Biobetters: Protein Engineering to Approach the Curative (Paperback, Softcover Repri)

Part I: Therapeutic enzymes Chapter 1: Targeting glucocerebrosidase to macrophages for effective treatment of patients with Gaucher disease: setting the paradigm of a "fit for purpose" approach to enzyme replacement therapy: Roscoe Brady Chapter 2: Challenges of Enzyme Replacement Therapy: Poor tissue distribution in lysosomal diseases using Pompe disease as a model: Priya Kishnani Chapter 3: Muscle targeting: Nancy Dahms Chapter 4: Blood-Brain Barrier Targeting of Therapeutic Lysosomal Enzymes: William Pardridge Chapter 5: Novel Methods for Addressing Immunogenicity in Therapeutic Enzymes: A De Groot Part II: Monoclonal antibodies: degradation mechanisms and potential improvements Chapter 6: Structure of monoclonal antibodies: Elizabeth Topp Chapter 7: Prediction of aggregation in vivo by studies of therapeutic proteins in human plasma: Tudor Arvinte Chapter 8: Effect of Hydrolytic Degradation on the In Vivo Properties of Monoclonal Antibodies: Elizabeth Topp Chapter 9: Oxidation of proteins in the in-vivo environment: what we know; what we need to study and potential mitigation strategies: Christian Schoneich Chapter 10: Vikas K Sharma, Molecular assessment: balancing affinity, PK and manufacturability. Part III: New Platforms Chapter 11: Perspectives on engineering biobetter therapeutic proteins with greater stability in inflammatory environments: V. Ashutosh Rao Chapter 12: Antibody-like molecules designed for superior targeting and pharmacokinetics: Alexey Lugovskoy Chapter 13: Alternative protein scaffolds as novel biotherapeutics: Arne Skerra Chapter 14: Current strategies for pharmacokinetic optimization: Arne Skerra Part IV: Economic and regulatory considerations Chapter 15: Biosimilar and Biobetter Scenarios for the US and Europe: What Should We Expect?: Ernst Berndt Chapter 16: Anne Pariser, Lynne Yao and Emanuela Lacana, Regulatory considerations for approval of biobetter products