Alveogene Ltd. has announced its novel inhaled gene therapy for lethal neonatal surfactant protein B (SP-B) deficiency, AVG-002, has been awarded orphan drug designation by the FDA.
Ipsen SA and Biomunex Pharmaceuticals SAS have signed an exclusive global licensing agreement for BMX-502, a preclinical novel T-cell engager with potential for solid tumors. BMX-502 is a bispecific antibody that engages and activates mucosal-associated invariant T (MAIT) cells and targets the GPC3 tumor antigen, to kill cancer cells.
Sunshine Biopharma Inc. has completed mouse model studies providing proof of concept for the company’s K1.1 mRNA product as a novel therapeutic agent for human hepatocellular carcinoma (HCC).
Researchers from Memorial Sloan Kettering Cancer Center and affiliated organizations revealed findings from the preclinical evaluation of 1H5, a novel monoclonal antibody (mAb) candidate that inhibits the NOTCH signaling pathway and is being developed for the treatment of high-grade gliomas (HGGs).
A U.K. study has uncovered distinctive genetic drivers of type 2 diabetes in South Asians that lead to faster development of complications, the need for earlier insulin replacement therapy and a weaker response to some widely prescribed drugs. That points to the need to refine care pathways. But in addition, the research provides a potent illustration of how the under-representation in genomics databases of people who are not of white European origin can skew results and be a source of discrimination.
Leal Therapeutics Inc. has divulged mitochondrial glutaminase kidney isoform (GLS, GLS1) inhibitors reported to be useful for the treatment of neurological and psychiatric disorders.
Nain Biotech (Hangzhou) Co. Ltd. has identified tetrahydrocarbazole derivatives reported to be useful for the treatment of cancer and viral infections.
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