Each year for the past three years, there have been about 50 new molecular medicines approved by the United States Food & Drug Administration (FDA), of which approximately 25% were new biopharmaceuticals. Over 200 recombinant proteins, monoclonal antibodies, antibody drug conjugates, fusion proteins, and Fab fragments are now in the marketplace in both the United States of America (USA) and European Union (EU). There are also now over 60 biosimilars available for all major classes of recombinant proteins and monoclonal antibodies. In addition, gene therapies using genetically engineered viruses and genetically engineered cells are now in the marketplace, and continually growing. This degree of change is reflected in the over 400 CMC regulatory compliance references listed in this book that were either issued or updated since the release of the third edition. Deficiencies in biopharmaceutical CMC regulatory compliance rarely result in termination of a product, but in can readily cause months if not years of delay in initiating clinical trials, or advancing clinical development stages, or even market approval. In summary, this book: Updates real-world CMC deficiency examples with current examples; Addresses current FDA and EMA requirements and expectations for CMC regulatory compliance; Now includes CMC regulatory compliance for the new gene-based biopharmaceuticals.

This book highlights the challenges facing quality assurance/quality control (QA/QC) in today's biopharmaceutical environment and presents the strategic importance and value generated by QA/QC for their involvement in control of manufacturing. It will put into perspective the need for a graded approach to QA/QC from early clinical trials through market approval. Since the first edition published in 2004, there have been more than 50 new regulatory guidances released by the Food and Drug Administration (FDA), European Medicines Agency (EMA) and ICH that affect the CMC regulatory compliance of biopharmaceuticals; also the application of biosimilars has been developed in Europe and is under development in the USA. The revised update will be broadened to include not only biopharmaceuticals (biotech drugs) but also other biologics (vaccines, cell therapy, plasma-derived proteins, etc.)

This book discusses the different regulatory pathways for gene therapy (GT) and cell therapy (CT) medicinal products implemented by national and international bodies throughout the world (e.g. North and South America, Europe, and Asia). Each chapter, authored by experts from various regulatory bodies throughout the international community, walks the reader through the applications of nonclinical research to translational clinical research to licensure for these innovative products. More specifically, each chapter offers insights into fundamental considerations that are essential for developers of CT and GT products, in the areas of product manufacturing, pharmacology and toxicology, and clinical trial design, as well as pertinent "must-know" guidelines and regulations. Regulatory Aspects of Gene Therapy and Cell Therapy Products: A Global Perspective is part of the American Society of Gene and Cell Therapy sub-series of the highly successful Advances in Experimental Medicine and Biology series. It is essential reading for graduate students, clinicians, and researchers interested in gene and cell therapy and the regulation of pharmaceuticals.

Rare diseases collectively affect millions of Americans of all ages, but developing drugs and medical devices to prevent, diagnose, and treat these conditions is challenging. The Institute of Medicine (IOM) recommends implementing an integrated national strategy to promote rare diseases research and product development.

Presents the many recent innovations and advancements in the field of biotechnological processes This book tackles the challenges and potential of biotechnological processes for the production of new industrial ingredients, bioactive compounds, biopolymers, energy sources, and compounds with commercial/industrial and economic interest by performing an interface between the developments achieved in the recent worldwide research and its many challenges to the upscale process until the adoption of commercial as well as industrial scale. Bioprocessing for Biomolecules Production examines the current status of the use and limitation of biotechnology in different industrial sectors, prospects for development combined with advances in technology and investment, and intellectual and technical production around worldwide research. It also covers new regulatory bodies, laws and regulations, and more. Chapters look at biological and biotechnological processes in the food, pharmaceutical, and biofuel industries; research and production of microbial PUFAs; organic acids and their potential for industry; second and third generation biofuels; the fermentative production of beta-glucan; and extremophiles for hydrolytic enzymes productions. The book also looks at bioethanol production from fruit and vegetable wastes; bioprocessing of cassava stem to bioethanol using soaking in aqueous ammonia pretreatment; bioprospecting of microbes for bio-hydrogen production; and more. Provides up to date information about the advancements made on the production of important biotechnological ingredients Complete visualization of the general developments of world research around diverse products and ingredients of technological, economic, commercial and social importance Investigates the use and recovery of agro-industrial wastes in biotechnological processes Includes the latest updates from regulatory bodies for commercialization feasibility Offering new products and techniques for the industrial development and diversification of commercial products, Bioprocessing for Biomolecules Production is an important book for graduate students, professionals, and researchers involved in food technology, biotechnology; microbiology, bioengineering, biochemistry, and enzymology.

Examining the implications and practical implementation of multi-disciplinary International Conference on Harmonization (ICH) topics, this book gives an integrated view of how the guidelines inform drug development strategic planning and decision-making. • Addresses a consistent need for interpretation, training, and implementation examples of ICH guidelines via case studies • Offers a primary reference point for practitioners addressing the dual challenge of interpretation and practical implementation of ICH guidelines • Uses case studies to help readers understand and apply ICH guidelines • Provides valuable insights into guidelines development, with chapters by authors involved in generating or with experience implementing the guidelines • Includes coverage of stability testing, analytical method validation, impurities, biotechnology drugs and products, and good manufacturing practice (GMP)

PART I: GENERAL ASPECTS 1: Introduction, Danny Busch and Guido Ferrarini PART II: INVESTMENT FIRMS AND INVESTMENT SERVICES 2: The Scope of MiFID II, Kitty Lieverse 3: Governance of Investment Firms under MiFID II, Jens-Hinrich Binder 4: The Overarching Duty to Act in the Best Interest of the Client in MiFID II, Luca Enriques and Matteo Gargantini 5: Product Governance and Product Intervention, Danny Busch 6: Independent Financial Advice, Paolo Giudici 7: Conflicts of Interest, Stefan Grundmann and Philipp Hacker 8: Inducements, Larissa Silverentand, Jasha Sprecher, and Lisette Simons 9: Agency and Principal Dealing Under MiFID, Danny Busch 10: MiFID II/MiFIR's Regime for Third-Country Firms, Danny Busch & Marije Louisse PART III: TRADING 11: TGovernance and Organization of Trading Venues: The Role of Financial Market Infrastructures Groups, Guido Ferrarini & Paolo Saguato 12: EU Financial Governance and Transparency Regulation: A Test for the Effectiveness of Post-Crisis Administrative Governance, Niamh Moloney 13: SME Growth Markets, Carmine di Noia & Rudiger Veil 14: Dark Trading Under MiFID II, Peter Gomber & Ilya Gvozdevskiy 15: Derivatives: Trading, Clearing, STP, Indirect Clearing, and Portfolio Compression, Rezah Stegeman & Aron Berket 16: Commodity Derivatives, Antonella Sciarrone Alibrandi & Edoardo Grossule 17: Algorithmic Trading and High Frequency Trading, Pierre-Henri Conac 18: An American perspective, Merritt Fox PART IV: SUPERVISION AND ENFORCEMENT 19: Public Enforcement of MiFID II, Christos Gortsos 20: The Private Law Effect of MiFID: the Genil Case and Beyond, Danny Busch PART V: THE BROADER VIEW AND THE FUTURE OF MIFID 21: MiFID II: Picking up the Crumbs of a Piecemeal Approach, Veerle Colaert 22: Shadow Banking and the Functioning of Financial Markets, Eddy Wymeersch 23: Investment-based Crowdfunding: Is MiFID II enough?, Guido Ferrarini & Eugenia Macchiavello.