Hemophilia future or investigational therapies

Future and investigational therapies of hemophilia include co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs) at the neonatal age and prevention of the anti-factor VIII memory B-cell response.

Future and investigational therapies of hemophilia include:

Co-transplantation of endothelial colony-forming cells (ECFCs) and placenta-derived mesenchymal stromal cells (PMSCs)

• This is done at the neonatal age.^[1]
• It is a potential strategy to achieve stable and long-term engraftment.^[1]
• This holds great promise for the cell-based treatment of hemophilia A.

Prevention of the anti-factor VIII memory B-cell response

• It is an investigational therapy to counter the formation of inhibitors.
• This is achieved via selective inhibition of the Bruton’s tyrosine kinase, (R)-5-amino-1-(1-cyanopiperidin-3-yl)-3-(4-[2,4-difluorophenoxyl] phenyl)-1H pyrazole-4-carboxamide (PF-06250112), to inhibit B-cell receptor signaling prior to challenge with exogenous factor VIII.^[2]

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