Biopharmaceuticals (i.e., biological medicines sourced from genetically-engineered living systems) for treatment of human diseases have become a significant percentage of the pharmaceutical industry. And not just the recombinant DNA-derived proteins and monoclonal antibodies (both from the innovators and biosimilars); but now, an increasing awareness of the importance of gene therapy and genetically engineered cellular medicinal products. These biopharmaceuticals are being developed by man...

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 Ad...

CMC regulatory compliance strategy for biopharmaceuticals has become vastly more complex and challenging in the past couple of years, especially as the number of gene therapies have entered development and even market approval. Biopharmaceuticals today can be either protein-based or gene-based. For gene-based biopharmaceuticals, the CMC regulatory compliance strategy must address adequate controls and patient safety concerns for viral vectors, non-viral vectors and genetically modified pat...

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 recom...

This comprehensive book encompasses various facets of sterile product development. Key concepts relevant to the successful development of sterile products are illustrated through case studies and are covered under three sections in this book:• Formulation approaches that discuss a variety of dosage forms including protein therapeutics, lipid-based controlled delivery systems, PEGylated biotherapeutics, nasal dosage form, and vaccines• Process, container closure and delivery...

Every one of us is unique. With recent advances in technology, we now know that that statement is more true that ever: we are each individuals, right down to a molecular level - a one-of-a-kind combination of genes, proteins, and metabolism. So why does healthcare still take a one-size-fits-all approach? The same methods are used on everyone to diagnose illness, and the same drugs are used to treat it - despite the fact that those methods and treatments are not effective for everyone and a...

Drug discovery increasingly requires a common understanding by researchers of the many and diverse factors that go into the making of new medicines. The scientist entering the field will immediately face important issues for which his education may not have prepared him: project teams, patent law, consultants, target product profiles, industry trends, Gantt charts, target validation, pharmacokinetics, proteomics, phenotype assays, biomarkers, and many other unfamiliar topics for which a ba...

The focus of early drug development has been the submission of an Investigational New Drug application to regulatory agencies. Early Drug Development: Strategies and Routes to First-in-Human Trials guides drug development organizations in preparing and submitting an Investigational New Drug (IND) application. By explaining the nuts and bolts of preclinical development activities and their interplay in effectively identifying successful clinical candidates, the book helps pharmaceu...

In this third edition of his popular undergraduate-level textbook, Des Nicholl recognises that a sound grasp of basic principles is vital in any introduction to genetic engineering. Therefore, the book retains its focus on the fundamental principles used in gene manipulation. It is divided into three sections: Part I provides an introduction to the relevant basic molecular biology; Part II, the methods used to manipulate genes; and Part III, applications of the technology. There is a new c...

This second edition of a very successful book is thoroughly updated with existing chapters completely rewritten while the content has more than doubled from 16 to 36 chapters. As with the first edition, the focus is on industrial pharmaceutical research, written by a team of industry experts from around the world, while quality and safety management, drug approval and regulation, patenting issues, and biotechnology fundamentals are also covered. In addition, this new edition now not only i...

Pharmaceutical Biotechnology offers students taking Pharmacy and related Medical and Pharmaceutical courses a comprehensive introduction to the fast-moving area of biopharmaceuticals. With a particular focus on the subject taken from a pharmaceutical perspective, initial chapters offer a broad introduction to protein science and recombinant DNA technology- key areas that underpin the whole subject. Subsequent chapters focus upon the development, production and analysis of these su...

This manual is a comprehensive compilation of "methods that work" for deriving, characterizing, and differentiating hPSCs, written by the researchers who developed and tested the methods and use them every day in their laboratories. The manual is much more than a collection of recipes; it is intended to spark the interest of scientists in areas of stem cell biology that they may not have considered to be important to their work. The second edition of the Human Stem Cell Manual is an extrao...