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. Read More
About 30%-40% of triple-negative breast cancers (TNBC) show HER2-low status and may benefit from the HER2-directed antibody-drug conjugate trastuzumab deruxtecan (T-DXd). Ataxia-telangiectasia mutated (ATM) kinase plays a crucial role in double-strand break (DSB) repair response, thus inhibitors of ATM, such as AZD-1390, may enhance the effect of DSB inducers, resulting in DNA damage accumulation. Read More
Biomea Fusion Inc. has released preclinical findings from a 28-day weight loss study in obese nonhuman primates evaluating BMF-650, a next-generation oral small-molecule glucagon-like peptide-1 receptor agonist (GLP-1RA). Read More
Diagonal Therapeutics Inc.’s DIAG-723 has been awarded orphan drug designation by the FDA for the treatment of hereditary hemorrhagic telangiectasia (HHT). Additionally, the EMA has provided a positive opinion for orphan drug designation, confirming that DIAG-723 meets the criteria for designation as an orphan drug in the E.U. Read More
Synfini Inc. and O2nix Bio have established a strategic collaboration to co-discover and develop novel drug candidates targeting FTSJ1, a tRNA-modifying enzyme implicated in metastatic cancer cell survival. Read More
Sillajen Inc. has recently presented data regarding their threonine tyrosine kinase (TTK) and Polo-like kinase 1 (PLK1) dual inhibitor BAL-0891 as a therapeutic approach for acute myeloid leukemia (AML) treatment. The compound was tested both in vitro and in vivo in the preclinical setting.
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. Read More
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. Read More
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. Read More
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. Read More
Sudo Biosciences Ltd. has divulged non-receptor tyrosine-protein kinase TYK2 (JH2 domain) inhibitors reported to be useful for the treatment of rheumatoid arthritis, multiple sclerosis, psoriasis, systemic lupus erythematosus, radiographic axial spondyloarthritis (ankylosing spondylitis), idiopathic pulmonary fibrosis, Sjögren’s syndrome and type 1 diabetes. Read More
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. Read More
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. Read More
