Sickle cell disease (SCD) and transfusion-dependent β-thalassemia (TDT) are severe monogenic blood disorders caused by mutations in the β-globin gene (HBB), resulting in abnormal or insufficient production of adult hemoglobin (HbA). Among emerging therapeutic approaches, the reactivation of fetal hemoglobin (HbF) represents one of the most promising strategies for both conditions.

Clarivate plc has unveiled the 2025 Citation Laureates. Widely considered a predictor of the Nobel Prizes, this recognition has highlighted the discovery of biomolecular condensates in chemistry and the innate immunity signaling pathway in physiology or medicine, as well as the identification of leukemia stem cells and ghrelin, the so-called hunger hormone.

Mutations in the F9 gene cause factor IX (FIX) deficiency, leading to severe bleeding in people with hemophilia B. Current therapies rely on frequent FIX infusions or newer agents that rebalance coagulation, but both approaches still require weekly to bimonthly dosing and often fail to entirely prevent breakthrough bleeding events, which remain common in patients. Therefore, new therapeutic strategies providing a predictable, durable, active and redosable source of FIX are urgently needed.

If a cancer patient has bone metastases, they will frequently have anemia as well. Given that the bone marrow is the site of blood cell formation, this observation is perhaps not unexpected. But its molecular underpinnings had been unclear to date. Now, researchers at Princeton University and the Rutgers Cancer Institute of New Jersey have identified a specialized group of iron-transporting macrophages in the metastasis-anemia link.

Enlaza Therapeutics Inc. will take charge of research through nominating candidates in its potentially $2 billion-plus deal with Vertex Pharmaceuticals Inc. For its efforts, Enlaza is getting $45 million in an up-front payment and equity investment, plus the opportunity to bring in more than $2 billion in research, development, regulatory and commercial milestones and tiered royalties on net sales.

The FDA has granted orphan drug designation to Vanda Pharmaceuticals Inc.’s VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV).

BMS-986470 is a potentially first-in-class molecular glue dual degrader, targeting zinc finger and BTB domain containing 7A (ZBTB7A) and widely interspaced zinc finger protein (WIZ). Bristol Myers Squibb Inc. recently presented details on the discovery of BMS-986470, which is now in phase I studies for the treatment of sickle cell disease (SCD) (NCT06481306).

Hemab ApS recently presented preclinical data on its human monoclonal antibody against von Willebrand factor (vWF), HMB-002, for the treatment of von Willebrand disease.

Novo Nordisk A/S recently presented data regarding their antibody fragment Inno-8 for the potential treatment of hemophilia A. Inno-8 was characterized in vitro alone or in combination with hemostatic agents used for treating hemophilia A.