Table 6.
Strategies for bioengineered factor concentrates with modified properties in hemophilia
| Target | Strategies |
|---|---|
| Increased efficiency of production | Improved secretion efficiency |
| Higher mRNA stability, facilitated intracellular transport* | |
| Increased potency and stability | Active site modifications* |
| FVIIIa stabilization* | |
| Prolonged half-life | Pegylated liposomes (FVIII)** |
| Pegylation (FVIII, FIX, FVIIa) | |
| Polysialylation (FVIII) | |
| Fc fragment fusion (FIX) | |
| Albumin fusion (FVIIa) | |
| Reduced immunogenicity | Porcine/human hybrids |
| C1/C2 domain and inhibitor epitope mapping-driven modifications* |
Notes:
On development by basic studies;
Phase II clinical trial ongoing.