Biobetters

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.

• 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.