Receptor-interacting protein kinase 1 (RIPK1) plays a pivotal role in ischemic stroke pathology by mediating necroptosis and promoting neuroinflammation, both of which contribute to secondary brain injury and worsen clinical outcomes.
Trethera Corp. has been awarded a $3 million Small Business Innovation Research (SBIR) grant from the National Institute of Health (NIH) to support evaluation of Trethera’s lead candidate, TRE-515, for the treatment of systemic lupus erythematosus (lupus).
Genfleet Therapeutics Co. Ltd. has presented preclinical data on their KRAS G12C inhibitor fulzerasib, also known as GFH-925, the third approved KRAS G12C inhibitor for treating non-small-cell lung cancer (NSCLC).
DNA-binding drugs have shown potential against the parasitic disease African trypanosomiasis (sleeping sickness), but they have several disadvantages, such as toxic effects or inability to cross the blood-brain barrier, which may prevent them from treating individuals in which the parasites have entered the central nervous system.
Dysregulation of the polycomb repressive complex 2 (PRC2) is associated with epigenetic silencing of tumor suppressor genes, leading to alterations in cell proliferation, differentiation and survival that contribute to cancer progression.
Vasthera Co. Ltd. has received IND clearance from the U.S. FDA, enabling it to initiate a phase I trial for VTB-10 for pulmonary arterial hypertension (PAH). Vasthera identified a deficiency of the enzyme peroxiredoxin (PRX) in PAH lesions and used its Redoxizyme platform to develop VTB-10, a small-molecule enzyme that precisely replicates PRX function.
RAS is the most mutated oncogene in cancer (about 30% rate), where mutations in KRAS are the most prevalent. The approval of covalent KRAS G12C inhibitors has shown allosteric inhibition of KRAS to be a feasible therapeutic strategy, and there is interest in developing new KRAS-directed therapies to target additional KRAS mutants. Amgen has presented data regarding its pan-KRAS inhibitor AMG-410.
Bladder cancer is among the 10 most frequent cancers around the world. While potentially effective treatments exist, they do not benefit all patients, so investigators continue to search for new targets. Researchers at Nanjing Drum Tower Hospital have identified sideroflexin 1 (SFXN1) as a potential therapeutic target.
Researchers at the Massachusetts Institute of Technology have developed a generative AI model that was able to generate novel antibiotic structures from either chemical fragments or de novo, starting from ammonia, methane, water or no starting point at all. In a study that was published online in Cell, the team tested two dozen of more than 10 million structures that were proposed as potential antibiotics by the model.
Adding another name to an impressive roster of partners assembled over the past few years, Skyhawk Therapeutics Inc. inked a neurology-focused deal with Merck KGaA aimed at discovering small-molecule RNA-targeted drugs that could be worth more than $2 billion.
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