Bruton tyrosine kinase (BTK) inhibitors are commonly used in the treatment of hematological cancers. However, approximately 67% of patients with relapsed chronic lymphocytic leukemia (CLL) develop resistance to acalabrutinib and ibrutinib due to mutations in BTK, initially most often C481S.
Hepatoblastoma is the most common pediatric liver cancer and has limited therapeutic options, with platin-based therapy showing severe side effects. WNTinib is a kinase inhibitor with specificity for β-catenin (CTNNB1)-mutated hepatocellular carcinoma (HCC) and is a therapeutic approach that shows promise for treating hepatoblastoma.
Collagen-producing activated myofibroblasts, which are key effector cells in fibrogenesis in different organs, express high levels of platelet-derived growth factor receptor β (PDGFRβ).
Methionine adenosyltransferase 2A (MAT2A), the rate-limiting enzyme in the methionine cycle, catalyzes the formation of S-adenosylmethionine (SAM) from methionine and adenosine triphosphate (ATP).
While there is tremendous enthusiasm for GLP-1 drugs for use in obesity, and 80% of the U.S. population is eligible to use the therapies, tremendous obstacles continue to block their access. The blockages include high prices that consumers currently cannot afford coupled with employer health plans that don’t offer the new treatments.
Cell and gene therapy companies are the beneficiaries of positive changes along the regulatory path that the U.S. FDA is paving for them, according to a panel of executives who spoke at the BioFuture 2024 conference in New York. The agency is trying to set up cell and gene companies for success and that’s a very different agency than what it was years ago, said Paul Bresge, CEO of Ray Therapeutics Inc.
The knockout of Col4a3 in mice is used as a model of Alport syndrome. Knockout mice develop glomerular disease associated with neutral lipid accumulation in the kidney and progressive renal failure, which can be treated with lipid-lowering agents.
The histone acetyltransferase KAT2A, and its paralog KAT2B have been identified as key drivers of tumor cell plasticity in small-cell lung cancer (SCLC). Researchers from Auron Therapeutics Inc. aimed to evaluate the novel KAT2A/B heterobifunctional protein degrader, AUTX-703, in models of SCLC.