Table 1 Protein . Name . Modification .... | American Society of Hematology

Table 1

Protein	Name	Modification	Clinical status*	Reference(s)
Protein replacement strategies
FVIII	wt FVIII	Pegylated liposomes	No increase in T_1/2; phase 3 completed	17, 18, 55
	N8-GP	Single 40 kDa PEG attached to 21 amino acid B-domain	Well-tolerated; in phase 3	31
	BAX855	∼ 2 mol PEG/full-length FVIII	∼ 1.5× increased T_1/2; in phase 1	29
	BAY94–9027	60 kDa PEG attached to single site in A3-domain	In phase 3	24, 28
	rFVIII-Fc	FcIg fusion to rFVIII	∼ 1.5-2× increased T_1/2; phase 3 completed	36, 56
	scFVIII	Single chain FVIII	∼ 1.14× increased T_1/2	41
	rhFVIII h-cl	rFVIII manufactured in a human cell line	In clinical trials	44, 45
FIX	N9-GP	40 kDa PEG attached to activation peptide	5× increased T_1/2; phase 3 completed	33, 57
	Fc-FIX	FcIg fusion protein to FIX	∼ 3-4× increased T_1/2; phase 3 finished	37
	rFIX-FP	rFIX fusion to albumin	∼ 5× increased T_1/2; in phase 3	39, 40
Bypassing strategies
FVIIa	N7-GP	40 kDa attached to 1 glycan	Characterization stopped due to lack of effect	32
FVIIa	rFVIIa-FP	hFVIIa fusion to albumin	∼ 5× increased T_1/2 in clinical trials	38, 58
mAb	hBS23 Xase mimetic	Bispecific mAb binds FIXa and FX	Preclinical	47
TFPI	mAb 2012	mAb binds TFPI kringle 2 to inhibit	Preclinical	48
	BAX499	Nucleic acid aptamer to inhibit TFPI	Characterization stopped	49
	BAX513	Sulfated polysaccharide	Characterization stopped	50
AT	ALN-AT3	siRNA targeting antithrombin 3	Preclinical	54

Protein	Name	Modification	Clinical status*	Reference(s)
Protein replacement strategies
FVIII	wt FVIII	Pegylated liposomes	No increase in T_1/2; phase 3 completed	17, 18, 55
	N8-GP	Single 40 kDa PEG attached to 21 amino acid B-domain	Well-tolerated; in phase 3	31
	BAX855	∼ 2 mol PEG/full-length FVIII	∼ 1.5× increased T_1/2; in phase 1	29
	BAY94–9027	60 kDa PEG attached to single site in A3-domain	In phase 3	24, 28
	rFVIII-Fc	FcIg fusion to rFVIII	∼ 1.5-2× increased T_1/2; phase 3 completed	36, 56
	scFVIII	Single chain FVIII	∼ 1.14× increased T_1/2	41
	rhFVIII h-cl	rFVIII manufactured in a human cell line	In clinical trials	44, 45
FIX	N9-GP	40 kDa PEG attached to activation peptide	5× increased T_1/2; phase 3 completed	33, 57
	Fc-FIX	FcIg fusion protein to FIX	∼ 3-4× increased T_1/2; phase 3 finished	37
	rFIX-FP	rFIX fusion to albumin	∼ 5× increased T_1/2; in phase 3	39, 40
Bypassing strategies
FVIIa	N7-GP	40 kDa attached to 1 glycan	Characterization stopped due to lack of effect	32
FVIIa	rFVIIa-FP	hFVIIa fusion to albumin	∼ 5× increased T_1/2 in clinical trials	38, 58
mAb	hBS23 Xase mimetic	Bispecific mAb binds FIXa and FX	Preclinical	47
TFPI	mAb 2012	mAb binds TFPI kringle 2 to inhibit	Preclinical	48
	BAX499	Nucleic acid aptamer to inhibit TFPI	Characterization stopped	49
	BAX513	Sulfated polysaccharide	Characterization stopped	50
AT	ALN-AT3	siRNA targeting antithrombin 3	Preclinical	54

Clinical status can be found at www.clinicaltrials.gov.