This contributed volume presents an overview of concepts, methods, and applications used in several quantitative areas of drug research, development, and marketing. Chapters bring together the theories and applications of various disciplines, allowing readers to learn more about quantitative fields, and to better recognize the differences between them. Because it provides a thorough overview, this will serve as a self-contained resource for readers interested in the pharmaceutical industry, and the quantitative methods that serve as its foundation. Specific disciplines covered include: Biostatistics Pharmacometrics Genomics Bioinformatics Pharmacoepidemiology Commercial analytics Operational analytics Quantitative Methods in Pharmaceutical Research and Development is ideal for undergraduate students interested in learning about real-world applications of quantitative methods, and the potential career options open to them. It will also be of interest to experts working in these areas.

This volume contains a collection of innovative techniques for studying targeted protein degradation. Chapters guide readers through heterobifunctional proteolysis-targeting chimeras (PROTACs) approaches, E3 ligase, E3 ligase-induced ubiquitylation, proteomic approaches, novel degrader molecules, molecular glue, and stabilize binding interaction between a target and E3 ubiquitin ligase. Written in the format of the highly successful Methods in Molecular Biology series, each chapter includes an introduction to the topic, lists necessary materials and reagents, includes tips on troubleshooting and known pitfalls, and step-by-step, readily reproducible protocols. Authoritative and cutting-edge, Targeted Protein Degradation: Methods and Protocols aims to ensure successful results in this emerging field of drug discovery.

The biotechnology/biopharmaceutical sector has tremendously grown which led to the invention of engineered antibodies such as Antibody Drug Conjugates (ADCs), Bispecific T-cell engager (BITES), Dual Variable Domain (DVD) antibodies, and fusion proteins that are currently being used as therapeutic agents for immunology, oncology and other disease conditions. Regulatory agencies have raised the bar for the development and manufacture of antibody-based products, expecting to see the use of Quality by Design (QbD) elements demonstrating an in-depth understanding of product and process based on sound science. Drug delivery systems have become an increasingly important part of the therapy and most biopharmaceuticals for self-administration are being marketed as combination products. A survey of the market indicates that there is a strong need for a new book that will provide "one stop shopping" for the latest information and knowledge of the scientific and engineering advances made over the last few years in the area of biopharmaceutical product development. The new book entitled Development of Biopharmaceutical Drug Device Products is a reference text for scientists and engineers in the biopharmaceutical industry, academia or regulatory agencies. With insightful chapters from experts in the field, this new book reviews first principles, covers recent technological advancements and provides case studies and regulatory strategies relating to the development and manufacture of antibody-based products. It covers topics such as the importance of early preformulation studies during drug discovery to influence molecular selection for development, formulation strategies for new modalities, and the analytical techniques used to characterize them. It also addresses important considerations for later stage development such as the development of robust formulations and processes, including process engineering and modeling of manufacturing unit operations, the design of analytical comparability studies, and characterization of primary containers (pre-filled syringes and vials).Finally, the latter half of the book reviews key considerations to ensure the development and approval of a patient-centered delivery system design. This involves the evolving regulatory framework with perspectives from both the US and EU industry experts, the role of international standards, design control/risk management, human factors and its importance in the product development and regulatory approval process, as well as review of the risk-based approach to bridging between devices used in clinical trials and the to-be-marketed device. Finally, case studies are provided throughout.The typical readership would have biology and/or engineering degrees and would include researchers, scientific leaders, industry specialists and technology developers working in the biopharmaceutical field.

Drug repurposing is the development of existing drugs for new uses: given that 9 in 10 drugs that enter drug development are never marketed and therefore represent wasted effort, it is an attractive as well as inherently more efficient process. Three repurposed drugs can be brought to market for the same cost as one new chemical entity; and they can also be identified more quickly, an important benefit for patients whose diseases are progressing faster than therapeutic innovation. But repurposing also requires a fresh look at configuring pharmaceutical R&D, considering clinical, regulatory and patent issues much earlier than would otherwise be the case; a holistic gedanken experiment almost needs to be undertaken at the very start of any repurposing development. In addition to new ways of thinking, the discovery of repurposing opportunities can take advantage of artificial intelligence techniques to match the perfect new use for an existing drug. And while repurposing of medicines has been in the mind of every doctor since Hypocrates, modern clinical practice will simply have to adapt to new repurposing techniques in an age where the number of known diseases is increasing much faster than the healthcare dollars available.

Continuous pharmaceutical manufacturing is currently receiving much interest from industry and regulatory authorities, with the joint aim of allowing rapid access of novel therapeutics and existing medications to the public, without compromising high quality. Research groups from different academic institutions have significantly contributed to this field with an immense amount of published research addressing a variety of topics related to continuous processing. The book is structured to have individual chapters on the different continuous unit operations involved in drug substance and drug product manufacturing. A wide spectrum of topics are covered, including basic principles of continuous manufacturing, applications of continuous flow chemistry in drug synthesis, continuous crystallization, continuous drying, feeders and blenders, roll compaction and continuous wet granulation.The underlying theme for each of these chapters is to present to the reader the recent advances in modeling, experimental investigations and equipment design as they pertain to each individual unit operation. The book also includes chapters on quality by design (QbD) and process analytical technology (PAT) for continuous processing, process control strategies including new concepts of quality-by-control (QbC), real-time process management and plant optimization, business and supply chain considerations related to continuous manufacturing as well as safety guidelines related to continuous chemistry. A separate chapter is dedicated to discussing regulatory aspects of continuous manufacturing, with description of current regulatory environment quality/GMP aspects, as well as regulatory gaps and challenges. Our aim from publishing this book is to make it a valuable reference for readers interested in this topic, with a desire to gain a fundamental understanding of engineering principles and mechanistic studies utilized in understanding and developing continuous processes. In addition, our advanced readers and practitioners in this field will find that the technical content of Continuous Pharmaceutical Processing is at the forefront of recent technological advances, with coverage of future prospects and challenges for this technology.

Many newly proposed drugs suffer from poor water solubility, thus presenting major hurdles in the design of suitable formulations for administration to patients. Consequently, the development oftechniques and materials to overcome these hurdles is a major area of research in pharmaceutical companies.

Drug Delivery Strategies for Poorly Water-Soluble Drugs provides a comprehensive overview of currently used formulation strategies for hydrophobic drugs, including liposome formulation, cyclodextrin drug carriers, solid lipid nanoparticles, polymeric drug encapsulation delivery systems, self-microemulsifying drug delivery systems, nanocrystals, hydrosol colloidal dispersions, microemulsions, solid dispersions, cosolvent use, dendrimers, polymer- drug conjugates, polymeric micelles, and mesoporous silica nanoparticles. For each approach the book discusses the main instrumentation, operation principles and theoretical background, with a focus on criticalformulation features and clinical studies. Finally, the book includes some recent and novel applications, scale-up considerations and regulatory issues.

Drug Delivery Strategies for Poorly Water-Soluble Drugs is an essential multidisciplinary guide to this important area of drug formulation for researchers in industry and academia working in drugdelivery, polymers and biomaterials.

This book offers an authoritative review of biopharmaceuticals and their clinical relevance. Biopharmaceuticals have been showing high therapeutic potential by means of biological and biosimilar medicines, particularly for the treatment of cancer, chronic diseases (e.g. diabetes, Crohn's disease, psoriasis and rheumatoid arthritis), neurodegenerative disorders (e.g. multiple sclerosis), and they have also been contributing to the progress of innovative therapies such as assisted reproductive medicine. Since the eighties, several biopharmaceuticals have been approved and, due to patents expiration, many biosimilars are also marketed. In this book, readers will find the most relevant updated information about the main clinical applications of pharmaceutical biotechnology. The authors provide expert analysis about the industrial challenges of recombinant proteins and the different classes of biopharmaceuticals, including monoclonal antibodies, vaccines, growth factors and stem cells. Topics such as bioprinting technologies in tissue engineering, gene therapy and personalized medicine are also covered in this book. Professionals, students and researchers interested in this field will find this work an important account.

This book presents the state of the art in glycoscience and proposes a road map for the coming decade, focusing on the potential of glycoscience research to shed light on important basic science issues and give rise to exciting new applications, especially in the field of diagnosis and therapeutics. Individual sections offer in-depth coverage of various topics relating to glycans and biopharmaceuticals, glycans in medical science and medicine, glycan technologies, glycans in food and nutrients, and glycan-related materials and their uses. In addition, the book presents an exemplary training course on glycomics and highlights educational and analytical web resources, and also includes glossaries and boxes summarizing key facts to ensure ease of understanding for non-expert readers and students. Written by more than 150 active participants in the Japan Consortium for Glycobiology and Glycotechnology (JCGG), whose goal is to promote the development of interdisciplinary glycoscience and establish a global network in the field, it is a valuable resource for students, postdocs, and researchers in the life sciences as well as for stakeholders and professionals in government, funding agencies and industry.

"Pharmacology for Chemists, Second Edition" is aimed at industrial and academic organic chemists holding advanced degrees who are entering the field of medicinal chemistry, and who have had little or no education in or exposure to the biological sciences, especially physiology and pharmacology. The first portion of this book concentrates on biological/pharmacological principles and concepts, and the second portion demonstrates how these concepts and principles are applicable to the medicinal chemists efforts, by describing some selected categories of drugs as examples. The book is not intended to be a textbook of pharmacology, but rather is intended to serve as a tool to prepare the reader for further study and more in depth reading.

The second edition defines the tools used in QA/QC, especially the application of statistical tools during analytical data treatment. Clearly written and logically organized, it takes a generic approach applicable to any field of analysis. The authors begin with the theory behind quality control systems, then detail validation parameter measurements, the use of statistical tests, counting the margin of error, uncertainty estimation, traceability, reference materials, proficiency tests, and method validation. New chapters cover internal quality control and equivalence method, changes in the regulatory environment are reflected throughout, and many new examples have been added to the second edition.

A comprehensive look at existing technologies and processes for continuous manufacturing of pharmaceuticals As rising costs outpace new drug development, the pharmaceutical industry has come under intense pressure to improve the efficiency of its manufacturing processes. Continuous process manufacturing provides a proven solution. Among its many benefits are: minimized waste, energy consumption, and raw material use; the accelerated introduction of new drugs; the use of smaller production facilities with lower building and capital costs; the ability to monitor drug quality on a continuous basis; and enhanced process reliability and flexibility. Continuous Manufacturing of Pharmaceuticals prepares professionals to take advantage of that exciting new approach to improving drug manufacturing efficiency. This book covers key aspects of the continuous manufacturing of pharmaceuticals. The first part provides an overview of key chemical engineering principles and the current regulatory environment. The second covers existing technologies for manufacturing both small-molecule-based products and protein/peptide products. The following section is devoted to process analytical tools for continuously operating manufacturing environments. The final two sections treat the integration of several individual parts of processing into fully operating continuous process systems and summarize state-of-art approaches for innovative new manufacturing principles. * Brings together the essential know-how for anyone working in drug manufacturing, as well as chemical, food, and pharmaceutical scientists working on continuous processing * Covers chemical engineering principles, regulatory aspects, primary and secondary manufacturing, process analytical technology and quality-by-design * Contains contributions from researchers in leading pharmaceutical companies, the FDA, and academic institutions * Offers an extremely well-informed look at the most promising future approaches to continuous manufacturing of innovative pharmaceutical products Timely, comprehensive, and authoritative, Continuous Manufacturing of Pharmaceuticals is an important professional resource for researchers in industry and academe working in the fields of pharmaceuticals development and manufacturing.

This book explains theoretical and technological aspects of amorphous drug formulations. It is intended for all those wishing to increase their knowledge in the field of amorphous pharmaceuticals. Conversion of crystalline material into the amorphous state, as described in this book, is a way to overcome limited water solubility of drug formulations, in this way enhancing the chemical activity and bioavailability inside the body. Written by experts from various fields and backgrounds, the book introduces to fundamental physical aspects (explaining differences between the ordered and the disordered solid states, the enhancement of solubility resulting from drugs amorphization, physical instability and how it can be overcome) as well as preparation and formulation procedures to produce and stabilize amorphous pharmaceuticals. Readers will thus gain a well-funded understanding and find a multi-faceted discussion of the properties and advantages of amorphous drugs and of the challenges in producing and stabilizing them. The book is an ideal source of information for researchers and students as well as professionals engaged in research and development of amorphous pharmaceutical products.

The book provides a reference for years to come, written by world-renowned expert investigators studying sex differences, the role of sex hormones, the systems biology of sex, and the genetic contribution of sex chromosomes to metabolic homeostasis and diseases. In this volume, leaders of the pharmaceutical industry present their views on sex-specific drug discovery. Many of the authors presented at the Keystone Symposium on "Sex and gender factors affecting metabolic homeostasis, diabetes and obesity" to be held in March 2017 in Lake Tahoe, CA. This book will generate new knowledge and ideas on the importance of gender biology and medicine from a molecular standpoint to the population level and to provide the methods to study them. It is intended to be a catalyst leading to gender-specific treatments of metabolic diseases. There are fundamental aspects of metabolic homeostasis that are regulated differently in males and females, and influence both the development of diabetes and obesity and the response to pharmacological intervention. Still, most preclinical researchers avoid studying female rodents due to the added complexity of research plans. The consequence is a generation of data that risks being relevant to only half of the population. This is a timely moment to publish a book on sex differences in diseases as NIH leadership has asked scientists to consider sex as a biological variable in preclinical research, to ensure that women get the same benefit of medical research as men.

This book examines stealth liposomes from a multidisciplinary approach, which includes theoretical polymer physics, organic synthesis, colloid science, and biology. Discussions include theory, chemistry, biochemistry, pharmacology, preclinical studies in model systems, and medical applications in humans.

Written in four parts, this book provides a dedicated and in-depth reference for blending within the pharmaceutical manufacturing industry. It links the science of blending with regulatory requirements associated with pharmaceutical manufacture. The contributors are a combination of leading academic and industrial experts, who provide an informed and industrially relevant perspective of the topic. This is an essential book for the pharmaceutical manufacturing industry, and related academic researchers in pharmaceutical science and chemical and mechanical engineering.

This book explores the use of various plant polysaccharides for pharmaceutical purposes, including drug delivery. It examines the exploitation of plant polysaccharides' auxiliary functions to enhance drug release, stability, bioavailability and target specificity. Plant-derived materials are at the center of drug-delivery research thanks to their non-toxicity, biodegradability, ready availability, eco-friendliness and low extraction costs. These materials include polysaccharides, a class of naturally occurring polymers consisting of glucose monomers, which serve as storage carbohydrates in cereals, root vegetables, rhizomes, seeds, fruits, etc.

A comprehensive overview of the current research on inflammation and immunopharmacology, with particular attention to the use of anti-inflammatory drugs, this book discusses future trends in this area of pharmacological research. It addresses an audience with basic knowledge in the inflammatory process, immune system and pharmacology. The book meets the needs of graduate students, junior and senior researchers and is useful as a source of the most current information for those already working in these fields.

An informative look at the intricacies of today's drug development process

Once a discovery organization has identified a potential new drug candidate, it is the daunting task of synthetic organic chemists to identify the chemical process suitable for preparation of this compound in a highly regulated environment. Only through a multi-layered chemical process that takes into account such factors as safety, environmental considerations, freedom to operate and cost-effectiveness can researchers begin to refine the drug in terms of quality and yield.

This book covers both recent advances in the design and synthesis of new drugs, as well as the myriad other issues facing a new drug candidate as it moves through the development process. Utilizing recent case studies, the authors provide valuable insights into the complexities of the process, from designing new synthetic methodologies and applying new automated techniques for finding optimal reaction conditions to selecting the final drug form and formulation.

Both novice and active researchers will appreciate the inclusion of chapters on such diverse topics as: Cross-coupling methods Asymmetric synthesis Automation Chemical Engineering Application of radioisotopes Final form selection Formulations Intellectual property

A wealth of real-world examples and contributions from leading process scientists, engineers, and related professionals make this book a valuable addition to the scientific literature.

This brief explores recent progress in the area of polysaccharides and their composites and nanocomposites. It is a complete resource for the evolving field of polysaccharide based biomaterials and their applications in different fields. The volume focuses on their composition, properties, characterization, chemistry and applications and also highlights recent developments in polysaccharide based composites and nanocomposites spurred by advances in polymer technology and biotechnology. Divided into different sections featuring alginates, carrageenans, cellulose, starch, chitin, chitosan, gums, etc, each chapter presents chemical, physical, and biological attributes, and reviews the recent research performed such as drug carriers, selective water absorption materials from oil-water emulsions, purification of water, biomedical applications, etc. The authors hope that this brief will help to inspire scientists towards novel sources for chemicals, materials, and energy in the years to come.

This book addresses a range of synthesis and characterization techniques that are critical for tailoring and broadening the various aspects of polymer gels, as well as the numerous advantages that polymer gel-based materials offer. It presents a comprehensive collection of chapters on the recent advances and developments in the science and fundamentals of both synthetic and natural polymer-based gels. Topics covered include: synthesis and structure of physically/chemically cross-linked polymer-gels/polymeric nanogels; gel formation through non-covalent cross-linking; molecular design and characterization; polysaccharide-based polymer gels: synthesis, characterization, and properties; modified polysaccharide gels: silica-based polymeric gels as platforms for the delivery of pharmaceuticals; gel-based approaches in genomic and proteomic sciences; emulgels in drug delivery; and organogels. The book provides a cutting-edge resource for researchers and scientists working in various fields involving polymers, biomaterials, bio-nanotechnology and functional materials.

This book documents the long, still ongoing battle between the US Food and Drug Administration and the dietary supplement industry. It presents the complex, often subtle, and sometimes overlooked series of events that had a major impact on how dietary supplements are manufactured, marketed, sold, and used today. While the first few chapters focus on some background topics, the remaining chapters walk the reader through timeline of events, legislative actions, FDA proposed and final rules, and judicial decisions that led to our current dietary supplement regulatory framework. Interwoven in narrative are examples of the roles of science, social and public policy, politics, and popular media.

Early anthropological evidence for plant use as medicine is 60,000 years old as reported from the Neanderthal grave in Iraq. The importance of plants as medicine is further supported by archeological evidence from Asia and the Middle East. Today, around 1.4 billion people in South Asia alone have no access to modern health care, and rely instead on traditional medicine to alleviate various symptoms. On a global basis, approximately 50 to 80 thousand plant species are used either natively or as pharmaceutical derivatives for life-threatening conditions that include diabetes, hypertension and cancers. As the demand for plant-based medicine rises, there is an unmet need to investigate the quality, safety and efficacy of these herbals by the "scientific methods". Current research on drug discovery from medicinal plants involves a multifaceted approach combining botanical, phytochemical, analytical, and molecular techniques. For instance, high throughput robotic screens have been developed by industry; it is now possible to carry out 50,000 tests per day in the search for compounds, which act on a key enzyme or a subset of receptors. This and other bioassays thus offer hope that one may eventually identify compounds for treating a variety of diseases or conditions. However, drug development from natural products is not without its problems. Frequent challenges encountered include the procurement of raw materials, the selection and implementation of appropriate high-throughput bioassays, and the scaling-up of preparative procedures. Research scientists should therefore arm themselves with the right tools and knowledge in order to harness the vast potentials of plant-based therapeutics. The main objective of Plant and Human Health is to serve as a comprehensive guide for this endeavor. Volume 1 highlights how humans from specific areas or cultures use indigenous plants. Despite technological developments, herbal drugs still occupy a preferential place in a majority of the population in the third world and have slowly taken roots as alternative medicine in the West. The integration of modern science with traditional uses of herbal drugs is important for our understanding of this ethnobotanical relationship. Volume 2 deals with the phytochemical and molecular characterization of herbal medicine. Specifically, it focuess on the secondary metabolic compounds, which afford protection against diseases. Lastly, Volume 3 discusses the physiological mechanisms by which the active ingredients of medicinal plants serve to improve human health. Together this three-volume collection intends to bridge the gap for herbalists, traditional and modern medical practitioners, and students and researchers in botany and horticulture.

This timely volume explores the impact of autophagy in various human diseases, emphasizing the cell biological aspects and focusing on therapeutic approaches to these diseases. The chapters cover autophagy and its potential applications on diseases ranging from obesity, osteoarthritis, pulmonary fibrosis, and inflammation, through ALS, Parkinson's, retinal degeneration, breast cancer, alcoholic liver disease and more. The final chapters round out the book with a discussion of autophagy in drug discovery and 'bench to bedside'. Chapters are contributed by leading authorities and describe the general concepts of autophagy in health and disease, marrying cell biology and pharmacology and covering: studies derived from preclinical experiments, manufacturing considerations,and regulatory requirements pertaining to drug discovery and manufacturing and production. This volume will be useful for basic scientists as well as already practicing clinicians and advanced graduate students.