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.
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.
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.
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.
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