Neuexcell Therapeutics Inc. has announced that NXL-004, an investigational AAV gene therapy product being developed for the treatment of malignant glioma, has been awarded orphan drug designation by the FDA.
Researchers from Zhejiang University and affiliated organizations have reported the discovery of novel orally bioavailable bromodomain-containing protein 9 (BRD9) proteolysis-targeting chimera (PROTAC) candidates for the treatment of acute myelocytic leukemia (AML). Synthesis and optimization led to the discovery of a novel series of BRD9 PROTACs, with compound [I] selected as the lead candidate with the best degradation and proliferation inhibition activities in vitro.
Isozymes that are overexpressed in cancer and key in some metabolic processes are potential therapeutic targets. Previous studies found that phosphoenolpyruvate carboxykinase 2 (PCK2) is required by cancer cells for maintaining high metabolic activity and proliferation in some cancer types, but no small-molecule PCK2 inhibitors currently exist.
Replay Holdings LLC and Miltenyi Biotec BV & Co. KG have announced a licensing and manufacturing agreement to support the development of a GMP-compliant T-cell receptor (TCR) natural killer (NK) cell therapy targeting the tumor-associated neoantigen, PRAME.
Following the recommendation of its Oncology Drugs Advisory Committee, the U.S. FDA approved U.S. Worldmeds LLC’s eflornithine 192-mg tablets for use as a maintenance therapy in patients with high-risk neuroblastoma. Branded Iwilfin, the specific, irreversible inhibitor of ornithine decarboxylase previously known as DFMO, is expected to be available in the coming weeks.
Pfizer Inc. proved its ongoing interest in antibody-drug conjugates (ADCs), as well as at least tentative faith in mesothelin as a target, by snatching up Nona Biosciences Inc. for up to $53 million in up-front and near-term cash along with possibly $1.05 billion in milestone payments as the drug advances.
MLA Diagnostics BV secured a strategic seven-figure investment in its recent investment round to support external validation of the company’s melanoma prognosis test. Co-founders NLC Health Ventures and Brightlands Life Sciences Ventures participated in the round along with LIOF, the regional development organization for the Limburg province of the Netherlands.
Previous studies with mucosa-associated lymphoid tissue lymphoma translocation protein 1 (MALT1) inhibitors have demonstrated their potential as antitumor agents across several tumor models when administered alone or in combination with standard treatments.
Researchers from Weill Cornell Medicine and Scenic Biotech BV presented promising preclinical data on SC-2882, a first-in-class specific glutaminyl-peptide cyclotransferase-like (QPCTL) inhibitor that induces secondary proteolytic degradation of the monocyte chemo attractants CCL2 and CCL7 and inactivation of the “don’t-eat-me” signal CD47, as a novel therapeutic for diffuse large B-cell lymphoma (DLBCL).
Zhejiang Wenda Pharma Technology Ltd. has identified HER2 (erbB2) and its (exon 20 insertion [Ex20Ins], (Ala775_Gly776insTyrValMetAla)) mutant inhibitors reported to be useful for the treatment of cancer.
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