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Table of Contents
Introduction
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
Challenges of Enzyme Replacement Therapy: Poor tissue distribution in lysosomal diseases using Pompe disease as a model
Muscle targeting
Blood-Brain Barrier Targeting of Therapeutic Lysosomal Enzymes
Novel Methods for Addressing Immunogenicity in Therapeutic Enzymes
Structure of monoclonal antibodies
Prediction of aggregation in vivo by studies of therapeutic proteins in human plasma
Effect of Hydrolytic Degradation on the In Vivo Properties of Monoclonal Antibodies
Oxidation of proteins in the in-vivo environment: what we know; what we need to study and potential mitigation strategies
Molecular assessment: balancing affinity, PK and manufacturability
Perspectives on engineering biobetter therapeutic proteins with greater stability in inflammatory environments
Antibody-like molecules designed for superior targeting and pharmacokinetics
Alternative protein scaffolds as novel biotherapeutics
Current strategies for pharmacokinetic optimization
Biosimilar and Biobetter Scenarios for the US and Europe: What Should We Expect?
Regulatory considerations for approval of biobetter products.
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
Challenges of Enzyme Replacement Therapy: Poor tissue distribution in lysosomal diseases using Pompe disease as a model
Muscle targeting
Blood-Brain Barrier Targeting of Therapeutic Lysosomal Enzymes
Novel Methods for Addressing Immunogenicity in Therapeutic Enzymes
Structure of monoclonal antibodies
Prediction of aggregation in vivo by studies of therapeutic proteins in human plasma
Effect of Hydrolytic Degradation on the In Vivo Properties of Monoclonal Antibodies
Oxidation of proteins in the in-vivo environment: what we know; what we need to study and potential mitigation strategies
Molecular assessment: balancing affinity, PK and manufacturability
Perspectives on engineering biobetter therapeutic proteins with greater stability in inflammatory environments
Antibody-like molecules designed for superior targeting and pharmacokinetics
Alternative protein scaffolds as novel biotherapeutics
Current strategies for pharmacokinetic optimization
Biosimilar and Biobetter Scenarios for the US and Europe: What Should We Expect?
Regulatory considerations for approval of biobetter products.