Dexoligo Therapeutics has presented data for their liver-targeted siRNA DXO-1801 for the potential treatment of inflammation-driven anemia, a serious complication of chronic diseases such as chronic kidney disease, myelofibrosis or advanced solid tumors characterized by elevated pro-inflammatory cytokines where the availability for iron for erythropoiesis is limited due to increased hepcidin levels.
JAK2 inhibitors (JAK2i) are the standard treatment for myelofibrosis (MF), offering symptom relief and reducing spleen size. However, all FDA-approved JAK2i are type I inhibitors, which fail to eliminate the mutant MPN clone, leading many patients to treatment discontinuation.
Using C-X-C chemokine receptor type 4 (CXCR4) antagonists as cell mobilization agents has resulted in some FDA approved agents, such as Plerixafor, for hematopoietic stem cell transplantation and neutropenia. Oral cell mobilizers could result in using them in conditions such as sickle-cell disease (SCD) and chronic neutropenia. Emory University has developed and presented data for their CXCR4 antagonist EMU-116.
Researchers from Chimerix, now part of Jazz Pharmaceuticals, presented preclinical data on ONC-206, a compound that functions as both an agonist of the mitochondrial protease Caseinolytic peptidase P (CLPP) and an antagonist of the G protein-coupled receptor DRD2, in models of triple-negative breast cancer (TNBC).
Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.
Although hormone receptor-positive (HR+) breast cancer accounts for over 70% of cases, 20%-30% of patients still experience relapse despite endocrine therapies such as tamoxifen. Recurrence is also observed in HER2+ breast cancer, even with HER2-targeted antibodies and antibody-drug conjugates (ADCs), the use of which can be limited by adverse effects such as interstitial lung disease.
Modex Therapeutics Inc. has developed MDX-2003, a first-in-class tetraspecific T-cell engager targeting CD19 and CD20 on B cells, while co-engaging CD3 and CD28 on T cells.
Researchers from Aera Therapeutics Inc. reported the preclinical profile of AERA-109, a novel, targeted in vivo CAR T therapy designed to treat B-cell-mediated autoimmune diseases.
Sumitomo Pharma Co. Ltd. is developing the Menin (MEN1)-myeloid/lymphoid or mixed-lineage leukemia (MLL) interaction inhibitor DSP-5336 (enzomenib) for the treatment of MLL-rearranged acute myeloid leukemia.
Nectin-4 is a cell-surface protein that is highly expressed in a variety of solid tumors, including bladder, head and neck, and certain aggressive breast cancers. Its low-level expression in some normal tissues creates a challenge for therapies, which can unintentionally damage healthy cells and trigger severe side effects.
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