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Shire Pharmaceuticals Group PLC

Drug Names(s): GA-GCB, glucocerebrosidase, DRX008A, velaglucerase alfa

Description: Gene-Activated human glucocerebrosidase (GA-GCB) is a long-term enzyme replacement therapy for the treatment of Gaucher's Disease. Patients with Gaucher disease have a deficiency of the enzyme glucocerebrosidase, resulting in the buildup of glucocerebroside.

GA-GCB is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. GA-GCB contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease.

Deal Structure: GA-GCB was originally developed by TKT Therapeutics. TKT was acquired by Shire in 2005.

Abbvie and Shire
In July 2014, AbbVie and Shire announced that they have reached agreement on the terms of a recommended combination of Shire with AbbVie.


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