BE-101 is a single IV infusion which does not require preconditioning or immunosuppression in recipients.

Heme Today
Heme Today covers the latest news and analyses in bleeding, clotting, red blood cell, and iron disorders for hematologists and oncologists focused on bleeding disorders.
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Nuvisertib, an oral, investigational, highly selective PIM1 kinase inhibitor, showed clinical activity in a phase 1/2 study.
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.
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A phase II trial calculated a 4-year event-free survival rate of 96% and overall survival rate of 100% from the combination.
Differences were identified between patient racial groups and by urban versus rural populations.
A magnetic resonance imaging study has compared tissue iron levels and biventricular function between the two conditions.