Technological Advances in Longer-Acting Factor

Replacement Therapy for Hemophilia

Program Goals

Factor Replacement Therapy

Challenges of Factor Replacement Therapy

Half-Life Extension Technology

rFIX Fc Fusion Protein (rFIX-Fc)

rFIX-Fc (cont)

rFIX Albumin Fusion Protein (rFIX-FP)

rFIX-FP (cont)

GlycoPEGylated rFIX (N9-GP)

N9-GP (cont)

GlycoPEGylated rFVIII (N8-GP)

rFVIII Fc Fusion Protein (rFVIII-Fc)

rFVIII-Fc (cont)

rFIX-Fc: Phase 3 Studies Dosing

rFIX-Fc: Bleeding Rate by Dose

rFVIII-Fc: Bleeding Rate by Dose

rFVIII-Fc: Dosing Frequency at the End of the Study Period

Summary

Clinical Impact of Half-life Extension

Abbreviations

References

References (cont)