CAR T-cell therapy that targets BCMA is an effective option for treating relapsed/refractory multiple myeloma, but there is a lack of persistence due to the inability to develop a memory phenotype.

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.

The industry is again raising concerns that new EU health technology assessment rules coming into force on Jan. 12, 2025, will have the opposite of the desired effect and slow down access to innovative therapies.

Acute myeloid leukemia (AML) is characterized by the accumulation of immature blasts in the bone marrow or in the peripheral blood.

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.

Targeting topoisomerase 2 (TOP2) is crucial in AML, and researchers from Wakunaga Pharmaceutical Co. Ltd. have developed and tested racemic WAC-224, a quinolone TOP2 inhibitor, and (R)-WAC-224 as a potential approach for AML treatment.

Among the acquired mutations in the calreticulin (CALR) gene, both 52 bp deletion (del52) and 5 bp insertion (ins5) are among the most frequent and are linked to two different types of myeloproliferative neoplasms (MPNs).