Luspatercept

Specification

• Cat#: THP-0398
• Product Name: Luspatercept
• Description: Luspatercept is a recombinant fusion protein comprised of a modified extracellular domain of activin receptor type IIB fused to the FC domain of human IgG1.
• Sequences: E T R E C I Y Y N A20 N W E L E R T N Q S30 G L E R C E G E Q D40 K R L H C Y A S W R50 N S S G T I E L V K60 K G C W D D D F N C70 Y D R Q E C V A T E80 E N P Q V Y F C C C90 E G N F C N E R F T100 H L P E A G G P E V110 T Y E P P P T G G G120 T H T C P P C P A P130 E L L G G P S V F L140 F P P K P K D T L M150 I S R T P E V T C V160 V V D V S H E D P E170 V K F N W Y V D G V180 E V H N A K T K P R190 E E Q Y N S T Y R V200 V S V L T V L H Q D210 W L N G K E Y K C K220 V S N K A L P A P I230 E K T I S K A K G Q240 P R E P Q V Y T L P250 P S R E E M T K N Q260 V S L T C L V K G F270 Y P S D I A V E W E280 S N G Q P E N N Y K290 T T P P V L D S D G300 S F F L Y S K L T V310 D K S R W Q Q G N V320 F S C S V M H E A L330 H N H Y T Q K S L S335 L S P G K
• Species: Human
• Molecular Weight: 76 KDa
• Source: CHO Cells
• Purity: >99% by SDS-Page and HPLC analysis
• Formula: Each 25 mg single vial provides nominal 25 mg of luspatercept-aamt and citric acid monohydrate (0.085 mg), polysorbate 80 (0.10 mg), sucrose (45.0 mg), and tri-sodium citrate dihydrate (1.35 mg) at pH 6.5.
• Endotoxin: <0.001 EU per 1 μg by the LAL method
• Form: Lyophilized Powder or Liquid
• Concentration: 50mg/ml

Pharmaceutical Information

• Targets: Beta thalassemia is a genetic red blood cell disorder caused by mutations in the β-globin gene - these mutations cause oxidative stress and premature apoptosis of erythroblasts, thereby leading to ineffective erythropoesis. The transforming growth factor beta (TGF-β) superfamily of endogenous ligands (including activins, growth differentiation factors, and bone morphogenetic proteins) are involved in the inhibition of erythroid differentiation via activation of the Smad2/3 subfamily of intracellular effectors. Luspatercept is a fusion protein comprising a modified extracellular domain of activin receptor type IIB (a target for many TGF-β ligands) fused to the FC domain of human IgG1. Luspatercept ameliorates ineffective erythropoiesis in patients with beta thalassemia by acting as a "ligand trap" for various members of the TGF-β superfamily, preventing their downstream signalling and subsequent inhibition of late-stage erythroid maturation. The specific members of the TGF-β superfamily targeted by luspatercept are currently unknown, though growth differentiation factor 11 (GDF11) has been experimentally excluded as a potential target.