Transmembrane serine protease 6 (TMPRSS6) is a negative regulator of hepcidin, which is the main iron homeostasis-regulating hormone. Regeneron Pharmaceuticals Inc. has recently presented preclinical data for the monoclonal antibody targeting TMPRSS6, REGN-7999, which is being developed for the treatment of iron overload.
Flamingo Therapeutics BV and Ionis Pharmaceuticals Inc.’s FTX-001 (also known as FLM-7523) is a potential first-in-class antisense oligonucleotide (ASO) targeting the human metastasis-associated lung adenocarcinoma transcript 1 (MALAT1) long noncoding RNA (lncRNA); it is being developed for the treatment of cancer.
A recent paper has identified the enhancer ETS2, located in a so-called gene desert, as a contributor to five separate immune disorders. It also showed that one of ETS2’s target genes mediating this inflammation was the eminently druggable MEK, a kinase that is the target of the FDA-approved inhibitors Mekinist (trametinib, GSK plc), Mektovi (binimetinib, Array Biopharma Inc.), Cotellic (cobimetinib, Roche Holding AG) and Koselugo (selumetinib, Astrazeneca plc/Merck & Co. Inc.).
SSO-110, also known as DOTA-JR11 or satoreotide tetraxetran, is a somatostatin SST2 receptor (SSTR2) antagonist that targets a higher number of binding sites and stays longer in SSTR2-positive tumors compared to SST2 receptor agonists. It is currently in clinical development as [177Lu]Lu-SSO-110 for small-cell lung cancer (SCLC). Ariceum Therapeutics GmbH has revealed preclinical data on [177Lu]Lu-SSO-110 as well as another SSO-110-based radiopharmaceutical, [225Ac]Ac-SSO-110, which yielded better results than DOTA-TATE-conjugated isotopes.
Aligos Therapeutics Inc. have discovered two novel non-heteroaryldihydropyrimidine (HAP) class A capsid assembly modulators (CAM-A) – ALG-006746 and ALG-006780 – which are being developed for the treatment of hepatitis B virus (HBV) infection.
Researchers from Incyte Corp. and collaborators presented the preclinical profile of INCB-160058, an ATP-competitive small-molecule inhibitor of the JAK2 V617F mutant sparing its wild-type (WT) form, designed for the treatment of JAC2 V617F mutation-positive myeloproliferative neoplasms with potential as a first-in-class JAK2 V617-inhibiting drug.
Bispecific CD20×CD3 T-cell engagers have shown efficacy in hematological malignancies; however, associated toxicities such as cytokine release syndrome and immune effector-associated neurotoxicity syndrome limit their use.
Bladder cancer is characterized by a high recurrence rate, with limited therapeutic options available. There is a need to discover new therapeutic targets and approaches to overcome this.
Residual leukemic stem cells (LSCs) are leukemia relapse-initiating cells that mediate treatment resistance in response to therapy stress. Different from normal hematopoietic stem cells (HSCs), both blasts and LSCs express T-cell immunoglobulin mucin-3 (TIM-3) on the surface.
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