Biobetters: Protein Engineering to Approach the Curative

ISBN-13: 9781493949946 / Angielski / Miękka / 2016 / 378 str.

Amy Rosenberg; Barthelemy Demeule

Spis treści

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 Schöneich

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

O autorze

Dr. Amy Rosenberg received her M.D. from Albert Einstein College of Medicine, trained in internal medicine and infectious diseases and is Board Certified in Internal Medicine. She was a post-doctoral fellow in Al Singer’s Laboratory in the NCI before coming to CBER, FDA. She is Director of the Division of Therapeutic Proteins, a division that regulates diverse protein therapeutics, including enzyme replacement therapies, hematologic and somatic cell growth factors and immunomodulatory agents including interferons and interleukins. Her particular interests are in tolerance induction in diverse clinical settings including autoimmunity, therapeutic protein immunogenicity and transplantation.

Dr. Barthélemy Demeule obtained his Ph.D. at the University of Geneva, Switzerland, where he started his investigations on the physico-chemical stability of biopharmaceuticals. After a postdoctoral work at Genentech, Inc. focused on the effect of the in vivo environment on antibody-antigen interactions, he stayed in the company where he held positions of increasing responsibilities. He currently leads a group of scientists responsible for the pharmaceutical development of monoclonal antibodies in the last phases of clinical development. He also serves on the editorial board of the European Journal of Pharmaceutics and Biopharmaceutics.

Blurb

Over the last few decades, biopharmaceuticals have transformed many areas of healthcare and have given hope and extended the lives of many patients. Curative treatments, however, remain elusive in most cases. This book describes strategies to develop improved versions of biopharmaceuticals (“biobetters”) to bridge the gap between existing therapies and curative therapies. With an aim to stimulate research and development in a wide array of therapeutic areas, diverse topics are covered, including the following: tissue specific targeting of enzyme replacement therapies; the development of novel protein structures with the potential to improve upon critical attributes of current monoclonal antibodies; the modification of current monoclonal antibody designs to improve their in vivo stability; and finally, the economic and regulatory considerations for the development of biobetters.