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| biomarin hemophilia gene therapy: A Rare Breed Daniel S. Levine, Daniel P. Maher, 2017-08-15 BioMarin Pharmaceutical Inc., named by Forbes magazine as one of the world¿s ten most innovative companies, is a world leader in developing therapies to treat rare orphan genetic diseases. A Rare Breed recounts the first 20 years of its history, the struggles it faced in developing drugs for diseases that represented markets most pharmaceutical companies saw as too small to pursue, and how it grew into a global enterprise valued at more than $16 billion. It follows the researchers, patients, and executives who played a role in taking novel ideas forward and how they overcame scientific, regulatory, and financial challenges to bring breakthrough therapies to children who needed them. |
| biomarin hemophilia gene therapy: Second Generation Cell and Gene-Based Therapies Alain Vertes, Nathan J. Dowden, Devyn Smith, Nasib Qureshi, 2020-02-07 Second Generation Cell and Gene-Based Therapies: Biological Advances, Clinical Outcomes, and Strategies for Capitalisation serves as the only volume to the market to bridge basic science, clinical therapy, technology development, and business in the field of cellular therapy/cytotherapy. After more than two decades of painstaking fundamental research, the concept of therapeutic cells (stem cells, genes, etc.), beyond the concept of vaccines, is reaching clinical trial, with mounting confidence in the safety and efficacy of these products. Nonetheless, numerous incremental technical advances remain to be achieved. Thus, this volume highlights the possible R&D paths, which will ultimately facilitate clinical delivery of cutting edge curative products.The next waves of innovation are reviewed in depth for hematopoietic stem cells, mesenchymal stem cells, tissue engineering, CAR-T cells, and cells of the immune system, as well as for enabling technologies such as gene and genome editing. Additionally, deep dives in product fundamentals, history of science, pathobiology of diseases, scientific and technological bases, and financing and technology adoption constraints are taken to unravel what will shape the cytotherapy industry to the horizon 2025 and beyond. The outcome is not simply a scientific book, but a global perspective on the nascent field combining science, business, and strategic fundamentals. - Helps readers learn about the most current trends in cell-based therapy, their overall effectiveness from a clinical prospective, and how the industry is moving therapies forward for capitalization - Perspectives section at the end of each chapter summarizes key learnings, hypotheses, and objectives highlighted and combines scientific and business insights - Edited and authored by scientists representing both basic and clinical research and industry, presenting a complete story of the current state and future promise of cellular therapies |
| biomarin hemophilia gene therapy: Quality Control and Regulatory Aspects for Biologicals Gauri Misra, 2024-04-22 This book serves as a comprehensive guide on quality control and regulatory aspects for biological products. It covers a wide range of topics, including regulatory requirements, quality control strategies, analytical methods, and risk management. It delves into the advantages and limitations of in vivo tests and discusses alternative methods that can be employed. The book explores the use of animal-based testing methods in quality control and examines viable alternatives. Key Features: Reviews various scientific and regulatory aspects involved in the quality control of biologicals Provides an overview of the roles of various national and international regulatory bodies and accreditation agencies Presents advanced analytical methods, innovative technologies, and the integration of molecular diagnostics in quality control processes Explores the use of animal-based testing methods in quality control, as well as their alternatives Discusses guidelines and methodologies involved in the development of biological products Overall, this book is an important reference source for various professionals in the pharmaceutical industry, including researchers, scientists, quality control personnel, and regulatory affairs professionals. |
| biomarin hemophilia gene therapy: Rare Disease Drug Development Raymond A. Huml, 2021-11-08 This book provides a broad overview of rare disease drug development. It offers unique insights from various perspectives, including third-party capital providers, caregivers, patient advocacy groups, drug development professionals, marketing and commercial experts, and patients. A unique reference, the book begins with narratives on the many challenges faced by rare disease patient and their caregivers. Subsequent chapters underscore the critical, multidimensional role of patient advocacy groups and the novel approaches to related clinical trials, investment decisions, and the optimization of rare disease registries. The book addresses various rare disease drug development processes by disciplines such as oncology, hematology, pediatrics, and gene therapy. Chapters then address the operational aspects of drug development, including approval processes, development accelerations, and market access strategies. The book concludes with reflections on the authors' case for real-world data and evidence generation in orphan medicinal drug development. Rare Disease Drug Development is an expertly written text optimized for biopharmaceutical R&D experts, commercial experts, third-party capital providers, patient advocacy groups, patients, and caregivers. |
| biomarin hemophilia gene therapy: Hemophilia and Von Willebrand Disease David Green, 2018-06-14 Hemophilia and Von Willebrand Disease: Factor VIII and Von Willebrand Factor serves as a must-have reference on the important role these essential blood-clotting proteins play in research and clinical medicine. Clinicians and researchers face the daily challenge of staying current on the vast amounts of research that is now generated. The reference to Janus in the title refers to the two roles of the Factor VIII/Von Willebrand Factor Complex: initiation of coagulation and propagation of clot formation. The complex prevents bleeding in hemophilia and Von Willebrand disease but also augments arterial and venous thrombosis. - Presents one source of information on Hemophilia and Von Willebrand Disease, as well as Factor VIII and Von Willebrand Factor, eliminating the search through hundreds of journal articles - Combines the multi-disciplinary research that is generated from Factor VIII/Von Willebrand Factor – hematology, drug discovery, genetics, cell biology, and oncology - Delves into unanswered questions and future directions of this important blood-clotting complex |
| biomarin hemophilia gene therapy: The Right Price Peter J. Neumann, Joshua T. Cohen, Daniel A. Ollendorf, 2021 The prescription drug market -- Proposed solutions for rising drug prices -- Measuring the value of prescription drugs -- Measuring drug value : whose job is it anyway? -- Institute for Clinical and Economic Review (ICER) -- Other US value assessment frameworks -- Do drugs for special populations warrant higher prices? -- Improving value measurement -- Aligning prices with value -- The path forward. |
| biomarin hemophilia gene therapy: Fast Facts: Gene Therapy Roland Herzog, Linda Popplewell, 2020-04-08 Gene therapy has emerged as a discipline in medicine that can provide treatments for diseases that have no other therapies available, save lives of patients for whom there is no other hope and replace suboptimal treatments with lasting cures. 'Fast Facts: Gene Therapy' provides an overview of the field, looking at the main vector systems used to transfer the therapeutic gene constructs, the molecular mechanisms and the history of gene therapy, as well as the safety and ethical considerations of this important advance. Multiple examples of diseases that are already successfully treated with gene therapy are given, with discussion of treatments that hold promise for the future. This book will be informative and of value to health professionals, researchers, students and anyone with an interest in this exciting and fast-moving area. Contents: • Principles of gene therapy • Gene therapy techniques • Ethical and safety considerations • Gene therapies with proven clinical efficacy • Genome editing • Research directions – the next wave of treatments |
| biomarin hemophilia gene therapy: Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing Ganapathy Subramanian, 2021-12-20 Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing Explore new trends in continuous biomanufacturing with contributions from leading practitioners in the field With the increasingly widespread acceptance and investment in the ??technology, the last decade has demonstrated the utility of continuous ??processing in the pharmaceutical industry. In Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing, distinguished biotechnologist Dr. Ganapathy Subramanian delivers a comprehensive exploration of the potential of the continuous processing of biological products and discussions of future directions in advancing continuous processing to meet new challenges and demands in the manufacture of therapeutic products. A stand-alone follow-up to the editor’s Continuous Biomanufacturing: Innovative Technologies and Methods published in 2017, this new edited volume focuses on critical aspects of process intensification, process control, and the digital transformation of biopharmaceutical processes. In addition to topics like the use of multivariant data analysis, regulatory concerns, and automation processes, the book also includes: Thorough introductions to capacitance sensors to control feeding strategies and the continuous production of viral vaccines Comprehensive explorations of strategies for the continuous upstream processing of induced microbial systems Practical discussions of preparative hydrophobic interaction chromatography and the design of modern protein-A-resins for continuous biomanufacturing In-depth examinations of bioprocess intensification approaches and the benefits of single use for process intensification Perfect for biotechnologists, bioengineers, pharmaceutical engineers, and process engineers, Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing is also an indispensable resource for chemical engineers seeking a one-stop reference on continuous biomanufacturing. |
| biomarin hemophilia gene therapy: Recent highlights in molecular medicine Ignazio Castagliuolo, Frank Emmert-Streib, Leon J. De Windt, Vincenzo Cerullo, Giorgio Stassi, 2023-10-04 |
| biomarin hemophilia gene therapy: Drug Development for Gene Therapy Yanmei Lu, Boris Gorovits, 2024-02-28 Drug Development for Gene Therapy Industry-centric perspective on translational and bioanalytical challenges and best practices for gene therapies Drug Development for Gene Therapy focuses on the translational and bioanalytical challenges and best practices for gene therapy modalities, presenting a significant body of data, including information related to safety and efficacy, necessary to advance through the development pipeline into clinical use. The text covers bioanalytical methods and platforms including patient screening assays, different PCR tests, enzyme activity assays, ELISpot, NGS, LC/MS, and immunoassays, with FDA and EMA guidelines on gene therapy safety and efficacy, along with companion diagnostics regulations from US and EU perspectives. The chapters offer an in-depth discussion of the basics and best practices for translational biomarkers, bioanalysis, and developing companion diagnostics / lab tests for gene therapies in the pharma and biopharma industries. To aid in reader comprehension, the text includes clinical examples of relevant therapies in related chapters. Some of the core topics covered include study design, immunogenicity, various bioanalytical methods and their applications, and global regulatory issues. Written by two highly qualified authors with significant experience in the field, Drug Development for Gene Therapy includes information on: Bioanalytical methods to detect pre-existing antibodies against adeno-associated viruses (AAV) capsids Detection of cellular immunity and humoral response to viral capsids and transgene proteins, and immunogenicity of gene therapy products Nonclinical and clinical study considerations and methods for biodistribution and shedding Quantification of transgene protein expression and biochemical function, and substrate and distal pharmacodynamic biomarker measurements for gene therapy Detection and quantification of rAAV integration and off-target editing Current regulatory landscape for gene therapy product development and the role of biomarkers and general regulatory considerations for gene therapy companion diagnostics With comprehensive coverage of the subject, Drug Development for Gene Therapy is a must-have resource for researchers and developers in the areas of pharmaceuticals, biopharmaceuticals, and contract research organizations (CROs), along with professors, researchers, and advanced students in chemistry, biological, biomedical engineering, pharmaceuticals, and medical sciences. |
| biomarin hemophilia gene therapy: Congenital Bleeding Disorders Akbar Dorgalaleh, 2018-07-25 This book describes in detail the clinical presentation, diagnosis, and management of a wide range of congenital bleeding disorders. It will assist readers in overcoming the significant challenges involved in clinical and laboratory diagnosis and in providing effective clinical care that makes optimal use of new products, including recombinant factor concentrate. The coverage ranges from hemophilia A and B and von Willebrand disease to rare bleeding disorders such as congenital factor V, factor X, factor XI, and factor XIII deficiency and inherited platelet function disorders. The exceptional attention to rarer conditions is of particular importance given the considerable risk of overlooking them during diagnosis, with potential consequences for disease-related morbidity and mortality. The authors are acknowledged specialists in the field from across the world who have particular expertise in the disorder that they discuss. The book will be of value to hematologists, oncologists, pediatricians, laboratory specialists and technicians, general physicians, and trainees. |
| biomarin hemophilia gene therapy: Laboratory Monitoring of Gastrointestinal and Hepatobiliary Disease, An Issue of Gastroenterology Clinics of North America Stanley J Naides, 2019-05-08 In consultation with Consulting Editor, Dr. Alan Buchman, Dr. Stanley J. Naides has put together a state-of the-art issue of the Gastroenterology Clinics of North America devoted to Laboratory Monitoring of Gastrointestinal and Hepatobiliary Disease. Clinical review articles from expert authors are specifically devoted to the following: Inborn Errors of Metabolism and the GI track; Primary Immunodeficiency and the gut; Liver fibrosis determination; IBD; Autoimmune liver disease diagnosis and classification; Laboratory Diagnosis of Viral hepatitis; IgG4-RD with Emphasis on its GI Manifestation; Bowel Microbiome in Health and Disease; AAV System; and Celiac Disease. Readers will come away with the latest information they need to diagnose and monitor patients with gastrointestinal and hepatobiliary disease. |
| biomarin hemophilia gene therapy: Therapeutic Enzymes: Function and Clinical Implications Nikolaos Labrou, 2019-09-03 Therapeutic enzymes exhibit fascinating features and opportunities, and represent a significant and promising subcategory of modern biopharmaceuticals for the treatment of several severe diseases. Research and drug developments efforts and the advancements in biotechnology over the past twenty years have greatly assisted the introduction of efficient and safe enzyme-based therapies for a range of both rare and common disorders. The introduction and regulatory approval of twenty different recombinant enzymes has enabled effective enzyme-replacement therapy. This volume aims to overview these therapeutic enzymes, focusing in particular on more recently approved enzymes produced by recombinant DNA technology. This volume is composed of four sections. Section 1 provides an overview of the production process and biochemical characterization of therapeutic enzymes, while Section 2 focuses upon the engineering strategies and delivery methods of therapeutic enzymes. Section 3 highlights the clinical applications of approved therapeutic enzymes, including aspects on their structure, indications and mechanisms of action. Together with information on these mechanisms, safety and immunogenicity issues and various adverse events of the recombinant enzymes used for therapy are discussed. Section 4, provides discussion on the prospective and future developments of new therapeutic enzymes. This book is aimed at academics, researchers and students undertaking advanced undergraduate/postgraduate programs in the biopharmaceutical/biotechnology area who wish to gain a comprehensive understanding of enzyme-based therapeutic molecules. |
| biomarin hemophilia gene therapy: The Business of Healthcare Innovation Lawton Robert Burns, 2005-08-25 The Business of Healthcare Innovation is the first wide-ranging analysis of business trends in the manufacturing segment of the health care industry. In this leading edge volume, Professor Burns focuses on the key role of the 'producers' as the main source of innovation in health systems. Written by professors of the Wharton School and industry executives, this book provides a detailed overview of the pharmaceutical, biotechnology, genomics/proteomics, medical device and information technology sectors. It analyses the market structures of these sectors as well as the business models and corporate strategies of firms operating within them. Most importantly, the book describes the growing convergence between these sectors and the need for executives in one sector to increasingly draw upon trends in the others. It will be essential reading for students and researchers in the field of health management, and of great interest to strategy scholars, industry practitioners and management consultants. |
| biomarin hemophilia gene therapy: Gene Therapy, An Issue of Hematology/Oncology Clinics of North America Daniel E. Bauer, Donald B Kohn, 2017-09-27 This issue of Hematology/Oncology Clinics will focus on Gene Therapy. Topics include, but are not limited to Historical Perspective and Current Renaissance, Integrating Vectors, Nonintegrating Vectors, Gene Editing, Conditioning Therapies for Autologous HSCT, Approaches to Immunodeficiency, Approaches to Hemoglobinopathy, Approaches to Hemophilia, Hematopoietic Gene Therapies for Neurologic and Metabolic Disease, Gene Therapy Approaches to HIV and other Infectious Diseases, HSC Approaches to Cancer, and Gene Modified T Cell Therapies for Cancer. |
| biomarin hemophilia gene therapy: Genomic Politics Jennifer Hochschild, 2021-08-03 A groundbreaking analysis of how the genomic revolution is transforming American society and creating new social divisions-some along racial lines-that promise to fundamentally shape American politics for years to come. The emergence of genomic science in the last quarter century has revolutionized medicine, the justice system, and our very understanding of who we are. We use genomics to determine guilt and exonerate the convicted; devise new medicines; test embryos; and discover our ethnic and national roots. One might think that, given these advances, most would favor the availability of genomic tools. Yet as Jennifer Hochschild explains in More Science, Less Fear?, the uses of genomic science are both politically charged and hotly contested. The political divisions around genomics do not follow the usual left-right ideological divides that dominate most of American politics. Through four controversial innovations resulting from genomic science--genetically modified medicines that target African-Americans, who are demographically more susceptible to heart disease; the use of DNA evidence in the criminal justice system; the current ancestry craze; and the use of genetic tests in prenatal exams--Hochschild reveals how the phenomenon is polarizing America in novel ways. Advocates of genomic science argue that these applications will make life better, but their opponents respond by pointing out the potential for misuse--from racial profiling to selecting out fetuses that gene tests show to have conditions like Down's Syndrome. Hochschild's central message is that the divide hinges on answers to two questions: How significant are genetic factors in explaining human traits and behaviors? And what is the right balance between risk acceptance and risk avoidance for a society grappling with innovations arising from genomic science? A deeply researched and original analysis of the politics surrounding one of the signal issues of our times, this is essential reading for anyone interested in how the genetics revolution is reshaping society. |
| biomarin hemophilia gene therapy: Development of Gene Therapies Avery McIntosh, Oleksandr Sverdlov, 2024-05-23 Cell and gene therapies have become the third major drug modality in pharmaceutical medicine of the 21st century after low molecular weight and antibody drugs. The gene therapy (GTx) field is rapidly advancing, and yet there are still fundamental scientific questions that remain to be answered. Development of GTx products poses unique challenges and opportunities for drug developers. However, there is lack of a systematic exposition of the GTx product development and the pivotal role of the biostatistician in this process. Development of Gene Therapies: Strategic, Scientific, and Regulatory, and Access Considerations attempts to summarize the current state-of-the-art strategic, scientific, statistical, and regulatory aspects of GTx development. Intended to provide an exposition to the GTx new product development through peer-reviewed papers written by subject matter experts in this emerging field, this book will be useful for researchers in gene therapy drug development, biostatisticians, regulators, patient advocates, graduate students, and the finance and business development community . Key Features: A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts. An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways. An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx. Insights into commercial models, access hurdles, and health economics of gene therapies. Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma. A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective. |
| biomarin hemophilia gene therapy: The Challenge of CMC Regulatory Compliance for Biopharmaceuticals John Geigert, 2019-05-08 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 many companies whose Chemistry, Manufacturing & Control (CMC) teams have varying degrees of familiarity or experience with the CMC strategy and regulatory compliance requirements for these challenging products. Companies clearly plan out the strategy for their clinical study plans, but frequently, the development of a strategy for CMC is an afterthought. Coupled with the complexity of the biopharmaceutical manufacturing processes and products, and this can be a recipe for disaster. The third edition of this book provides insights and practical guidance for the CMC teams to develop an acceptable cost-effective, risk-based CMC regulatory compliance strategy for all biopharmaceuticals (recombinant proteins, monoclonal antibodies, genetically engineered viruses and genetically engineered human cells) from early clinical stage development through market approval. The third edition of this book provides added coverage for the biosimilars, antibody drug conjugates (ADCs), bispecific antibodies, genetically engineered viruses, and genetically engineered cells. This third edition of the book also addresses the heightened pressure on CMC regulatory compliance timelines due to the introduction of expedited clinical pathways moving the clinical development closer to a seamless phase process (e.g., FDA Breakthrough Therapy designation, CBER Regenerative Medicine Advanced Therapy (RMAT) designation, EMA Priority Medicines (PRIME) designation). The Challenge of CMC Regulatory Compliance for Biopharmaceuticals is essential, practical information for all pharmaceutical development scientists, Manufacturing and Quality Unit staff, Regulatory Affairs personnel, and senior management involved in the manufacture of biopharmaceuticals. |
| biomarin hemophilia gene therapy: Current Applications of Pharmaceutical Biotechnology Ana Catarina Silva, João Nuno Moreira, José Manuel Sousa Lobo, Hugo Almeida, 2020-02-06 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. |
| biomarin hemophilia gene therapy: Regenerative Medicine Tingting Qiu, Mondher Toumi, 2023-05-23 A comprehensive review of the challenges that exist in patient accessibility to regenerative medicines (RMs), presenting clinical trials, marketing authorization, HTA, pricing, reimbursement, affordability, payment and partnership agreements of RMs and commercialization. Specfically, we investigated how COVID-19 has impacted the RM industry by elaborating on the disruptions it caused but also the new opportunities it brought. The ultimate goal of this work is to make strategic recommendations for manufacturers and decisions-makers on effective strategies to address the above obstacles and facilitate patient access to promising regenerative medicines. FEATURES Regenerative medicine (RM) is an emerging interdisciplinary field aiming to replace or regenerate human cells, tissues, or organs in order to restore normal function. RM holds the promise of revolutionizing treatment in the 21st century. RMs bring new hope for some previously untreatable diseases, as well as holding promise for the treatment of common chronic diseases. Rapid advancements in biotechnology and improved understanding of disease pathophysiology have attracted tremendous interests in the development of RMs. Discusses the high cost of RMs which may challenge the sustainability of healthcare insurers (public and private). |
| biomarin hemophilia gene therapy: Fusion Protein Technologies for Biopharmaceuticals Stefan R. Schmidt, 2013-01-28 The state of the art in biopharmaceutical FUSION PROTEIN DESIGN Fusion proteins belong to the most lucrative biotech drugs—with Enbrel® being one of the best-selling biologics worldwide. Enbrel® represents a milestone of modern therapies just as Humulin®, the first therapeutic recombinant protein for human use, approved by the FDA in 1982 and Orthoclone® the first monoclonal antibody reaching the market in 1986. These first generation molecules were soon followed by a plethora of recombinant copies of natural human proteins, and in 1998, the first de novo designed fusion protein was launched. Fusion Protein Technologies for Biopharmaceuticals examines the state of the art in developing fusion proteins for biopharmaceuticals, shedding light on the immense potential inherent in fusion protein design and functionality. A wide pantheon of international scientists and researchers deliver a comprehensive and complete overview of therapeutic fusion proteins, combining the success stories of marketed drugs with the dynamic preclinical and clinical research into novel drugs designed for as yet unmet medical needs. The book covers the major types of fusion proteins—receptor-traps, immunotoxins, Fc-fusions and peptibodies—while also detailing the approaches for developing, delivering, and improving the stability of fusion proteins. The main body of the book contains three large sections that address issues key to this specialty: strategies for extending the plasma half life, the design of toxic proteins, and utilizing fusion proteins for ultra specific targeting. The book concludes with novel concepts in this field, including examples of highly relevant multifunctional antibodies. Detailing the innovative science, commercial realities, and brilliant potential of fusion protein therapeutics, Fusion Protein Technologies for Biopharmaceuticals is a must for pharmaceutical scientists, biochemists, medicinal chemists, molecular biologists, pharmacologists, and genetic engineers interested in determining the shape of innovation in the world of biopharmaceuticals. |
| biomarin hemophilia gene therapy: Orphan Drugs and Rare Diseases David Pryde, Michael J Palmer, 2014-07-30 Orphan drugs are designated drug substances that are intended to treat rare or ‘orphan’ diseases. More than 7000 rare diseases are known that collectively affect some 6-7% of the developed world’s population; however, individually, any single, rare disease may only affect a handful of people making them commercially unattractive for the biopharmaceutical industry to target. Ground breaking legislation, starting with the Orphan Drug Act that was passed in the US in 1983 to provide financial incentives for companies to develop orphan drugs, has sparked ever increasing interest from biopharmaceutical companies to tackle rare diseases. These developments have made rare diseases, and the orphan drugs that treat them, sufficiently attractive to pharmaceutical development and many pharmaceutical companies now have research units dedicated to this area of research. It is therefore timely to review the area of orphan drugs and some of the basic science, drug discovery and regulatory factors that underpin this important, and growing, area of biomedical research. Written by a combination of academic and industry experts working in the field, this text brings together expert authors in the regulatory, drug development, genetics, biochemistry, patient advocacy group, medicinal chemistry and commercial domains to create a unique and timely reference for all biomedical researchers interested in finding out more about orphan drugs and the rare diseases they treat. Providing an up-to-date monograph, this book covers the basic science, drug discovery and regulatory elements behind orphan drugs and will appeal to medicinal and pharmaceutical chemists, biochemists and anyone working within the fields of rare disease research and drug development or pharmaceuticals in industry or academia. |
| biomarin hemophilia gene therapy: MoneyBall Medicine Harry Glorikian, Malorye Allison Branca, 2017-11-20 How can a smartwatch help patients with diabetes manage their disease? Why can’t patients find out prices for surgeries and other procedures before they happen? How can researchers speed up the decade-long process of drug development? How will Precision Medicine impact patient care outside of cancer? What can doctors, hospitals, and health systems do to ensure they are maximizing high-value care? How can healthcare entrepreneurs find success in this data-driven market? A revolution is transforming the $10 trillion healthcare landscape, promising greater transparency, improved efficiency, and new ways of delivering care. This new landscape presents tremendous opportunity for those who are ready to embrace the data-driven reality. Having the right data and knowing how to use it will be the key to success in the healthcare market in the future. We are already starting to see the impacts in drug development, precision medicine, and how patients with rare diseases are diagnosed and treated. Startups are launched every week to fill an unmet need and address the current problems in the healthcare system. Digital devices and artificial intelligence are helping doctors do their jobs faster and with more accuracy. MoneyBall Medicine: Thriving in the New Data-Driven Healthcare Market, which includes interviews with dozens of healthcare leaders, describes the business challenges and opportunities arising for those working in one of the most vibrant sectors of the world’s economy. Doctors, hospital administrators, health information technology directors, and entrepreneurs need to adapt to the changes effecting healthcare today in order to succeed in the new, cost-conscious and value-based environment of the future. The authors map out many of the changes taking place, describe how they are impacting everyone from patients to researchers to insurers, and outline some predictions for the healthcare industry in the years to come. |
| biomarin hemophilia gene therapy: Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing Ganapathy Subramanian, 2021-12-15 Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing Explore new trends in continuous biomanufacturing with contributions from leading practitioners in the field With the increasingly widespread acceptance and investment in the ??technology, the last decade has demonstrated the utility of continuous ??processing in the pharmaceutical industry. In Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing, distinguished biotechnologist Dr. Ganapathy Subramanian delivers a comprehensive exploration of the potential of the continuous processing of biological products and discussions of future directions in advancing continuous processing to meet new challenges and demands in the manufacture of therapeutic products. A stand-alone follow-up to the editor’s Continuous Biomanufacturing: Innovative Technologies and Methods published in 2017, this new edited volume focuses on critical aspects of process intensification, process control, and the digital transformation of biopharmaceutical processes. In addition to topics like the use of multivariant data analysis, regulatory concerns, and automation processes, the book also includes: Thorough introductions to capacitance sensors to control feeding strategies and the continuous production of viral vaccines Comprehensive explorations of strategies for the continuous upstream processing of induced microbial systems Practical discussions of preparative hydrophobic interaction chromatography and the design of modern protein-A-resins for continuous biomanufacturing In-depth examinations of bioprocess intensification approaches and the benefits of single use for process intensification Perfect for biotechnologists, bioengineers, pharmaceutical engineers, and process engineers, Process Control, Intensification, and Digitalisation in Continuous Biomanufacturing is also an indispensable resource for chemical engineers seeking a one-stop reference on continuous biomanufacturing. |
| biomarin hemophilia gene therapy: Insights in Regulatory Science: 2021 Bruno Sepodes, Peter G. M. Mol, 2022-11-04 |
| biomarin hemophilia gene therapy: Essentials of Translational Pediatric Drug Development Elke Gasthuys, Karel Allegaert, Lien Dossche, Mark Turner, 2024-07-23 Essentials of Translational Pediatric Drug Development: From Past Needs to Future Opportunities provides integrated and up-to-date insights relevant for both translational researchers and clinicians active in the field of pediatric drug development. The book covers all key aspects from different stakeholder perspectives, providing a literature overview and careful reflection on state-of-the-art approaches. It will be an ideal guide for researchers in the field who are designing and performing high quality, innovative pediatric-adapted drug development by helping them define needs/challenges and possible solutions that advance and harmonize pediatric drug development.Despite the broad consensus that children merit the same quality of drug treatment as any other age group, children remain frequently neglected during drug research and development. Even with the adoption of multiple legislations addressing this problem, the lack of efficacy and safety data of marketed as well as newly developed drugs still remain in the pediatric population. - Covers both theoretical and practical aspects of translational pediatric drug development - Approaches the topic from different stakeholder perspectives (academics, industry, regulators, clinicians and patient/parent advocacy groups) - Offers best practices and future perspectives for the improvement of translational pediatric drug development |
| biomarin hemophilia gene therapy: The Changing Economics of Medical Technology Institute of Medicine, Committee on Technological Innovation in Medicine, 1991-02-01 Americans praise medical technology for saving lives and improving health. Yet, new technology is often cited as a key factor in skyrocketing medical costs. This volume, second in the Medical Innovation at the Crossroads series, examines how economic incentives for innovation are changing and what that means for the future of health care. Up-to-date with a wide variety of examples and case studies, this book explores how payment, patent, and regulatory policiesâ€as well as the involvement of numerous government agenciesâ€affect the introduction and use of new pharmaceuticals, medical devices, and surgical procedures. The volume also includes detailed comparisons of policies and patterns of technological innovation in Western Europe and Japan. This fact-filled and practical book will be of interest to economists, policymakers, health administrators, health care practitioners, and the concerned public. |
| biomarin hemophilia gene therapy: Pediatric Bleeding Disorders Amy L. Dunn, Bryce A. Kerlin, Sarah H. O'Brien, Melissa J. Rose, Riten Kumar, 2020-03-28 This clinical casebook provides a concise, state-of-the-art review of pediatric bleeding disorders. Presented in a case-based format, each case presents a different variant of bleeding disorder, illustrates the etiology, pathology, genetics, diagnosis, and management of the disorder, and offers clinical pearls. Disorders covered include hemophilia A and B, rare factor deficiencies, von Willebrand disease, immune thrombocytopenia purpura, and platelet dysfunctions. Written by experts in the field, Pediatric Bleeding Disorders: A Clinical Casebook is a valuable resource for clinicians and practitioners treating patients with challenging coagulation issues. |
| biomarin hemophilia gene therapy: Translational Surgery Adam E.M. Eltorai, 2023-03-22 Translational Surgery covers the principles of evidence-based medicine and applies these principles to the design of translational investigations. The reader will come to fully understand important concepts including case-control studies, prospective cohort studies, randomized trials, and reliability studies. Investigators will benefit from greater confidence in their ability to initiate and execute their own investigations, avoid common pitfalls in surgical research, and know what is needed for collaboration. Further, this title is an indispensable tool in grant writing and funding efforts. The practical, straightforward approach helps the translational research navigate challenging considerations in study design and implementation. The book provides valuable discussions of the critical appraisal of published studies in surgery, allowing the reader to learn how to evaluate the quality of such studies. Thus, they will improve at measuring outcomes; making effective use of all types of evidence in patient care. In short, this practical guidebook will be of interest to every surgeon or surgical researcher who has ever had a good clinical idea, but not the knowledge of how to test it. - Focuses on translational research in Surgery, covering the principles of evidence-based medicine and applying those principles to the design of translational investigations - Provides a practical, straightforward approach to help surgeons and researchers navigate challenging aspects of study design and implementation - Details valuable discussions on the critical appraisal of published studies in Surgery, allowing the reader to effectively use all types of evidence for patient care |
| biomarin hemophilia gene therapy: AAV Gene Therapy: Immunology and Immunotherapeutics Jose Martinez-Navio, Nicole K. Paulk, Guangping Gao, 2022-02-09 Dr. Gao is the co-founder of Voyager Therapeutics, Adrenas Therapeutics and Aspa Therapeutics. His research laboratory receives financial support from sponsored research agreements with various companies including Merck and LuYe Pharma. The other Topic Editors declare no conflict of interest with regards to the Research Topic theme |
| biomarin hemophilia gene therapy: Gene-Based Therapies for Pediatric Blood Diseases, An Issue of Hematology/Oncology Clinics of North America, E-Book Nirali N. Shah, Sung-Yun Pai, 2022-07-19 In this issue of Hematology/Oncology Clinics, guest editors Drs. Sung-Yun Pai and Nirali N. Shah bring their considerable expertise to the topic of Gene-Based Therapies for Pediatric Blood Diseases. Top experts in the field cover key topics such as CAR T-cell therapy: current status; engineered T cells; NK-cell therapy; hemoglobinopathies: beta-thalassemia, sickle cell disease; hemophilia A/B; primary immunodeficiencies; and more. - Contains 14 relevant, practice-oriented topics including the evolution of gene therapy; viral vectors in hematopoietic stem cell gene therapy; gene editing in hematopoietic stem cells; nonintegrating vectors and engineered capsids; regulatory aspects of gene therapy; and more. - Provides in-depth clinical reviews on gene-based therapies for pediatric blood diseases, offering actionable insights for clinical practice. - Presents the latest information on this timely, focused topic under the leadership of experienced editors in the field. Authors synthesize and distill the latest research and practice guidelines to create clinically significant, topic-based reviews. |
| biomarin hemophilia gene therapy: The Forever Fix Ricki Lewis, 2012-03-13 Lewis pens the first book to tell the fascinating story of gene therapy: how it works, the science behind it, how patients (mostly children) have been helped and harmed, and how scientists learned from each trial to get one step closer to the promise of a cure. |
| biomarin hemophilia gene therapy: Textbook of Hemophilia Christine A. Lee, Erik E. Berntorp, W. Keith Hoots, 2008-04-15 The only up-to-date definitive reference source onhemophilia This book is an invaluable resource that provides an overview ofall aspects of the care of patients with haemophilia. Covering how to assess both bleeding children and adults,Haemophilia A and B, molecular basis of the disease, the role offactors in coagulation, epidemiology, pharmacokinetics, andtreatment of inhibitors. There will also be a section onmusculoskeletal aspects of haemophilia as well as newerdevelopments such as gene therapy and rare bleedingdisorders. Textbook of Hemophilia is ideal for: Trainees and residents in hematology Hematologists in practice Specialists working in thrombosis and hemostasis as well astransfusion medicine Why Buy This Book? The only up-to-date definitive reference source onhemophilia Essential for all those managing hemophilia patients Detailed guidance on assessment, diagnosis, management andtreatment Advice for everyday clinical questions Edited by three of the world’s leading experts onhemophilia |
| biomarin hemophilia gene therapy: Curing Genetic Diseases through Genome Reprogramming , 2021-06-24 Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy—the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome. - Provides a comprehensive view of gene therapy and genome editing technologies, including epigenetic editing - Describes the state-of-the-art and future directions for the treatment of genetic diseases, also considering economical aspects - Presents chapters that each give a thorough review of a specific disease, target organ or visionary approach, including ethical considerations |
| biomarin hemophilia gene therapy: HIV and the Blood Supply Institute of Medicine, Committee to Study HIV Transmission Through Blood and Blood Products, 1995-10-05 During the early years of the AIDS epidemic, thousands of Americans became infected with HIV through the nation's blood supply. Because little reliable information existed at the time AIDS first began showing up in hemophiliacs and in others who had received transfusions, experts disagreed about whether blood and blood products could transmit the disease. During this period of great uncertainty, decision-making regarding the blood supply became increasingly difficult and fraught with risk. This volume provides a balanced inquiry into the blood safety controversy, which involves private sexual practices, personal tragedy for the victims of HIV/AIDS, and public confidence in America's blood services system. The book focuses on critical decisions as information about the danger to the blood supply emerged. The committee draws conclusions about what was doneâ€and recommends what should be done to produce better outcomes in the face of future threats to blood safety. The committee frames its analysis around four critical area: Product treatmentâ€Could effective methods for inactivating HIV in blood have been introduced sooner? Donor screening and referralâ€including a review of screening to exlude high-risk individuals. Regulations and recall of contaminated bloodâ€analyzing decisions by federal agencies and the private sector. Risk communicationâ€examining whether infections could have been averted by better communication of the risks. |
| biomarin hemophilia gene therapy: Emerging Medical Technologies Gennady Ermak, 2015-08-27 The book provides an overview of new technologies in medicine. It defines each technology and gives an account of its history, and the principles, technical problems, and applications as well as controversies involved. The goal of the book is to provide a comprehensive overview of the emerging medical technologies in a simple way that is understandable to non-specialists. Biomedical science and medical technologies are developing at such a fast pace that it is hard to follow even for specialists. In addition, they have become more and more complex. This book provides readers with a basic understanding of these technologies and helps them to keep up with new developments. The value of the book is that it is comprehensive and yet concise, which makes the learning process fast and easy. |
| biomarin hemophilia gene therapy: I-Bytes Healthcare Industry V-Gupta, 2019-12-18 This document brings together a set of latest data points and publicly available information relevant for Healthcare. We are very excited to share this content and believe that readers will benefit immensely from this periodic publication immensely. |
| biomarin hemophilia gene therapy: Pediatric Liver Transplantation Nedim Hadzic, Ulrich Baumann, Valérie MCLIN, 2020-11-13 Written and edited by global leaders in the field, Pediatric Liver Transplantation: A Clinical Guide covers all aspects of treatment and management regarding this multifaceted procedure and unique patient population. This practical reference offers detailed, focused guidance in a highly templated, easy-to-consult format, covering everything from pre-transplantation preparation to surgical techniques to post-operative complications. - Provides an in-depth understanding of all aspects of pediatric liver transplantation, ideal for pediatric hepatologists, pediatric transplant surgeons, and others on the pediatric transplant team. - Covers all surgical techniques in detail, including split graft, living related, auxiliary, and domino. - Discusses pediatric liver transplantation consideration for an increasing number of additional metabolic, hematologic and renal conditions; breakthroughs in grafting and stem cell therapy; and techniques and present role of hepatocyte transplantation. - Uses a quick-reference templated format; each chapter includes an overview, pathophysiology, conventional management, controversies, and bulleted summary of key take-aways. - Includes state-of-the-art mini-reviews based on updated references and author experience throughout the text. - Features a full-color design with numerous algorithms, figures, and radiological and histopathological photos. |
| biomarin hemophilia gene therapy: Decision Modelling for Health Economic Evaluation Andrew Briggs, Mark Sculpher, Karl Claxton, 2006-08-17 In financially constrained health systems across the world, increasing emphasis is being placed on the ability to demonstrate that health care interventions are not only effective, but also cost-effective. This book deals with decision modelling techniques that can be used to estimate the value for money of various interventions including medical devices, surgical procedures, diagnostic technologies, and pharmaceuticals. Particular emphasis is placed on the importance of the appropriate representation of uncertainty in the evaluative process and the implication this uncertainty has for decision making and the need for future research. This highly practical guide takes the reader through the key principles and approaches of modelling techniques. It begins with the basics of constructing different forms of the model, the population of the model with input parameter estimates, analysis of the results, and progression to the holistic view of models as a valuable tool for informing future research exercises. Case studies and exercises are supported with online templates and solutions. This book will help analysts understand the contribution of decision-analytic modelling to the evaluation of health care programmes. ABOUT THE SERIES: Economic evaluation of health interventions is a growing specialist field, and this series of practical handbooks will tackle, in-depth, topics superficially addressed in more general health economics books. Each volume will include illustrative material, case histories and worked examples to encourage the reader to apply the methods discussed, with supporting material provided online. This series is aimed at health economists in academia, the pharmaceutical industry and the health sector, those on advanced health economics courses, and health researchers in associated fields. |
| biomarin hemophilia gene therapy: Insanely Simple Ken Segall, 2012-04-26 'Simple can be harder than complex. You have to work hard to get your thinking clean to make it simple. But it's worth it in the end, because once you get there, you can move mountains' Steve Jobs, BusinessWeek, May 25, 1998 To Steve Jobs, Simplicity wasn't just a design principle. It was a religion and a weapon. The obsession with Simplicity is what separates Apple from other technology companies. It's what helped Apple recover from near death in 1997 to become the most valuable company on Earth in 2011, and guides the way Apple is organized, how it designs products, and how it connects with customers. It's by crushing the forces of Complexity that the company remains on its stellar trajectory. As creative director, Ken Segall played a key role in Apple's resurrection, helping to create such critical campaigns as 'Think Different' and naming the iMac. Insanely Simple is his insider's view of Jobs' world. It reveals the ten elements of Simplicity that have driven Apple's success - which you can use to propel your own organisation. Reading Insanely Simple, you'll be a fly on the wall inside a conference room with Steve Jobs, and on the receiving end of his midnight phone calls. You'll understand how his obsession with Simplicity helped Apple perform better and faster. |
Gene therapy in hemophilia A: The impact of valoctocogene …
Jun 25, 2023 · •Understand application of a novel, hemophilia-specific, patient-reported outcome measure in a clinical trial involving a phase 3 gene therapy •Understand the effect of a one …
ROCTAVIAN - biomarin-rareconnections.com
ROCTAVIAN is a one-time gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to the virus, AAV5 which is determined by a blood test. …
GENE THERAPY FOR HEMOPHILIA
Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including the various approaches of gene therapy. Discuss the pros and cons of gene therapy …
BioMarin RareConnections Brochure - roctavian.com
ROCTAVIANTM is the only FDA approved gene therapy for eligible adults with severe hemophilia A. This one-time infusion is matched by one-to-one, personalized product and education …
Gene Therapy for Hemophilia B and An Update on Gene …
Oct 3, 2022 · from Bayer, BioMarin, CSL-Behring, Freeline Therapeutics, Novo Nordisk, F. HoffmanLa Roche,- Sanofi, Sobi, Takeda, uniQure. The PROBE study is an independent, …
NBDF 2024 GENEr8-1 4-year clinical PLS FINAL
Valoctocogene roxaparvovec is the first approved gene therapy for hemophilia A. Gene therapy is meant to address the lack of genetic instructions for making FVIII
PRODUCT PIPELINE GENE THERAPY (March 8, 2023)
Canada by Biomarin is expected in late 2024 or 2025. BMN 270 uses adeno-associated viruses (AAV5) as the delivery vehicle, or vector, to carry the genetic codes that prompt the production …
Gene Therapy for Hemophilia: Are Expectations Matching …
BioMarin Pharmaceuticals’ ongoing phase 3 trial (ClinicalTrials.-gov: NCT03370913) is designed to test a single dose (6E13 vg/kg) of valoctocogene roxaparvovec (an adeno-associated viral …
Perceptions, needs and expectations of patients with …
Q18b. Would you be willing to use gene therapy for the treatment of your hemophilia? Q25. Potential gene therapy might require frequent visits to the hemophilia,
Update for the Hemophilia Community
BioMarin is providing an update to the community regarding their ongoing gene therapy clinical trial program in hemophilia A. The safety of patients and study participants is BioMarin’s top …
BioMarin Pharmaceutical Inc. Response to 18-month Follow …
Dec 22, 2022 · While current liver-based AAV -based gene therapy depends on healthy hepatocyte function, those with hemophilia may have past or current hepatitis C virus and/or …
BioMarin and hemophilia timeline - BioMarin Hemophilia
Researchers report first successful gene therapy for hemophilia A. American Society of Hematology website. https://www.ashclinicalnews.org/on- location/researchers-report-first …
GENE THERAPY FOR HEMOPHILIA
Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including the various approaches of gene therapy. Discuss the pros and cons of gene therapy …
Determining eligibility for ROCTAVIAN - BioMarin
ROCTAVIAN is indicated for the treatment of adults with severe hemophilia A (congenital Factor VIII deficiency with Factor VIII activity <1 IU/dL) without antibodies to adeno-associated virus …
Results from the phase 3 GENEr8-1 trial - BioMarin Medical …
Valoctocogene roxaparvovec gene therapy for severe hemophilia A 3 • Hemophilia A is an X -linked bleeding disorder caused by deficiency in coagulation FVIII • Valoctocogene …
An update for the hemophilia community from BioMarin, …
investigational gene therapy. The first trial of valoctocogene roxaparvovec, a Phase 1/2 study with 15 participants, was initiated in 2015 and completed enrollment in 2017. These participants are …
Safety and efficacy of valoctocogene roxaparvovec …
(AAV5-hFVIII-SQ) is a liver-directed gene therapy that transfers a FVIII coding sequence to enable FVIII production in people with severe hemophilia A (FVIII ≤1 IU/dL)1,2 FVIII protein …
PRODUCT PIPELINE GENE THERAPY (November 2, 2023)
Gene therapy for hemophilia B Pfizer initiated the BENEGENE-2 Phase 3, open-label, single-arm study to evaluate efficacy and safety of factor IX (FIX) gene transfer with PF-06838435 (Raav …
Gene Therapy in Hemophilia A: Redefining the Treatment …
clinical impact of gene therapy for hemophilia A. ... This activity is supported by an educational grant from BioMarin Pharmaceutical Inc. Gene Therapy in Hemophilia A: Redefining the …
Impact of Gene Therapy on the Economic Burden of Adults …
Gene therapy has the potential to replace the high annual cost of prophylaxis with a single upfront treatment cost and may also affect non-medical costs in multiple years, significantly altering the …
Gene therapy in hemophilia A: The impact of valoctocogene …
Jun 25, 2023 · •Understand application of a novel, hemophilia-specific, patient-reported outcome measure in a clinical trial involving a phase 3 gene therapy •Understand the effect of a one …
ROCTAVIAN - biomarin-rareconnections.com
ROCTAVIAN is a one-time gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to the virus, AAV5 which is determined by a blood test. …
GENE THERAPY FOR HEMOPHILIA
Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including the various approaches of gene therapy. Discuss the pros and cons of gene therapy …
BioMarin RareConnections Brochure - roctavian.com
ROCTAVIANTM is the only FDA approved gene therapy for eligible adults with severe hemophilia A. This one-time infusion is matched by one-to-one, personalized product and education …
Gene Therapy for Hemophilia B and An Update on Gene …
Oct 3, 2022 · from Bayer, BioMarin, CSL-Behring, Freeline Therapeutics, Novo Nordisk, F. HoffmanLa Roche,- Sanofi, Sobi, Takeda, uniQure. The PROBE study is an independent, …
NBDF 2024 GENEr8-1 4-year clinical PLS FINAL
Valoctocogene roxaparvovec is the first approved gene therapy for hemophilia A. Gene therapy is meant to address the lack of genetic instructions for making FVIII
PRODUCT PIPELINE GENE THERAPY (March 8, 2023)
Canada by Biomarin is expected in late 2024 or 2025. BMN 270 uses adeno-associated viruses (AAV5) as the delivery vehicle, or vector, to carry the genetic codes that prompt the production …
Gene Therapy for Hemophilia: Are Expectations Matching …
BioMarin Pharmaceuticals’ ongoing phase 3 trial (ClinicalTrials.-gov: NCT03370913) is designed to test a single dose (6E13 vg/kg) of valoctocogene roxaparvovec (an adeno-associated viral …
Perceptions, needs and expectations of patients with …
Q18b. Would you be willing to use gene therapy for the treatment of your hemophilia? Q25. Potential gene therapy might require frequent visits to the hemophilia,
Update for the Hemophilia Community
BioMarin is providing an update to the community regarding their ongoing gene therapy clinical trial program in hemophilia A. The safety of patients and study participants is BioMarin’s top …
BioMarin Pharmaceutical Inc. Response to 18-month Follow …
Dec 22, 2022 · While current liver-based AAV -based gene therapy depends on healthy hepatocyte function, those with hemophilia may have past or current hepatitis C virus and/or …
BioMarin and hemophilia timeline - BioMarin Hemophilia
Researchers report first successful gene therapy for hemophilia A. American Society of Hematology website. https://www.ashclinicalnews.org/on- location/researchers-report-first …
GENE THERAPY FOR HEMOPHILIA
Discuss up-to-date information on the current and emerging approaches for treating hemophilia, including the various approaches of gene therapy. Discuss the pros and cons of gene therapy …
Determining eligibility for ROCTAVIAN - BioMarin
ROCTAVIAN is indicated for the treatment of adults with severe hemophilia A (congenital Factor VIII deficiency with Factor VIII activity <1 IU/dL) without antibodies to adeno-associated virus …
Results from the phase 3 GENEr8-1 trial - BioMarin Medical …
Valoctocogene roxaparvovec gene therapy for severe hemophilia A 3 • Hemophilia A is an X -linked bleeding disorder caused by deficiency in coagulation FVIII • Valoctocogene …
An update for the hemophilia community from BioMarin, …
investigational gene therapy. The first trial of valoctocogene roxaparvovec, a Phase 1/2 study with 15 participants, was initiated in 2015 and completed enrollment in 2017. These participants …
Safety and efficacy of valoctocogene roxaparvovec …
(AAV5-hFVIII-SQ) is a liver-directed gene therapy that transfers a FVIII coding sequence to enable FVIII production in people with severe hemophilia A (FVIII ≤1 IU/dL)1,2 FVIII protein …
PRODUCT PIPELINE GENE THERAPY (November 2, 2023)
Gene therapy for hemophilia B Pfizer initiated the BENEGENE-2 Phase 3, open-label, single-arm study to evaluate efficacy and safety of factor IX (FIX) gene transfer with PF-06838435 (Raav …
Gene Therapy in Hemophilia A: Redefining the Treatment …
clinical impact of gene therapy for hemophilia A. ... This activity is supported by an educational grant from BioMarin Pharmaceutical Inc. Gene Therapy in Hemophilia A: Redefining the …
Impact of Gene Therapy on the Economic Burden of Adults …
Gene therapy has the potential to replace the high annual cost of prophylaxis with a single upfront treatment cost and may also affect non-medical costs in multiple years, significantly altering …
