Arbor Biotechnologies Inc.’s ABO-101 has been awarded orphan drug and rare pediatric disease designations by the FDA for the treatment of primary hyperoxaluria type 1 (PH1).
Hepatocellular carcinoma (HCC) is an aggressive disease that accounts for 80%-90% of all primary liver cancers. Previous findings have shown fibroblast growth factor 19 (FGF-19) to be overexpressed in up to 30% of HCC cases, exerting its oncogenic effect through its receptors fibroblast growth factor receptor 3 (FGFR3) and FGFR4.
Ariceum Therapeutics GmbH’s radiopharmaceutical 225Ac-SSO110 ([225Ac]satoreotide tetraxetran) has been awarded orphan drug designation by the FDA for the treatment of small-cell lung cancer (SCLC). [225Ac]Satoreotide is a first-in-class Actinium-labeled somatostatin SST2 receptor antagonist.
Entos Pharmaceuticals Inc. has been awarded a $4 million grant by California Institute for Regenerative Medicine (CIRM) to support the completion of IND-enabling activities with ENTLEP-001, a durable genetic medicine for the treatment of congenital generalized lipodystrophy.
Researchers from Nanjing Drum Tower Hospital reported findings from their evaluation of the role of NLR family CARD domain containing 5 (NLRC5) in post-stroke neuroinflammation. Immunofluorescence staining of mouse brains revealed that NLRC5 was mainly expressed in microglia.
Newco Akribion Therapeutics GmbH has raised €8 million (US$8.3 million) in a seed round to develop a new and potent class of RNA-targeted CRISPR nucleases, which, rather than cleaving specific nucleic acids, can destroy every type of nucleic acid in a cell.
F. Hoffmann-La Roche Ltd. and Hoffmann-La Roche Inc. have divulged stimulator of interferon genes protein (STING; TMEM173) antagonists reported to be useful for the treatment of cancer, autoimmune diseases, inflammatory, neurological, metabolic, cardiovascular and ocular disorders.
Etern Biopharma (Shanghai) Co. Ltd. has identified proteolysis targeting chimeras (PROTACs) comprising an E3 ubiquitin ligase binding moiety coupled to an androgen receptor targeting moiety through a linker acting as androgen receptor degradation inducers reported to be useful for the treatment of cancer.
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