Titlebook: Biobetters; Protein Engineering Amy Rosenberg,Barthélemy Demeule Book 2015 American Association of Pharmaceutical Scientists 2015 drug tar

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Challenges of Enzyme Replacement Therapy: Poor Tissue Distribution in Lysosomal Diseases Using Pomperidging the gap between currently available therapies and a cure for LSDs are best viewed through the lens of our collective experience with Pompe disease. While ERT dramatically improved the prognosis in both infantile and later onset forms of Pompe disease, several limitations exist: the inefficie

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Prediction of Aggregation In Vivo by Studies of Therapeutic Proteins in Human Plasmamixing Herceptin. and Avastin. in 5 % dextrose alone failed to reveal aggregates. However, mixing these formulations with human plasma resulted in the formation of large aggregates. No such aggregation was observed when the antibodies were formulated in 0.9 % NaCl and mixed with human plasma. The ag

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Oxidation of Proteins in the In Vivo Environment: What We Know; What We Need to Study and Potential -epitopes cross-react with native, unmodified proteins, breaking immune tolerance (Griffiths 2008; Omersel et al. 2008, 2011; van Beers et al. 2011; Sauerborn et al. 2010; Jiskoot et al. 2009; Schellekens and Jiskoot 2006). Here, autoantibody generation may lead to autoimmune disorders. Analogous to

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Antibody-Like Molecules Designed for Superior Targeting and Pharmacokinetics incorporating additional mechanisms of action, including altered effector function against established therapeutic targets. These molecules are commonly termed biobetters, which, formally speaking, are biologic drugs that are developed against previously validated target antigens but have some prop

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