The acquired von Willebrand syndrome (AVWS) is a rare bleeding disorder with laboratory findings similar to those of inherited von Willebrand disease. Researchers from the Nara Medical University and collaborating institutions presented a potential therapeutic approach for AVWS.
Sickle cell disease (SCD) is an inherited hemoglobinopathy caused by a mutation in the gene encoding β-globin that results in hemoglobin S polymerization, red blood cell (RBC) sickling and hemolytic anemia, among others.
IMV-101 is a new CAR T-cell therapy targeting CD19 developed by Suzhou Immunofoco Biotechnology Co. Ltd. for the potential treatment of B-cell malignancies and autoimmune diseases. The company has presented results of the evaluation of its in vitro and in vivo properties.
Acute myeloid leukemia (AML) is a hematological cancer with limited treatment options and characterized by frequent relapse and poor prognosis. The only approved antibody-drug conjugate for AML is gemtuzumab ozogamicin, which targets CD33.
Eilean Therapeutics LLC has presented data for ZE74-0282, a novel small molecule that targets the JAK2 JH2 domain harboring the V617F mutation with no impact on wild-type JAK2.
Despite therapeutic advances, multiple myeloma (MM) remains incurable, with most patients relapsing and developing resistance, especially those refractory to proteasome inhibitors, immunomodulatory drugs and anti-CD38 antibodies. Limited options and poor prognosis highlight the need for new agents with distinct mechanisms and better safety.
Crossbow Therapeutics Inc. has provided details on the preclinical characterization for the telomerase reverse transcriptase (TERT)-targeting T-cell engager CBX-663.
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.
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