the STAT6 gene that are associated with early-onset, severe allergic diseases. As a result, STAT6 has emerged as a promising therapeutic target in conditions such as asthma, eosinophilic inflammation, food allergies and atopic dermatitis, particularly in cases that are refractory to standard therapies. Read More
Avenzo Therapeutics Inc. has gained IND clearance from the FDA for AVZO-023 (formerly ARTS-023), a CDK4-selective inhibitor. The company also announced it has exercised its exclusive option for AVZO-023 from Allorion Therapeutics Inc., securing global (excluding Greater China) development, manufacturing and commercialization rights. Read More
Chronic obstructive pulmonary disease (COPD) is among the most leading causes of death around the world and there are insufficient treatment options that prevent exacerbations or alter the progression of the disease. COPD is a complex disease with multiple factors driving inflammation, emphysema or small airway remodeling, among others, where interleukin-1 receptor-associated kinase 4 (IRAK-4) plays a crucial role in the pathogenesis of the disease. Read More
Ascletis Pharma Inc. has announced IND clearance by the FDA for a phase I trial of ASC-50 for the treatment of mild to moderate plaque psoriasis. Dosing is expected to start in the third quarter of this year. Read More
Peptide-drug conjugates (PDCs) are emerging as a promising alternative to antibody-drug conjugates (ADCs), offering enhanced tumor penetration and reduced immunogenicity. Carbonic anhydrase IX (CAIX) and epidermal growth factor receptor (EGFR) are both well-validated targets in oncology due to their role in cancer cell survival, invasion and migration. Read More
Scientists at Ascentage Pharma (Suzhou) Co. Ltd. and China Pharmaceutical University have synthesized molecular glue degrader compounds acting as eukaryotic peptide chain release factor GTP-binding subunit ERF3A (GSPT1)/CRBN interaction inducers for GSPT1 degradation reported to be useful for the treatment of cancer, viral infections, aging, immunological disorders and neurological disorders. Read More
Scientists at Bayer AG and Dana Farber Cancer Institute Inc. have identified EGFR (exon 20 insertion [Ex20Ins] mutant) and/or HER2 (erbB2) (Ex20Ins mutant) inhibitors reported to be useful for the treatment of lung cancer. Read More
Pulmonary fibrosis is a lung disease with limited therapeutic options and the development of new therapeutics is a clinical unmet need. Little is known about the role of endoplasmic reticulum stress and unfolded protein response seen in macrophages during pulmonary fibrosis. Read More
Chiesi Farmaceutici SpA has divulged sodium channel protein type 9 subunit α (Nav1.7) channel blockers reported to be useful for the treatment of chronic cough. Read More
Chengdu Easton Biopharmaceuticals Co. Ltd. has described menin (MEN1)/KMT2A (MLL) interaction inhibitors reported to be useful for the treatment of cancer and diabetes. Read More
Ovarian cancer remains a leading cause of cancer-related deaths among women, with high recurrence rates and resistance to chemotherapy. CAR T-cell therapies present limited efficacy in solid tumors due to tumor heterogeneity and immune suppression in the tumor microenvironment. Read More
Pancreatic ductal adenocarcinoma (PDAC) is a highly aggressive cancer. The more common BRCA wild-type (wt) subtype is particularly resistant to standard treatments such as gemcitabine and DNA-targeting agents like olaparib. Read More
