2024 was another banner year for GLP-1 receptor agonists (GLP-1RAs) on multiple fronts. They continued to expand into new indications, and provide their developers with both rich remuneration and scientific acclamation. There are now seven approved GLP-1RAs. Commercially, the most successful one so far is semaglutide, sold under the brand name Wegovy or Ozempic depending on the indication.
Vanda Pharmaceuticals Inc.’s VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor, has been awarded orphan drug designation by the FDA for the treatment of polycythemia vera.
At the recently concluded American Society of Hematology meeting, F Hoffmann-La Roche Ltd. presented promising results for their next-generation coagulation factor VIIIa (FVIIIa) mimetic bispecific antibody, NXT-007 (RO-7589655), supporting the ongoing phase I/II studies.
Investigators from Duke University hypothesized that hirudin-like protease inhibitors could be generated by linking exosite-binding aptamers with small-molecule active site inhibitors, thus generating more potent “EXACT” inhibitors.
An international consortium of thousands of scientists is creating the Human Cell Atlas, a three-dimensional map of all the cells in the body. The goal is to understand all the cells that make up human tissues, organs and systems, which will enable multiple medical applications. This collection of cell maps is openly available for navigation at single-cell resolution, identified through omics analyses that reveal the tridimensional distribution of each cell.
Kalvista Pharmaceuticals Ltd. has identified coagulation factor XII (FXIIa) inhibitors reported to be useful for the treatment of hereditary angioedema, atherosclerosis, inflammatory disorders, neurodegeneration, diabetic retinopathy, SARS-CoV-2 infection (COVID-19), stroke and thrombotic disorders, among others.
Work at Hepaitech (Beijing) Biopharma Technology Co. Ltd. has led to the identification of Mas-related G-protein coupled receptor member X4 (MRGPRX4; SNSR5; SNSR6) antagonists reported to be useful for the treatment of anemia, fungal infections, HIV infections, renal disorders, liver diseases, psoriasis, pruritus and urticaria, among others.
Editas Medicine Inc. has achieved in vivo preclinical proof of concept of hematopoietic stem and progenitor cell (HSPC) editing and fetal hemoglobin (HbF) induction in humanized mice engrafted with human hematopoietic stem cells and lacking their own hematopoietic cells.
Shaanxi Micot Technology Co. Ltd. has described theophylline derivatives acting as anticoagulant reversal agents reported to be useful for the treatment of hemorrhagic disorders.
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