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GNR-021 is a long-acting recombinant Factor VIII (FVIII) replacement therapy being developed by XL-protein for the treatment of Hemophilia A (hemostasis). The drug utilizes XL-protein's proprietary PASylation technology, which involves the genetic fusion of a conformationally disordered polypeptide sequence consisting of Proline, Alanine, and Serine (PAS) to the protein. This modification increases the hydrodynamic volume of the Factor VIII molecule, thereby reducing renal clearance and extending its plasma half-life. By prolonging the circulation of Factor VIII, GNR-021 aims to provide a long-acting replacement therapy that requires less frequent dosing compared to standard recombinant FVIII products. It is currently in the IND-enabling stage of development.
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