Heme
Nuvisertib, an oral, investigational, highly selective PIM1 kinase inhibitor, showed clinical activity in a phase 1/2 study.
BE-101 is a single IV infusion which does not require preconditioning or immunosuppression in recipients.
Concizumab-mtci improves thrombin production by blocking the tissue factor pathway inhibitor protein.
In the phase 3 explorer8 trial, concizumab reduced the bleeding rate in patients with hemophilia A and B.
The phase 2a trial is investigating the safety and efficacy of HBI-002, an oral low-dose carbon monoxide therapy.
The agent is already approved to treat thrombocytopenia in adults with chronic ITP for whom prior therapy was unsuccessful.
Annette Von Drygalski, MD, PharmD, discussed the promising role of etranacogene dezaparvovec gene therapy in hemophilia B.
Safi Biotherapeutics expects to initiate a clinical trial in 2027 following completion of IND-enabling activities.
The primary end point for RBC transfusion independence was not met but the cohort still had significant anemia improvement.
Oral avatrombopag shows promise in kids with ITP, offering a safer, effective alternative to current treatments.
Researchers also measured in the study cohort a very high discontinuation rate for this agent.
In a cohort of adults and adolescents marstacimab produced bleed control superior to that from on-demand bypassing agent.
The agent notably in preclinical studies has accomplished increased HbF without any evident cytotoxicity.
The phase 3 VERONA trial, which compared this combination with placebo plus azacitidine, observed no new safety signals.
Dr. John Gansner and Dr. Martina H. Slingsby argue this new agent can provide benefit across all bleeding disorders.
Heme Today spoke with Ulrike Reiss, MD, and Andrew Davidoff, MD, of the trial's investigator team.
Gene therapy is reshaping sickle cell care, raising awareness and advancing treatments beyond the lab.
ASH warns NIH cuts would jeopardize blood disorder care and halt key progress in hematology research.
Reduced-intensity HSCT conditioning with lentiviral gene therapy decreased severe vaso-occlusive events by more than 80%.
An interim analysis of the COMPLETE study found that pegcetacoplan improved indicators of blood health.
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