Volumes are organized topically and provide a comprehensive discussion of developments in the respective field over the past 3-5 years. The series also discusses new discoveries and applications. Special volumes are dedicated to selected topics which focus on new biotechnological products and new processes for their synthesis and purification. In general, special volumes are edited by well-known guest editors. The series editor and publisher will however always be pleased to receive suggestions and supplementary information. Manuscripts are accepted in English.

This book presents the fundamentals and applications of Matrix Assisted Laser Desorption/Ionization Time-of-Flight Mass Spectrometry (MALDI-ToF-MS) technique. It highlights the basic principles, the history of invention as well as the mechanism of ionization and mass determination using this technique. It describes the fundamental principles and methods for MALDI spectra interpretation and determination of exact chemical structures from experimental data. This book guides the reader through the interpretation of MALDI data where complex macromolecular spectra are simplified in order to present the major principles behind data interpretation. In addition, each chapter describes how MALDI-ToF-MS analysis provides necessary understanding of the copolymer systems that have been designed for specialized biomedical applications.

This volume focuses on current evidence-based pharmacological treatments of various forms of pulmonary hypertension and provides a comprehensive review of the latest developments in this area. The first part of the book covers the definition, classification, pathophysiology, pathology, biomarkers and animal models of the disease, thus laying the conceptual basis for what follows. The middle section provides an overview of the established therapies, such as calcium channel blockers, prostanoids, endothelin receptor antagonists, phosphodiesterase-5 inhibitors and inhaled nitric oxide. The last section explores novel pathways and emerging therapeutic approaches including soluble guanylate cyclase stimulators, Rho-kinase inhibitors, inhibitors of serotonin receptors and transporters, peptide growth factors, vasoactive peptides, modulators of redox equilibrium and cyclic nucleotide homeostasis, as well as immunosuppressive and anti-proliferative agents. Particular attention is given to the clinical applications of these experimental therapies, that are on the horizon. The book thus spans the continuum from basic science to clinical applications.

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.)

Until the 1990s, it was generally accepted that medicines were first developed for adults and their use in children was investigated later, if at all. One of the main tasks of hospital pharmacies was the manufacturing of child-appropriate formulations in a more or less makeshift way. The first change came in 1997 with U.S. legislation that rewarded manufacturers to do voluntary pediatric research. Ten years later, the European Union passed legislation that required manufacturers to discuss all pediatric aspects, including formulations, with the regulatory authorities as a condition of starting the registration procedure. In consequence, manufacturers must now cover all age groups, including the youngest ones. So far, pediatric formulations were more a focus for academic researchers. Through the changed regulatory environment, there is now a sudden high commercial demand for age-appropriate formulations. This book begins by highlighting the anatomical, physiological and developmental differences between adults and children of different ages. It goes on to review the existing technologies and attempts to draw a roadmap to better, innovative formulations, in particular for oral administration. The regulatory, clinical, ethical and pharmaceutical framework is also addressed.

Examples from various organs and diseases illustrate the potential benefit obtained when both therapeutic approaches are combined with delivery strategies. Representing the combined effort of several leading international research and clinical experts, this book, Emerging Trends in Cell and Gene Therapy, provides a complete account on and brings into sharp focus current trends and state-of-the-art in important areas at the interface of cell- and gene-based therapies. This book addresses the current fragmented understanding regarding these two research areas and fills the vast unmet educational need and interest of both students and researchers in academia and industry. Main features of the book: * Biological aspects of stem cell sources, differentiation and engineering. * Application of microfluidics to study stem cell dynamics * Potential clinical application of stem cells and gene therapy to specific human disease. * Utilization of biomaterials and stem cells in regenerative medicine with particular emphasis on spinal cord repair, ligament and bone tissue engineering. * Biomimetic multiscale topography for cell alignment.

Satya P. Gupta's Hydroxamics Acids is the first book to compile invited articles written by international experts on the class of compounds hydroxamic acids. Found to possess a wide spectrum of biological activities, the hydroxamic acids are of interest to theoretical and experimental chemists who can study and make use of them in drug design and development. Chapters in this book provide a diverse and comprehensive coverage of this compound class and consequently this publication is a valuable resource for researchers in chemical, pharmaceutical and biological sciences.

This book is devoted to the graphics of patient data: good graphs enabling straightforward and intuitive interpretation, efficient creation, and straightforward interpretation. We focus on easy access to graphics of patient data: the intention is to show a large variety of graphs for different phases of drug development, together with a description of what the graph shows, what type of data it uses, and what options there are. The main aim is to provide inspiration in form of a "graphics cookbook." Many graphs provide creative ideas about what can be done. The book is not intended to be technical. It introduces general principles of good visualization to make readers understand the concepts, but the main focus is on the creativity and usefulness: readers are enabled to browse through the book to get ideas of how their own data can be analyzed graphically. For additional information visit Editor's companion website: http://www.elmo.ch/doc/life-science-graphics/

The purpose of this publication is to introduce a new, simpler and more effective way in which to interpret pharmaceutical aerosol particle size data from orally inhaled products (OIPs). Currently, the compendial and regulatory requirements dictate the need for measurements by full resolution multi-stage cascade impactor (CI), a process that is demanding for the operator, time consuming, prone to experimental error, and challenging for method transfers from one laboratory to another. Furthermore, we shall show that the current practice of reducing information from mass-weighted aerodynamic particle size distribution (APSD) measurements through the use of CI stage groupings is not the most effective decision-making tool for OIP quality control (QC) in comparison with newly introduced, mutually-independent efficient data analysis (EDA) metrics that can be derived either from full resolution or abbreviated impactor measurements (AIM).

The author successfully developed novel anti-HIV PD 404182 derivatives that exhibited submicromolar inhibitory activity against both HIV-1 and HIV-2. His thesis is in three parts. The first part expounds efficient methods for the synthesis of tricyclic heterocycles related to PD 404182 based on the sp2-carbon−heteroatom bond formations. Starting from arene or haloarene, C-O, C-N, or C-S bonds were formed by simply changing the reactants. These synthetic methods provide powerful approaches for the divergent preparation of pyrimido-benzoxazine, -quinazoline, or -benzothiazine derivatives. The second part explains SAR studies of PD 404182 for the development of anti-HIV agents. Through optimization studies of the central 1,3-thiazin-2-imine core, the benzene and cyclic amidine ring parts, 3-fold more potent inhibitors were obtained compared with the lead compound. The author also reveals by a time-of-drug-addition experiment that PD 404182 derivatives impaired HIV replication at the binding or fusion stage. The third part of the thesis elucidates the development of photoaffinity probes for the target identification of PD 404182. By the photolabeling experiment of HIV-1-infected H9 cells using these probes, the author detected proteins specifically bound to PD 404182. These new anti-HIV agents may be promising agents for anti-HIV therapy because their mechanisms of action differ from those of the currently approved anti-HIV agents.

Nanofiber Composite Materials for Biomedical Applications presents new developments and recent advances in nanofiber-reinforced composite materials and their use in biomedical applications, including biomaterial developments, drug delivery, tissue engineering, and regenerative medicine. Unlike more conventional titles on composite materials, this book covers the most innovative new developments in nanofiber-based composites, including polymers, ceramics, and metals, with particular emphasis on their preparation and characterization methodology. Selected case studies illustrate new developments in clinical and preclinical use, making the information critical for the development of new medical materials and systems for use in human health care, and for the exploration of new design spaces based on these nanofibers. This book is essential reading for those working in biomedical science and engineering, materials science, nanoscience, biomedical nanotechnology, and biotechnology.

Extensive experimentation and high failure rates are a well-recognised downside to the drug discovery process, with the resultant high levels of inefficiency and waste producing a negative environmental impact. Sustainable and Green Approaches in Medicinal Chemistry reveals how medicinal and green chemistry can work together to directly address this issue. After providing essential context to the growth of green chemistry in relation to drug discovery in Part 1, the book goes on to identify a broad range of practical methods and synthesis techniques in Part 2. Part 3 reveals how medicinal chemistry techniques can be used to improve efficiency, mitigate failure and increase the environmental benignity of the entire drug discovery process, whilst Parts 4 and 5 discuss natural products and microwave-induced chemistry. Finally, the role of computers in drug discovery is explored in Part 6.

This book presents a detailed overview of the development of new viral vector-based vaccines before discussing two major applications: preventive vaccines for infectious diseases and therapeutic cancer vaccines. Viral vector-based vaccines hold a great potential for development into successful pharmaceutical products and several examples at the advanced pre-clinical or clinical stage are presented. Nevertheless, the most efforts were focused on novel and very innovative technologies for new generation of vector-based vaccines. Furthermore, specific topics such as delivery and adjuvant and protection strategies for cell-mediated-based vaccines are presented. Given its scope, the book is a "must read" for all those involved in vaccine development, both in academia and industrial vaccine development.

The complexity of biological systems has intrigued scientists from many disciplines and has given birth to the highly influential field of systems biology wherein a wide array of mathematical techniques, such as flux balance analysis, and technology platforms, such as next generation sequencing, is used to understand, elucidate, and predict the functions of complex biological systems.  More recently, the field of synthetic biology, i.e., de novo engineering of biological systems, has emerged. Scientists from various fields are focusing on how to render this engineering process more predictable, reliable, scalable, affordable, and easy.  Systems and control theory is a branch of engineering and applied sciences that rigorously deals with the complexities and uncertainties of interconnected systems with the objective of characterising fundamental systemic properties such as stability, robustness, communication capacity, and other performance metrics. Systems and control theory also strives to offer concepts and methods that facilitate the design of systems with rigorous guarantees on these properties. Over the last 100 years, it has made stellar theoretical and technological contributions in diverse fields such as aerospace, telecommunication, storage, automotive, power systems, and others. Can it have, or evolve to have, a similar impact in biology? The chapters in this book demonstrate that, indeed, systems and control theoretic concepts and techniques can have a significant impact in systems and synthetic biology.  Volume I provides a panoramic view that illustrates the potential of such mathematical methods in systems and synthetic biology.  Recent advances in systems and synthetic biology have clearly demonstrated the benefits of a rigorous and systematic approach rooted in the principles of systems and control theory - not only does it lead to exciting insights and discoveries but it also reduces the inordinately lengthy trial-and-error process of wet-lab experimentation, thereby facilitating significant savings in human and financial resources.  In Volume I, some of the leading researchers in the field of systems and synthetic biology demonstrate how systems and control theoretic concepts and techniques can be useful, or should evolve to be useful, in order to understand how biological systems function.  As the eminent computer scientist Donald Knuth put it, "biology easily has 500 years of exciting problems to work on". This edited book presents but a small fraction of those for the benefit of (1) systems and control theorists interested in molecular and cellular biology and (2) biologists interested in rigorous modelling, analysis and control of biological systems.

This comprehensive text presents a rigorous framework from within which regulators can respond strategically to the claim by the pharmaceutical industry that lower drug prices today lead to a loss for the population's future health due to less innovation. It starts with a critical review of the empirical evidence of the return to consumers on their ongoing investment into high drug prices in order to increase future innovation. The implicit, critical and unrealistic assumption inherent in these studies is identified, namely that the health budget can be expanded to purchase drugs at higher prices without an opportunity cost, for example, the foregone benefits of alternative investments in health care infrastructure. Price effectiveness analysis (PEA), is introduced. PEA informs the question of how the innovative surplus from the new drug should be allocated between the manufacturer and the consumer so as to optimise society's welfare. The method allows the decisions by the regulator and the firm to be analysed jointly by specifying the firm's production and revenue functions in terms of the clinical innovation of a new drug; the incremental effect used in the summary metric of cost effectiveness analysis. An economic value of innovation that takes into account opportunity cost under conditions of economic efficiency in the health system is proposed: the health shadow price. The limitations of the non-strategic methods that currently inform the highly contested new drug subsidy game are presented and the relative strengths of PEA are demonstrated. Health technology assessment quantifies both the clinical innovation of a new drug and its financial impact on the health system. Cost effectiveness analysis tests the relationship between the incremental cost and incremental effect of a new drug for target patients, at a given price. PEA tests the relationship between the price of a new drug and the health of the whole population, now and into the future. It achieves this by taking into account current inefficiency in both resource allocation and the displacement process, and the relationship between price and future innovation.

Dynamic Treatment Regimes: Statistical Methods for Precision Medicine provides a comprehensive introduction to statistical methodology for the evaluation and discovery of dynamic treatment regimes from data. Researchers and graduate students in statistics, data science, and related quantitative disciplines with a background in probability and statistical inference and popular statistical modeling techniques will be prepared for further study of this rapidly evolving field. A dynamic treatment regime is a set of sequential decision rules, each corresponding to a key decision point in a disease or disorder process, where each rule takes as input patient information and returns the treatment option he or she should receive. Thus, a treatment regime formalizes how a clinician synthesizes patient information and selects treatments in practice. Treatment regimes are of obvious relevance to precision medicine, which involves tailoring treatment selection to patient characteristics in an evidence-based way. Of critical importance to precision medicine is estimation of an optimal treatment regime, one that, if used to select treatments for the patient population, would lead to the most beneficial outcome on average. Key methods for estimation of an optimal treatment regime from data are motivated and described in detail. A dedicated companion website presents full accounts of application of the methods using a comprehensive R package developed by the authors. The authors' website www.dtr-book.com includes updates, corrections, new papers, and links to useful websites.

The Medical Review Officer's Guide to Drug Testing makes it easy to understand current federal guidelines and select the best approaches for your needs. Tables, checklists, and record-keeping forms help you standardize your drug testing operations. This reference also reviews federal drug testing regulations, describes drug testing procedures and addresses risk management strategies.

This volume presents the proceedings of the 1st World Congress on Electroporation and Pulsed Electric Fields in Biology, Medicine and Food & Environmental Technologies (WC2015). The congress took place in Portoroz, Slovenia, during the week of September 6th to 10th, 2015. The scientific part of the Congress covered different aspects of electroporation and related technologies and included the following main topics: * Application of pulsed electric fields technology in food: challenges and opportunities * Electrical impedance measurement for assessment of electroporation yield * Electrochemistry and electroporation * Electroporation meets electrostimulation * Electrotechnologies for food and biomass treatment * Food and biotechnology applications * In vitro electroporation - basic mechanisms * Interfacial behaviour of lipid-assemblies, membranes and cells in electric fields * Irreversible electroporation in clinical use * Medical applications: electrochemotherapy * Medical applications: gene therapy * Non-electric field-based physical methods inducing cell poration and enhanced molecule transfer * Non-thermal plasmas for food safety, environmental applications and medical treatments * PEF for the food industry: fundamentals and applications * PEF proce ss integration - complex process chains and process combinations in the food industry * Predictable animal models * Pulsed electric fields and electroporation technologies in bioeconomy * Veterinary medical applications

Discussing existing techniques, ongoing research, new approaches, and basic concepts in the treatment of nail diseases, Topical Nail Products and Ungual Drug Delivery summarizes the current knowledge and represents a jumping-off point for creating novel techniques of drug delivery across the nail. A growing area of pharmaceutical research, the search for efficient delivery techniques that can cross the nail barrier has yielded several effective strategies, brought together in this book. Topics Include Nail anatomy, physiology, diseases, and treatment Permeability of the nail plate Topical nail formulations Approaches to enhance ungual and trans-ungual drug delivery In vitro and in vivo models to evaluate topical nail formulations Nail sampling techniques Bioengineering of the nail Trans-ungual iontophoresis Pharmacokinetics of drugs in the nail apparatus Onychopharmacokinetics: proposed model insight Photodynamic therapy of nail diseases The nail as a surrogate for investigating drug use

Essential Elements for a GMP Analytical Chemistry Department is a systematic approach to understanding the essential elements required for a successful GMP Analytical Department to function as an efficient and effective organization. It describes in detail a department structure which allows for the necessary processes to become available to all its personnel in a way where there is a free flow of information and interaction. The environment and culture created by this approach encourages and rewards the sharing of ideas, skills, and abilities among department personnel. The essential elements such as , SOP's, regulatory guidance's/guidelines, project teams, technical and department processes, personnel motivation, outsourcing, and hiring the best is among the many topics that are discussed in detail and how they can be implemented to build an efficient and effective Analytical Department. This book will serve as a valuable asset to the many companies required to perform GMP analytical method development, validation, analyses etc including start-up, virtual, and generic pharmaceutical companies.

The book provides an overview of current trends in biotechnology and medicinal plant sciences. The work includes detailed chapters on various advance biotechnological tools involved in production of phytoactive compounds of medicinal significance. Some recent and novel research studies on therapeutic applications of different medicinal plants from various geographical regions of the world have also been included. These studies report the antimicrobial activity of various natural plant products against various pathogenic microbial strains. Informative chapters on recent emerging applications of plant products such as source for nutraceuticals and vaccines have been integrated to cover latest advances in the field. This book also explores the conservation aspect of medicinal plants. Thus, chapters having comprehensively complied in vitro conservation protocols for various commercially important rare, threatened and endangered medicinal plants were provided in the present book.

Modified Cyclodextrins for Chiral Separation offers a review of the latest advances in developing modified cyclodextrins as chiral selectors for various chromatographic and electromigration techniques. Over the years, many descriptions of chiral separation have appeared in academic journals and books, but most of them have been devoted to either the development of analytical methods and protocols or the summary of different chiral selectors, including cyclodextrins for chiral separation applications. This is in marked contrast to this volume which focuses on the research endeavors concerning the development of cyclodextrin derivatives specifically as either chiral mobile phases for capillary electrophoresis, or chiral stationary phases for various chromatographic techniques including gas chromatography, or high-performance liquid chromatography and supercritical fluid chromatography. The ongoing thread in this book is the synthesis of structurally-defined cyclodextrin derivatives and their applications in enantiomer separation by means of different analytical techniques. Modified Cyclodextrins for Chiral Separation is intended for those who are interested in expanding their knowledge of cyclodextrin chemistry and chiral separation, and in what cyclodextrin modification can be made to suit the needs of chiral selectors for different analytical techniques. It primarily focuses on the state-of-the-art cyclodextrin chemistry which is the basis for all chiral selectors used in these chiral separation techniques. Weihua Tang, PhD, is a professor at the Key Laboratory of Soft Chemistry and Functional Materials, Ministry of Education, Nanjing University of Science and Technology, China. Siu-Choon Ng, PhD, is a professor at the Division of Chemical and Biomedical Engineering, School of Chemical and Biomedical Engineering, Nanyang Technological University, Singapore. Dongping Sun, PhD, is a professor at the Key Laboratory of Soft Chemistry and Functional Materials, Ministry of Education, Nanjing University of Science and Technology, China.

This book highlights current approaches and future trends in the use of mass spectrometry to characterize protein therapies. As one of the most frequently utilized analytical techniques in pharmaceutical research and development, mass spectrometry has been widely used in the characterization of protein therapeutics due to its analytical sensitivity, selectivity, and specificity. This book begins with an overview of mass spectrometry techniques as related to the analysis of protein therapeutics, structural identification strategies, quantitative approaches, followed by studies involving characterization of process related protein drug impurities/degradants, metabolites, higher order structures of protein therapeutics. Both general practitioners in pharmaceutical research and specialists in analytical sciences will benefit from this book that details step-by-step approaches and new strategies to solve challenging problems related to protein therapeutics research and development.

The blurring of boundaries between hitherto distinct scientific disciplines, technologies or markets is a common and powerful phenomenon. Subjects of this convergence often change consumer behaviours, favouring products and platforms with multiple functions. The Anticipation of Converging Industries provides a detailed focus on the triggers, drivers and consequences of convergence to create a more concise definition of convergence. This detailed analysis includes a specifically developed toolbox for 'convergence foresight', creating a forecasting method for convergence trends. With the focus on the chemical, biotechnological and pharmaceutical industries, several indicators of convergence in the areas of Nutraceuticals/Functional Foods, Cosmeceuticals and ICT are derived from samples including over 1million patents and scientific publications. By supporting this methodical approach with real world data, The Anticipation of Converging Industries is perfect for industry practitioners looking for a competitive edge in the present and for the future. Similarly, academics will find a comprehensive theoretical concept for better understanding the underlying rationale of convergence at their disposal

Renowned experts give all essential aspects of the techniques and applications of graft copolymers based on polysaccharides. Polysaccharides are the most abundant natural organic materials and polysaccharide based graft copolymers are of great importance and widely used in various fields. Natural polysaccharides have recently received more attention due to their advantages over synthetic polymers by being non-toxic, biodegradable and available at low cost. Modification of polysaccharides through graft copolymerization improves the properties of polysaccharides. Grafting is known to improve the characteristic properties of the backbones. Such properties include water repellency, thermal stability, flame resistance, dye-ability and resistance towards acid-base attack and abrasion. Polysaccharides and their graft copolymers find extensive applications in diversified fields. Applications of modified polysaccharides include drug delivery devices, controlled release of fungicides, selective water absorption from oil-water emulsions, purification of water etc.