Recombinant protein drugs are intimately associated with the impressive success story of the Biotech Industry during the past thirty years, some of them belonging to the most successful pharmaceutical products. More than thirty different proteins are available for a variety of clinical applications, over 300 proteins are presently being evaluated in clinical trials. In this new volume of the MDT series, historical, technical and clinical aspects of recombinant protein drug discovery and development are presented, covering past, present and future highlights. Leading scientists and co-founders of early Biotech companies describe technical breakthroughs and the fascinating story of pioneering discoveries, as well as the long way of translating them into products and business. Therefore, this book represents an exciting documentation of the beginning of a new era in the pharmaceutical industry. In addition, scientists from basic research, clinic and industry actively involved in new developments discuss...

We have surpassed the omics era and are truly in the Age of Molecular Therapeutics. The fast-paced development of SARS-CoV-2 vaccines, such as the mRNA vaccines encoding the viral spike protein, demonstrated the need for and capability of molecular therapy and nanotechnology-based solutions for drug delivery. In record speed, the SARS-CoV-2 viral RNA genome was sequenced and shared with the scientific community, allowing the rapid design of molecular therapeutics. The mRNA vaccines exploit the host cell endoplasmic reticulum to produce viral spike proteins for antigen presentation and recognition by the innate and adaptive immune system. Lipid nanoparticles enable the delivery of the fragile, degradation-sensitive nucleic acid payloads. Molecular-based therapeutics and nanotechnology solutions continue to drive the scientific and medical response to the COVID-19 pandemic as new mRNA, DNA, and protein-based vaccines are developed and approved and the emergency use approved vaccines are rapidly manufactured and distributed throughout the globe. The need for molecular therapies and drug delivery solutions is clear, and as these therapies progress and become more specialized there will be important advancements in organelle targeting. For example, using organelle targeting to direct lipid nanoparticles with mRNA payloads to the endoplasmic reticulum would increase the efficacy of mRNA vaccines, reducing the required dose and therefore the biomanufacturing demand. Likewise, improving the delivery of DNA therapeutics to the nucleus would improve efficacy. Organelles and molecules have always been drug targets, but until recently we have not had the tools or capability to design and develop such highly specific therapeutics. Organelle targeting has far-reaching implications. For example, mitochondria are central to both energy production and intrinsic apoptosis. Effectively targeting and manipulating mitochondria has therapeutic applications for diseases such as myopathies, cancer, neurodegeneration, progerias, diabetes, and the natural aging process. The SARS-CoV-2 vaccines that exploit the endoplasmic reticulum (for mRNA vaccines) and the nucleic translational process (DNA vaccines) attest to the need for organelle and molecular therapeutics. This book covers the status, demand, and future of organelle- and molecularly targeted therapeutics that are critical to the advancement of modern medicine. Organelle and molecular targeting is the drug design and drug delivery approach of today and the future; understanding this approach is essential for students, scientists, and clinicians contributing to modern medicine.

Global simultaneous development is becoming more necessary as the cost of developing medical products continues to grow. The strategy of using multiregional clinical trials (MRCTs) has become the preferred method for developing new medicines. Implementing the same protocol to include subjects from many geographical regions around the world, MRCTs can speed up the patient enrolment, thus resulting in quicker drug development and obtaining faster approval of the drug globally. After the publication of the editors' first volume on this topic, there have been new developments on MRCTs. The International Council for Harmonisation (ICH) issued ICH E17, a guideline document on MRCTs, in November 2017, laying out principles on MRCTs. Beyond E17, new methodologies have been developed as well. Simultaneous Global New Drug Development: Multi-Regional Clinical Trials after ICH E17 collects chapters providing interpretations of principles in ICH E17 and new ideas of implementing MRCTs. Authors are from different regions, and from academia and industry. In addition, in contrast to the first book, new perspectives are brought to MRCT from regulatory agencies. This book will be of particular interest to biostatisticians working in late stage clinical development of medical products. It will also be especially helpful for statisticians in regulatory agencies, and medical research institutes. This book is comprehensive across the MRCT topic spectrum, including Issues regarding ICH E17 Implementation MRCT Design and Analysis Methodologies Perspectives from authorities in regulatory agencies, as well as statisticians practicing in the medical product industry Many examples of real-life applications based on actual MRCTs.

Covers four pillars of safety statistics: cross-disciplinary scientific engagement, effective and efficient operational process, visual analytics, and intelligent data architecture Links safety monitoring to benefit risk evaluation Presents an emerging topic that links to ICH E19 and TransCelerate safety efforts

Design and Analysis of Cross-Over Trials is concerned with a specific kind of comparative trial known as the cross-over trial, in which subjects receive different sequences of treatments. Such trials are widely used in clinical and medical research, and in other diverse areas such as veterinary science, psychology, sports science, and agriculture. The first edition of this book was the first to be wholly devoted to the subject. The second edition was revised to mirror growth and development in areas where the design remained in widespread use and new areas where it had grown in importance. This new Third Edition: Contains seven new chapters written in the form of short case studies that address re-estimating sample size when testing for average bioequivalence, fitting a nonlinear dose response function, estimating a dose to take forward from phase two to phase three, establishing proof of concept, and recalculating the sample size using conditional power Employs the R package Crossover, specially created to accompany the book and provide a graphical user interface for locating designs in a large catalog and for searching for new designs Includes updates regarding the use of period baselines and the analysis of data from very small trials Reflects the availability of new procedures in SAS, particularly proc glimmix Presents the SAS procedure proc mcmc as an alternative to WinBUGS for Bayesian analysis Complete with real data and downloadable SAS code, Design and Analysis of Cross-Over Trials, Third Edition provides a practical understanding of the latest methods along with the necessary tools for implementation.

The over-riding premise for biotechnology in this book is bringing novel products to market to substantially advance patient care and disease mitigation. Biotechnology, over its relatively brief existence of 40 years, has experienced a mercurial growth. The vast educational need for biotechnology information in this rapidly burgeoning field is a basic rationale here. However a more prominent underpinning is that, bringing biotech products to market for patient care involves success in the following four areas of engagement simultaneously - scientific advances for healthcare technologies, novel and varied products for untreated diseases, regulatory authorities, and biotech companies. Features Comprehensive coverage of biotechnology science topics used in development and manufacturing Addresses all the scientific technologies within biotechnology responsible for products on the market and the pipeline Presents business issues such as marketing and sales of the products, as well as companies engaged, and how biotech business has evolved

Phylogenetic presentation of medicinal plants and a chemotaxonomical rationale of antiviral, antibacterial, and antifungal action. Discusses chemical structure-activity relationship, pharmacokinetics, and oral bioavailability of antimicrobial principles Introduces the molecular mechanism of natural products on viruses, bacteria, and fungi. Contains a selection of botanical plates and useful bibliographic references A useful research tool for postgraduates, academics, and the pharmaceutical, herbal, or nutrition industries.

Provide readers and front line health care products manufacturers, R&D management and biotech laboratories all the information they need to know to develop a GMP oriented industry with trained and skilled personnel and manufacture products that meet GMP and regulatory requirements Covers basic quality concepts and the elements of vendor's assessment, qualification and approval required by the pharmaceutical educational institutions and professional certification bodies Provides stepwise guidance on how to evaluate, audit, qualify and approve an API and packaging material manufacturer and supplier to enhance the GMP within the industry Provides ready to use regulatory documentation, e.g. letter of commitment, Questionnaire, SOP etc. required for API and Packaging Materials contract Provided material can be easily tailored to incorporate changes to add in-house vendor's qualification requirements

Provides information about the different surfactants. Describes constraints in production and novel production strategies. Includes application oriented aspects of biosurfactants in various fields such as agriculture, Environmental, Pharmaceutical, and Food industry. Authored by experts in the field of biosurfactants.

Drug products are complex mixtures of drugs and excipients and, as such, their chemical and physical stability kinetics are complex. This book discusses the stability of these dosage forms with preformulation studies through to the studies on the final products. The book is intended for graduate students, researchers and professionals in the field of Pharmaceutics and Pharmaceutical Chemistry.

A contribution to the series on Natural Products Chemistry of Global Plants, Natural Products Chemistry of Botanical Medicines from Cameroon focuses on the sources and chemistry of natural products from plants in Cameroon, West Africa. The plants selected offer an opportunity to trace a route through history from ancient civilizations to the modern day, showing the important value to man of natural products in medicines and in foods. This book highlights how many of the extracts from Cameroon are today associated with important drugs, nutrition products, beverages, perfumes, cosmetics and pigments, as well as presenting their complex chemistry and structure. Key Features: Forms an important part of the series on Natural Products Chemistry of Global Plants, as Cameroon is a country with rich experience in the use of medicinal plants and with a wide diversity of botanical resources Addresses the current development of pharmacognosy research in Cameroon Provides readers with updated information on the chemistry and pharmacology of natural products with pharmaceutical potential Covers an extensive range of chemical, botanical and pharmacological diversities Xavier Siwe Noundou is a Scholar/Scientist based at Rhodes University in Grahamstown, South Africa. He has been a EU FP7 Marie Curie Fellow (2015-2016), Kaposvar University in Hungary (2015, 2016), Trakia Univesity in Bulgaria (2016), TWAS Fellow (2013), National Research Foundation South Africa Fellow (2014-2016). Dr Noundou works on Medicinal Chemistry focusing on Chemistry, Pharmacognosy and Nanotechnology. His main research interests include terrestrial natural products chemistry (from Cameroon and South Africa) and marine natural products chemistry (from the South African coastline): bioactive metabolites isolated as potential antiparasitic, antimicrobial, antiviral and antiproliferative candidates. He is author of more than forty scientific publications in his field of expertise.

The cost for bringing new medicine from discovery to market has nearly doubled in the last decade and has now reached $2.6 billion. There is an urgent need to make drug development less time-consuming and less costly. Innovative trial designs/ analyses such as the Bayesian approach are essential to meet this need. This book will be the first to provide comprehensive coverage of Bayesian applications across the span of drug development, from discovery, to clinical trial, to manufacturing with practical examples. This book will have a wide appeal to statisticians, scientists, and physicians working in drug development who are motivated to accelerate and streamline the drug development process, as well as students who aspire to work in this field. The advantages of this book are: Provides motivating, worked, practical case examples with easy to grasp models, technical details, and computational codes to run the analyses Balances practical examples with best practices on trial simulation and reporting, as well as regulatory perspectives Chapters written by authors who are individual contributors in their respective topics Dr. Mani Lakshminarayanan is a researcher and statistical consultant with more than 30 years of experience in the pharmaceutical industry. He has published over 50 articles, technical reports, and book chapters besides serving as a referee for several journals. He has a PhD in Statistics from Southern Methodist University, Dallas, Texas and is a Fellow of the American Statistical Association. Dr. Fanni Natanegara has over 15 years of pharmaceutical experience and is currently Principal Research Scientist and Group Leader for the Early Phase Neuroscience Statistics team at Eli Lilly and Company. She played a key role in the Advanced Analytics team to provide Bayesian education and statistical consultation at Eli Lilly. Dr. Natanegara is the chair of the cross industry-regulatory-academic DIA BSWG to ensure that Bayesian methods are appropriately utilized for design and analysis throughout the drug-development process.

This book provides insight into the world of pharmaceutical quality systems and the key elements that must be in place to change the business and organizational dynamics from task-oriented procedure-based cultures to truly integrated quality business systems that are self-detecting and correcting. Chapter flow has been changed to adopt a quality systems organization approach, and supporting chapters have been updated based on current hot topics including the impact of the worldwide supply chain complexity and current regulatory trends. Key Features: Presents insight into the world of pharmaceutical quality systems Analyzes regulatory trends and expectations Includes approaches and practices used in the industry to comply with regulatory requirements Discusses recent worldwide supply chain issues Delivers valuable information to a worldwide audience regarding the current GMP practices in the industry

Recent Advances in the Science of Cannabis describes progress in a variety of significant areas of cannabis science. This unique book covers topics in cultivation and secondary metabolites, aroma and chemotypes, cannabinoid structures, physiology and pharmacology, as well as the development of unique topical products. State-of-the-art analytical methods and instrumentation are covered, including current developments in mass spectrometry and chromatography, as well as microbial testing. Given the popularity of smoking and vaporizing cannabis, the chemistry of vaping cannabinoid and terpene concentrates is also presented, along with emerging regulatory issues. Key Features: A guide to emerging modern cannabis technology in a dynamic regulatory climate and appealing to both novices and specialists. Building upon pioneering studies of terpene and cannabinoid chemistry, this distinctive volume describes current best practices, technological breakthroughs and historical context. Written by researchers in industry and academia, a greater understanding of the risks of exposure to emissions from vaping or dabbing cannabis concentrates is provided here. A selection of the book content reviewing Thermal Degradation of Cannabinoids and Cannabis Terpenes has been included in "Hot 2021" RSC Advances.

Phylogenetic presentation of medicinal plants and a chemotaxonomical rationale of antiviral, antibacterial, and antifungal action. Discusses chemical structure-activity relationship, pharmacokinetics, and oral bioavailability of antimicrobial principles Introduces the molecular mechanism of natural products on viruses, bacteria, and fungi. Contains a selection of hand-made botanical plates and useful bibliographic references A useful research tool for postgraduates, academics, and the pharmaceutical, herbal, or nutrition industries.

Confidence Intervals for Discrete Data in Clinical Research is designed as a toolbox for biomedical researchers. Analysis of discrete data is one of the most used yet vexing areas in clinical research. The array of methodologies available in the literature to address the inferential questions for binomial and multinomial data can be a double-edged sword. On the one hand, these methods open a rich avenue of exploration of data; on the other, the wide-ranging and competing methodologies potentially lead to conflicting inferences, adding to researchers' confusion and frustration and also leading to reporting bias. This book addresses the problems that many practitioners experience in choosing and implementing fit for purpose data analysis methods to answer critical inferential questions for binomial and count data. The book is an outgrowth of the authors' collective experience in biomedical research and provides an excellent overview of inferential questions of interest for binomial proportions and rates based on count data, and reviews various solutions to these problems available in the literature. Each chapter discusses the strengths and weaknesses of the methods and suggests practical recommendations. The book's primary focus is on applications in clinical research, and the goal is to provide direct benefit to the users involved in the biomedical field.

Withania is a genus of the nightshade family of flowering plants distributed in the subtropical regions from the Mediterranean to South East Asia. Only two species, W. somnifera and W. coagulans, are found in India. The most common species is W. somnifera (WS), which occurs naturally in the subtropical regions from the Mediterranean through Africa to the Middle East, the Indian Continent, Sri Lanka, South East Asia, subtropical America and Australia. It is a perennial shrub that grows to 75 cm (.75 m) tall with tomentose branches, oval yellowish green leaves, orange red berries and a papery calyx, and it survives harsher climatic conditions. In Ayurveda it is believed the plants which survive harsh conditions have strong healing and tonification properties. The main bioactive phytoconstituents of WS are withanolides (steroidal lactones), alkaloids, flavonoids, sterols, phenolics and others. Among the various withanolides, withanolide A, withaferin A, withanone and withanolide D are the most abundant, having various activities. WS is a wonder herb with a broad spectrum of pharmacological properties, such as antioxidant, antidepressant, aphrodisiac, antiulcerogenic, antivenom, anti-inflammatory, antiarthritic, anticancer, antiparasitic, antimicrobial, anticancerous, antidiabetic, antitumor, hemopoetic neuroregenerative, immunomodulatory, cardioprotective, radio-sensitizing, rejuvenating, antistress, sedative, hypoglycemic, thyroprotective, adaptogenic, antispasmodic, immunomodulatory, immunostimulant and antiaging properties. The simultaneous quantitative analysis of six major bioactive withanolides in five varieties of WS and in different plant parts (root, stem and leaf) of WS was accomplished. This method is also applicable to control the quality of commercially formulated products which contain WS bioactive compounds. Results indicated the WS variety NMITLI-135 showed the maximum abundance of withanolides at pH 8.5, EC-0.5 dS m 1, ESP-13 in sodic soil. Our results showed this readily available, rapid and reliable method is suitable for the routine analysis and effective quality control of raw materials and finished products. FEATURES Presents a collection of Ayurvedic features and scientific analytical and pharmacological evidence of important medicinal plants of Withania somnifera Useful for natural product researchers, faculty, students and herbal product manufacturers Uses advanced hyphenated techniques for assessing phytoconstituents

Describes 'real life' examples in Medicinal Chemistry. Integrates the use of physical, chemical, and biological concepts that are important in drug design. Highlights the "ips" and "downs" that come with drug discovery. Aims to inspire students who may be struggling with the challenges and thought process in drug design. Intends to be an excellent companion text, illustrating real life experiences, for graduate and postgraduate students.

Coherent treatment of a variety of approaches to multiple comparisons Broad coverage of topics, with contributions by internationally leading experts Detailed treatment of applications in medicine and life sciences Suitable for researchers, lecturers / students, and practitioners

Covers recent research on the following aspects of vitamin B12: structure, constitution, properties, nomenclature, organometallic reactivity, usage in diagnosis and therapy, and role in biological systems Discusses the chemistry of the carbon cobalt bond Is illustrated throughout with excellent figures and references in each chapter

Vitamin C holds a unique place in scientific and cultural history. In this book, a group of leading scientific researchers describe new insights into the myriad ways vitamin C is employed during normal physiological functioning. In addition, the text provides an extensive overview of the following: the rationale for utilizing vitamin C in the clinic, updates on recent uses of vitamin C in cancer treatment through high-dose intravenous therapies, the role vitamin C plays in the treatment of sepsis and infectious disease, management of the ways vitamin C can improve stem cell differentiation, as well as vitamin C use in other important health situations. Features Includes chapters from a team of leading international scholars Reviews the history and recent research on the functions, benefits, and uses of vitamin C Focuses special attention on the way vitamin C can be used in the treatment of cancers Discusses how vitamin C can be employed against infectious disease

A contribution to the series on Natural Products Chemistry of Global Plants, Natural Products Chemistry of Botanical Medicines from Cameroon focuses on the sources and chemistry of natural products from plants in Cameroon, West Africa. The plants selected offer an opportunity to trace a route through history from ancient civilizations to the modern day, showing the important value to man of natural products in medicines and in foods. This book highlights how many of the extracts from Cameroon are today associated with important drugs, nutrition products, beverages, perfumes, cosmetics and pigments, as well as presenting their complex chemistry and structure. Key Features: Forms an important part of the series on Natural Products Chemistry of Global Plants, as Cameroon is a country with rich experience in the use of medicinal plants and with a wide diversity of botanical resources Addresses the current development of pharmacognosy research in Cameroon Provides readers with updated information on the chemistry and pharmacology of natural products with pharmaceutical potential Covers an extensive range of chemical, botanical and pharmacological diversities Xavier Siwe Noundou is a Scholar/Scientist based at Rhodes University in Grahamstown, South Africa. He has been a EU FP7 Marie Curie Fellow (2015-2016), Kaposvar University in Hungary (2015, 2016), Trakia Univesity in Bulgaria (2016), TWAS Fellow (2013), National Research Foundation South Africa Fellow (2014-2016). Dr Noundou works on Medicinal Chemistry focusing on Chemistry, Pharmacognosy and Nanotechnology. His main research interests include terrestrial natural products chemistry (from Cameroon and South Africa) and marine natural products chemistry (from the South African coastline): bioactive metabolites isolated as potential antiparasitic, antimicrobial, antiviral and antiproliferative candidates. He is author of more than forty scientific publications in his field of expertise.

Drug Discovery with Privileged Building Blocks traces back PharmaBlock's founding philosophy of designing privileged building blocks. High-quality building blocks are crucial not only to biological activities of different molecules but also to ADMET properties, which eventually will impact the success rate of drug discovery projects. A thorough study of how building blocks perform in drug molecules and a regular analysis of new building block structures in the latest researches have proven to be a fruitful strategy to generate novel building blocks. Using this strategy, PharmaBlock has supplied the drug industry with a great number of building blocks, which are increasingly being adopted by drug hunters, and these are identified in this book. Each chapter may be read and studied without learning the previous chapters. This book will be a good starting point for novice medicinal chemists, and veteran medicinal chemists will find it useful as well. Key Feature The book covers privileged building blocks appearing most frequently on patents for novel drugs. The latest relevant tactics are explained in the context of drug design and medicinal chemistry. Key synthesis, especially large-scale synthesis, is described. The most recent literature references are cited.

Drug Discovery with Privileged Building Blocks traces back PharmaBlock's founding philosophy of designing privileged building blocks. High-quality building blocks are crucial not only to biological activities of different molecules but also to ADMET properties, which eventually will impact the success rate of drug discovery projects. A thorough study of how building blocks perform in drug molecules and a regular analysis of new building block structures in the latest researches have proven to be a fruitful strategy to generate novel building blocks. Using this strategy, PharmaBlock has supplied the drug industry with a great number of building blocks, which are increasingly being adopted by drug hunters, and these are identified in this book. Each chapter may be read and studied without learning the previous chapters. This book will be a good starting point for novice medicinal chemists, and veteran medicinal chemists will find it useful as well. Key Feature The book covers privileged building blocks appearing most frequently on patents for novel drugs. The latest relevant tactics are explained in the context of drug design and medicinal chemistry. Key synthesis, especially large-scale synthesis, is described. The most recent literature references are cited.

An introductory but detailed treatise which includes some 1,000 references and solved examples and end-of-chapter problems, making it useful to both students and practitioners. The pharmokinetics, pharmacodynamics, and biological and biopharmaceutical parameters pertinent to each route of administra