The Alphafold machine learning system for predicting a protein’s structure from its amino acid sequence has been adapted to make it possible to design de novo proteins that fold in a particular way and bind to prespecified target proteins. The sister system, called Alphadesign, works by generating random strings of amino acids, using Alphafold to predict their structure, and then iteratively optimizing the design.
Wuhan Humanwell Pharmaceutical Co. Ltd. has described heterocyclic compounds acting as transcriptional enhancer factor (TEAD) inhibitors reported to be useful for the treatment of cancer.
Actio Biosciences Inc. has synthesized potassium channel subfamily T member 1 (KCNT1) inhibitors reported to be useful for the treatment of Brugada’s syndrome, early infantile epileptic encephalopathy, epilepsy of infancy with migrating focal seizures, epileptic encephalopathy, nocturnal frontal lobe epilepsy, infantile spasm, Lennox-Gastaut syndrome and myocardial infarction, among others.
Evopoint Biosciences Co. Ltd. has disclosed proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase binding moiety coupled to an Aurora kinase A (AURKA; ARK1)-targeting moiety through a linker potentially useful for the treatment of cancer.
Researchers from the Universitat de Barcelona and Oregon Health & Science University have developed a novel gene silencing technique using polypurine reverse Hoogsteen hairpins (PPRH) to target and inhibit the expression of PCSK9.
Researchers at Queen’s University of Belfast and collaborators have developed a DNA vaccine against high-grade serous ovarian cancer. The vaccine encodes PRAME, which the researchers found to be upregulated in several cohorts of patients with the malignancy.
Researchers from Genentech Inc. have developed a novel single-domain antibody (VHH) targeting the IL-23 signaling pathway, which can be administered orally to effectively treat inflammatory bowel disease in animal models.