Table 1.

OS and CS activity of recombinant FVIII-SQ variants

Production cell typeRecombinant FVIII-SQ variant testedClone/production lot/ medium conditionsOS FVIII activity (IU/dL)CS FVIII activity (IU/dL)OS/CS ratio
Sequence polymorphism      
 CHO Leu1880 (Xyntha-like) Clone 4-E2 96.2 92.8 1.037 
Clone 1-A12 132.0 129.5 1.020 
Clone 1-A12, 1:10 retest 11.5 14.6 0.788 
 CHO Phe1880 (BMN270-like) Clone 5-A5 44.9 54.5 0.823 
Clone 2-B4 29.8 39.3 0.758 
Clone 1-F5 23.9 27.3 0.877 
Production cell type      
 CHO* Xyntha (Wyeth/Pfizer) Lot W05255 76.0 92.7 0.820 
 HepG2 FVIII-SQ (BioMarin) Lot 1 128.0 61.0 2.098 
Lot 2 322.6 176.7 1.826 
Lot 3 71.5 35.2 2.031 
Codon optimization      
 HepG2 Codon optimized† Serum-free 179.1 136.0 1.317 
  Serum-containing 882.1‡ 456.8 1.931 
 HepG2 Not codon optimized Serum-free 5.0 3.8 1.316 
Serum-containing 28.7 14.2 2.021 
 HEK 293 Codon optimized† Serum-containing 56.9 32.6 1.744 
Production cell typeRecombinant FVIII-SQ variant testedClone/production lot/ medium conditionsOS FVIII activity (IU/dL)CS FVIII activity (IU/dL)OS/CS ratio
Sequence polymorphism      
 CHO Leu1880 (Xyntha-like) Clone 4-E2 96.2 92.8 1.037 
Clone 1-A12 132.0 129.5 1.020 
Clone 1-A12, 1:10 retest 11.5 14.6 0.788 
 CHO Phe1880 (BMN270-like) Clone 5-A5 44.9 54.5 0.823 
Clone 2-B4 29.8 39.3 0.758 
Clone 1-F5 23.9 27.3 0.877 
Production cell type      
 CHO* Xyntha (Wyeth/Pfizer) Lot W05255 76.0 92.7 0.820 
 HepG2 FVIII-SQ (BioMarin) Lot 1 128.0 61.0 2.098 
Lot 2 322.6 176.7 1.826 
Lot 3 71.5 35.2 2.031 
Codon optimization      
 HepG2 Codon optimized† Serum-free 179.1 136.0 1.317 
  Serum-containing 882.1‡ 456.8 1.931 
 HepG2 Not codon optimized Serum-free 5.0 3.8 1.316 
Serum-containing 28.7 14.2 2.021 
 HEK 293 Codon optimized† Serum-containing 56.9 32.6 1.744 
*

Based on a literature reference.19 

†

Same codon optimization as in BMN 270.

‡

The back-calculated result is above the upper reportable limit, but raw data for the diluted test sample were obtained within the validated range of quantification.

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