Table 3.

Remaining challenges in the hemophilia treatment landscape, possible solutions, and novel agents in the pipeline

Remaining challengesPossible solutionsNovel agents
Burden of prophylaxis Subcutaneous delivery Factor 8 
 N8-GP 
Factor 9 
 BIVV002 
Substitutive 
 Emicizumab 
Rebalancing 
 AT: fitusiran 
 TFPI: concizumab, PF-06741086, BAY1093884 
 APC: SerpinPC 
 PS: PS siRNA 
Oral delivery Expression within plant cells 
pH-responsive anionic complexation hydrogels 
Immunogenicity Eliminate nonhuman glycan epitopes, expression in human cell lines Human cl-rhFVIII 
Enhance affinity to VWF via nanobody technology FVIII-KB013bv 
Use of nonfactor modalities All substitutive and rebalancing therapies 
Extended time to tolerization Exploit immune-modulatory and tolerogenic properties of Fc fragment rFVIIIFc 
Breakthrough bleeding Resistance to anticoagulant inhibition FIX variant CB2679d 
Increased affinity for cofactor FIX variant CB2679d 
Target steady-state hemostatic control Substitutive 
 Emicizumab 
Rebalancing 
 AT: fitusiran 
 TFPI: concizumab, PF-06741086, BAY1093884 
 APC: SerpinPC 
 PS: PS siRNA 
Remaining challengesPossible solutionsNovel agents
Burden of prophylaxis Subcutaneous delivery Factor 8 
 N8-GP 
Factor 9 
 BIVV002 
Substitutive 
 Emicizumab 
Rebalancing 
 AT: fitusiran 
 TFPI: concizumab, PF-06741086, BAY1093884 
 APC: SerpinPC 
 PS: PS siRNA 
Oral delivery Expression within plant cells 
pH-responsive anionic complexation hydrogels 
Immunogenicity Eliminate nonhuman glycan epitopes, expression in human cell lines Human cl-rhFVIII 
Enhance affinity to VWF via nanobody technology FVIII-KB013bv 
Use of nonfactor modalities All substitutive and rebalancing therapies 
Extended time to tolerization Exploit immune-modulatory and tolerogenic properties of Fc fragment rFVIIIFc 
Breakthrough bleeding Resistance to anticoagulant inhibition FIX variant CB2679d 
Increased affinity for cofactor FIX variant CB2679d 
Target steady-state hemostatic control Substitutive 
 Emicizumab 
Rebalancing 
 AT: fitusiran 
 TFPI: concizumab, PF-06741086, BAY1093884 
 APC: SerpinPC 
 PS: PS siRNA 

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