Son of sevenless 1 (SOS1) is a guanine exchange factor (GEF) primarily responsible for linking cell-surface receptors to RAS protein activation converting the inactive form of GDP-loaded RAS proteins into the active GTP-loaded RAS. This role together with its function in inhibiting MAPK pathway reactivation suggest that SOS1 may be a therapeutic target to treat KRAS-driven cancers.
Barring truly major surprises, exagamglogene autotemcel (Exa-cel, Vertex Pharmaceuticals Inc.) is on track to become the first approved CRISPR-based gene editing therapy. It is partly in expectation of Exa-cel’s approval that the European Hematology Association (EHA) and the European Society for Bone Marrow Transplantation hosted a session on “transplantation versus gene therapy in sickle cell disease.”
Tuberculosis (TB) is the 13th leading cause of death in the world, and it is rising together with the increased prevalence of drug-resistant TB in many countries. The Bacille Calmette-Guerin (BCG) vaccine is the only available TB vaccine, and it has been given to more people than any other vaccine. While the BCG vaccine has saved tens of millions of lives, it confers suboptimal protection against pulmonary TB as it is limited to providing protection only until early childhood. Significantly, the BCG vaccine is administered intradermally to confer exceptional mucosal immunity as compared to most other vaccines, which are more commonly administered intramuscularly. Novel strategies to improve the duration of TB mucosal immunity are urgently needed.
At the recent ASCO meeting, researchers from the University of Rochester reported preclinical data for on a new small-molecule inhibitor of WAP four-disulfide core domain protein 2 (HE4) – UR-238 – that is being investigated for the treatment of cancer.
Autotaxin (ATX) is an enzyme responsible for the production of lysophosphatidic acid (LPA), which plays a role in the pathogenesis of systemic sclerosis (SSc). Researchers from Mitsubishi Tanabe Pharma Corp. and colleagues have reported on the preclinical evaluation of MT-5562, a novel oral ATX inhibitor, as a therapeutic option for SSc.
At the recent American Transplant Congress, researchers from McGill University presented the discovery and preclinical evaluation of a novel retinoic acid receptor-related orphan receptor γt (RORγt) inhibitor, TF-S14, being developed as a potential therapy to delay skin allograft rejection.
New and updated preclinical and clinical data presented by biopharma firms at the American Society of Clinical Oncology annual meeting, including: Amgen, Ascentage, Beyondspring, Bicara, Cohbar, Immvira, Jazz, Kite, Merck & Co., Moderna, Morphogenesis, Nascent, Neximmune, Nkgen, Oncoinvent, Oncolytics, Portage, Precigen, Promontory, Puma, Replimune, Servier, Sorrento, Sutro, Zentalis, Zymeworks.
Bronchiectasis is an irreversible chronic respiratory condition arising from secondary infections that presents as chronic cough with daily purulent sputum due to a permanent abnormal widening of bronchi.
Researchers from Structure Therapeutics Inc. recently presented details on the discovery and preclinical evaluation of two new apelin receptor (APJ, APLNR) agonists – ANPA-0073 and ANPA-0137 – being developed for the treatment of pulmonary diseases.
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