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.
N6-methyladenosine (m6A) modification, mainly controlled by the m6A methyltransferase METTL3, is crucial for RNA regulation and the development of leukemia. However, how METTL3 stability and function are regulated after translation is not fully understood. Since O-GlcNAcylation commonly modifies nuclear and cytosolic proteins, researchers have hypothesized that METTL3 may be O-GlcNAcylated, affecting its stability and oncogenic activity in myeloid malignancies.
Myelodysplastic syndromes (MDS) are marked by ineffective hematopoiesis and dysplasia, which elevate the risk of progression to acute myeloid leukemia (AML). The inflammatory environment in MDS alters the bone marrow niche, facilitating the growth of cancerous cells while inhibiting the healthy formation of blood cells.
Medexus Pharmaceuticals Inc. looks set for its U.S. launch of bifunctional alkylating agent treosulfan in the first half of 2025 following a long-awaited FDA approval of the drug, branded Grafapex, for use in combination with fludarabine as a preparative regimen for allogeneic hematopoietic stem cell transplantation (allo-HSCT) in adults and pediatric patients, 1 and older, with acute myeloid leukemia or myelodysplastic syndrome.
Takeda Pharmaceutical Co. Ltd. is licensing Keros Therapeutics Inc.’s activin inhibitor elritercept in a global development and commercialization deal worth up to $1.3 billion that excludes mainland China, Hong Kong and Macau.
Takeda Pharmaceutical Co. Ltd. is licensing Keros Therapeutics Inc.’s activin inhibitor elritercept in a global development and commercialization deal worth up to $1.3 billion that excludes mainland China, Hong Kong and Macau.
The U.S. FDA said it is investigating the risk of hematologic malignancies associated with Bluebird Bio Inc.’s Skysona (elivaldogene autotemcel), approved in 2022 as a one-time gene therapy for treating early active cerebral adrenoleukodystrophy in boys, ages 4 to 17.
Syros Pharmaceuticals Inc.’s phase III failure with oral retinoic acid receptor alpha agonist tamibarotene in myelodysplastic syndrome (MDS) meant not only severe stock damage but also defaulting on the loan facility with Oxford Finance LLC, which means an obligation may be accelerated for the company of about $43.6 million, including principal, interest, and other amounts, according to an SEC filing.
Shattuck Labs Inc. opted, as one analyst put the matter, to do “the right thing early” by ending the clinical program with phase I-stage SL-172154 and shift resources to SL-325, a death receptor 3 antagonist, initially for inflammatory bowel disease, where TL1A/DR3-blocking antibodies have shown compelling monotherapy efficacy.
Shwachman-Diamond syndrome is an inherited bone marrow failure syndrome characterized by exocrine pancreatic insufficiency, cytopenia, growth restriction and skeletal abnormalities, and for which primary treatment is allogeneic hematopoietic stem cell transplantation, which is associated with significant toxicity.
