Csl Behring Gene Therapy

  csl behring gene therapy: Sickle Cell Disease Mark T. Gladwin, Gregory Kato, Enrico M. Novelli, 2021-01-05 The most comprehensive, current sickle cell disease resource—for both clinicians and researchers A Doody's Core Title for 2023! The first and only resource of its kind, Sickle Cell Disease examines this blood disorder through both clinical and research lenses. More than 80 dedicated experts in the field present their combined clinical knowledge of basic mechanisms, screening, diagnosis, management, and treatment of myriad complex complications of a single base point mutation in the human genome. Case studies with “How I Treat” authoritative insights provide overviews of common and rare complications, and Key Facts offer at-a-glance high-yield information. Filled with clinical photos, illustrations, numerous original diagrams, and with free updates available online, this unmatched resource covers: Mechanisms of sickle cell disease Historic and current research approaches The latest work in gene therapy and editing Guidelines for patient care, diagnosis, unique cases, and therapies Rare and common complications, including domestic and internationally relevant topics Psychosocial and supportive care The newest standards of therapy and future treatment options in children and adults Cardiopulmonary complications
  csl behring gene therapy: Drug Development for Gene Therapy Yanmei Lu, Boris Gorovits, 2024-02-09 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.
  csl behring gene therapy: Raising a Rare Girl Heather Lanier, 2021-07-06 “A remarkable book . . . I found myself thinking that all expectant and new parents should read it.” —Michelle Slater A New York Times Book Review Editors' Choice In Raising a Rare Girl, Lanier explores how to defy the tyranny of normal and embrace parenthood as a spiritual practice that breaks us open in the best of ways. Like many women of her generation, when Heather Lanier was expecting her first child she did everything by the book in the hope that she could create a SuperBaby, a supremely healthy human destined for a high-achieving future. But her daughter Fiona challenged all of Lanier’s preconceptions. Born with an ultra-rare syndrome known as Wolf-Hirschhorn, Fiona received a daunting prognosis: she would experience significant developmental delays and might not reach her second birthday. The diagnosis obliterated Lanier’s perfectionist tendencies, along with her most closely held beliefs about certainty, vulnerability, God, and love. With tiny bits of mozzarella cheese, a walker rolled to library story time, a talking iPad app, and a whole lot of pop and reggae, mother and daughter spend their days doing whatever it takes to give Fiona nourishment, movement, and language. Loving Fiona opens Lanier up to new understandings of what it means to be human, what it takes to be a mother, and above all, the aching joy and wonder that come from embracing the unique life of her rare girl.
  csl behring gene therapy: Extraordinary! A Book for Children with Rare Diseases (Mandarin) Evren And Kara Ayik, 2022-03-03 是什么让罕见病儿童非同寻常？ 聆听罕见病儿童埃夫伦自我成长的心路历程，探索问题答案！ 本书由母子联手创作，基于罕见病患者面临的挑战与机遇，就身份、包容与自我认知展开适合孩子的讨论。书中的经验全部是小作者作为超罕见病患者一路长大的亲身经历，旨在为小读者理解个人身份和罕见病对个人身份的正面影响提供指南。家庭成员和护理人员也可以积极参与孩子的探索过程，根据孩子的成长需求制定读书计划。 本书插图由获奖童书插画家伊恩-戴尔绘制。《非同寻常的你！》旨在通过其由衷的文字和敏感又真实的插画鼓舞、激励所有患有罕见病的孩子活出最美好的一生。 What makes a child with a rare disease extraordinary? Explore the answer to this question while sharing a conversation with Evren about what he has learned while growing up with his own rare disease. Written collaboratively by mother and son, this book opens up a child-friendly discussion about identity, inclusion, and self-concept in light of the challenges and silver linings of living with a rare disease. The gentle lessons draw on the co-author's first-hand experience of growing up with an ultra-rare disease and offer young readers a framework for understanding personal identity and how their rare diseases can help shape it in positive ways. Family members and caregivers are invited to share in this conversation and to customize the reading according to each young reader's developmental needs. Accompanied by sensitive yet realistic illustrations created by award-winning artist and children's book illustrator Ian Dale, the heartfelt messages introduced in Extraordinary! are intended to uplift and encourage any children living with rare diseases to live their very best lives.
  csl behring 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
  csl behring 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
  csl behring 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).
  csl behring gene therapy: Rossi's Principles of Transfusion Medicine Toby L. Simon, Jeffrey McCullough, Edward L. Snyder, Bjarte G. Solheim, Ronald G. Strauss, 2016-05-23 Rossi's Principles of Transfusion Medicine is the most comprehensive and practical reference on transfusion science and medicine available Led by a world class Editor team, including two past-presidents of AABB, a past- President of the American Board of Pathology and members of the FDA Blood Products Advisory Committee , and international contributor team Comprehensive reference resource, considered the gold standard in transfusion Covers current hot topics such as donor care – including the frequency of donation and management of iron deficiency/status), patient blood management, hemovigilance, cstem cell therapies, and global aspects of the organization of transfusion and transplant services New material on molecular immunohematology Companion website includes figures, full text and references
  csl behring gene therapy: Life Sciences Industry Basanta Kumara Bahera, Ram Prasad, Shyambhavee Behera, 2021-08-24 Basic principles of applied life sciences such as recombinant DNA technology is used in most life sciences industries marketing bio-formulations for designing more effective protein-based drugs, such as erythropoietin and fast-acting insulin etc. In recent times genetically engineered host cells from mammal, animal and plants are also being used in life sciences industries to manufacture biologics. This book discusses the most basic as well advanced issues on biological products for successfully managing a life sciences industry. It elucidates the life cycle of biological molecules, right from the conceptual development of different types of biopolymers, and their subsequent transfer from the conical flasks in laboratory to life sciences industries for large scale production and marketing. It focuses on sustainable longevity in the life cycle of commercial biopolymers. Cumulative facts and figures in this volume would immensely help in inspiring life sciences industry promoters to monitor value chain transfer process of biologics for better profitability. Additionally, it would serve as a perusal document for the students and researchers interested in entrepreneurial ventures or having their own start-up projects for the commercialization of biologics.
  csl behring 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.
  csl behring gene therapy: Inflammatory immune disease: Molecular mechanisms, translational approaches and therapeutics volume II Tao Xu, Qingzhong Xiao, Jian Gao, Cheng Chen, 2023-03-17
  csl behring gene therapy: Blood Farm Cara McGoogan, 2023-10-10 “A stunning investigation.” ­­ —Publishers Weekly How a miracle treatment turned deadly and changed the course of the AIDS crisis. By the mid 1980s, AIDS hysteria was so rampant that a fearful and prejudiced public ignored stories of gay men falling ill with lesions and mouth ulcers. President Reagan avoided mentioning the disease entirely. Then, as chronicled in Blood Farm, a new HIV-positive population emerged, one that included kids like Ken Dixon, Brad Cross, and Ryan White who had been infected as young as ten years old. But how? Unbeknownst to doctors and patients, pharmaceutical companies like Bayer, Baxter, and Armour collected plasma on skid row, in night clubs, and in some of America’s most notorious prisons to make Factor VIII, a new miracle treatment for hemophilia. Companies knew these practices put patients at high risk of HIV, but miracles are a lucrative business, so they knowingly sold an infected product and effectively played Russian Roulette with hemophiliacs’ lives. The results were catastrophic. In America, some 8,000 people with hemophilia contracted HIV; only 700 are alive today. Award-winning journalist Cara McGoogan daringly exposes an expansive map of corporate greed and negligence that led to one of the biggest overlooked medical scandals in history. Alongside her we meet survivors turned activists, determined small town lawyers, and fearless reporters desperate for justice. Their fight for retribution created a critical inflection point in the AIDS crisis: stigmas shifted, settlements were awarded, and, later, President George H.W. Bush signed into law the largest federal program on HIV. In shocking, riveting detail, Blood Farm uncovers how a miracle treatment became a deadly poison and forever changed our understanding of AIDS.
  csl behring gene therapy: Digital Health Dipu Patel, 2024-10-21 Digital Health: Telemedicine and Beyond describes practical ways to use digital health tools in clinical practice. With a strong focus on case studies and patient outcomes, this title provides an overview of digital medicine, terms, concepts, and applications for the multidisciplinary clinical practitioner. Chapters provide a concise, yet comprehensive understanding of digital health, including telemedicine, mHealth, EHRs, and the benefits and challenges of each. The book gives insights on risks and benefits associated with storing and transmitting patient information via digital tools and educates clinicians in the correct questions to ask for advocacy regarding state laws, scope of practice, and medicolegal implications. It also addresses the ethical and social challenges that digital health raises, how to engage patients to improve shared decision-making models and how digital health tools can be integrated into clinical practice. This book is a valuable resource for clinicians and medical educators of all health professions, including physicians, physician associates, nurses, pharmacists, physical therapists, occupational therapists, speech therapists, students, and all those who wish to broaden their knowledge in the allied field. - Provides a clinical perspective on digital health - Written by clinicians for clinicians with the patient in mind - Describes practical ways to use digital health tools in clinical practice - Includes case studies to incorporate workflows into practice to improve patient outcomes
  csl behring gene therapy: Physiology Or Medicine, 1901-1921 , 1999
  csl behring gene therapy: A Glimpse at Medicine in the Future Mandana Hasanzad,
  csl behring gene therapy: Congenital Bleeding Disorders Akbar Dorgalaleh, 2024-01-28 This significantly updated new edition describes in detail the clinical presentations, 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.
  csl behring gene therapy: Biopharmaceutical Manufacturing Ralf Pörtner, 2024-02-11 This volume “Cell Engineerring 11 - Biopharmaceutical Manufacturing: Progress, Trends and Challenges” is a source of the latest innovative research and technical development in biomanufacturing systems. It is organised into 2 parts: 1) Manufacturing of recombinant therapeutic proteins (e.g. therapeutic antibodies, biosimilars/biogenerics) and 2) Manufacturing aspects of cell and gene therapy. Each with selected chapters on the following topics for both up- and downstream, such as: Advanced process strategies, especially continuous manufacturing, Advanced culture techniques, especially single-use systems, Process transfer, scale-up/scale-down models, Processing advances/Manufacturing productivity/efficiency, Model-assisted process understanding and development/Digital Twins, Process controls and analytics, Quality control, Quality by design, Facility design and full-scale commercial systems, manufacturing technology innovation. The book comprises contributions of experts from academia and industry active in the field of cell culture development for the production of recombinant proteins, cell therapy and gene therapy, with consideration of Digital Twin ́s and facility design. The knowledge and expertise of the authors cover disciplines like cell biology, engineering, biotechnology and biomedical sciences. Inevitably, some omissions will occur in the test, but the authors have sought to avoid duplications by extensive cross-referencing to chapters in other volumes of this series and elsewhere. We hope the volume provides a useful compendium of techniques for scientists in industrial and research laboratories active in this field.
  csl behring gene therapy: Production of Plasma Proteins for Therapeutic Use Joseph Bertolini, Neil Goss, John Curling, 2012-12-06 Sets forth the state of the science and technology in plasma protein production With contributions from an international team of eighty leading experts and pioneers in the field, Production of Plasma Proteins for Therapeutic Use presents a comprehensive overview of the current state of knowledge about the function, use, and production of blood plasma proteins. In addition to details of the operational requirements for the production of plasma derivatives, the book describes the biology, development, research, manufacture, and clinical indications of essentially all plasma proteins with established clinical use or therapeutic potential. Production of Plasma Proteins for Therapeutic Use covers the key aspects of the plasma fractionation industry in five sections: Section 1: Introduction to Plasma Fractionation initially describes the history of transfusion and then covers the emergence of plasma collection and fractionation from its earliest days to the present time, with the commercial and not-for-profit sectors developing into a multi-billion dollar industry. Section 2: Plasma Proteins for Therapeutic Use contains 24 chapters dedicated to specific plasma proteins, including coagulation factors, albumin, immunoglobulin, and a comprehensive range of other plasma-derived proteins with therapeutic indications. Each chapter discusses the physiology, biochemistry, mechanism of action, and manufacture of each plasma protein including viral safety issues and clinical uses. Section 3: Pathogen Safety of Plasma Products examines issues and procedures for enhancing viral safety and reducing the risk of transmissible spongiform encephalopathy transmission. Section 4: The Pharmaceutical Environment Applied to Plasma Fractionation details the requirements and activities associated with plasma collection, quality assurance, compliance with regulatory requirements, provision of medical affairs support, and the manufacture of plasma products. Section 5: The Market for Plasma Products and the Economics of Fractionation reviews the commercial environment and economics of the plasma fractionation industry including future trends, highlighting regions such as Asia, which have the potential to exert a major influence on the plasma fractionation industry in the twenty-first century.
  csl behring gene therapy: Biologics and Biosimilars Xiaodong Feng, Hong-Guang Xie, Ashim Malhotra, Catherine F. Yang, 2022-06-13 Biologics and Biosimilars: Drug Discovery and Clinical Applications is a systematic integration and evaluation of all aspects of biologics and biosimilars, encompassing research and development, clinical use, global regulation, and more. Biosimilars are biological therapeutic agents designed to imitate a reference biologic with high similarities in structure, efficacy, and safety, but also with potential clinical effective and cost-efficient options for the manufacturers, payers, clinicians, and patients. Most of the top-selling prescription drugs in the current market are biologics, which have revolutionized the treatment strategies and modalities for life-threatening and/or rare diseases. This book outlines the key processes and challenges in drug development, regulations, and clinical applications of biologics, biosimilars, and even interchangeable biosimilars. Global experts in the field discuss essential categories and prototype drugs of biologics and biosimilars in clinical practice such as allergenics, blood and blood components, cell treatment, gene therapy, recombinant therapeutic proteins or peptides, tissues, and vaccines. Additional features: Integrates the latest bench and bedside evidence of drug development and regulations of biologics and biosimilars Contains key study questions for each chapter to guide the readers, as well as drug charts for all therapeutic applications of biologics and biosimilars Presents detailed schematic illustrations to explain the drug development, clinical trials, regulations, and clinical applications of biologics and biosimilars This book is an invaluable tool for health care professional students, providers, and pharmaceutical and health care industries, as well as the public, providing readers with educational updates about the drug development and clinical affairs of biological medications and their similar drugs.
  csl behring gene therapy: Navigating Non-coding RNA Joanna Sztuba-Solinska, 2023-06-17 Navigating Non-coding RNA: From Biogenesis to Therapeutic Application provides a concise overview of the field of non-coding RNA (ncRNA). Chapters cover the history of discoveries that have occurred in the area of ncRNA, specific types of ncRNA, housekeeping ncRNAs such as ribosomal RNA, transfer RNA, small nuclear RNA and telomerase RNA, regulatory ncRNAs such as microRNA, small interfering RNA, long non-coding RNA and Y RNA. Biogenesis, structure, function, and regulation of each of these are also explored in addition to traditional and cutting-edge methods for the identification, functional characterization and structural characterization of ncRNA. The book also focuses on the different types of epitranscriptomic modifications and their involvement in regulating ncRNA structure, stability and intermolecular interactions in addition to the role of ncRNAs in a range of diseases and potential therapeutic applications. - Covers a wide range of non-coding RNAs, including ribosomal RNA, transfer RNA, telomerase RNA, microRNA, small interfering RNA and circular RNA - Features both traditional and novel methodologies for investigating ncRNA, from microarray and conventional chemical probing to CAGE-seq and computational methods - Includes chapters on ncRNAs in a range of diseases, including cancers, neurological disorders, cardiovascular conditions and infectious illnesses - Discusses novel therapeutic strategies for targeting ncRNAs, including CRISP/Ca9 applications and RNAi-based strategies - Explores the molecular mechanisms and intermolecular interactions of ncRNA
  csl behring gene therapy: Alpha-1-antitrypsin Deficiency Noor Kalsheker, Robert Andrew Stockley, 2017-06-06 Alpha-1-antitrypsin Deficiency: Biology, Diagnosis, Clinical Significance, and Emerging Therapies is the authoritative reference on AATD, providing standards for diagnosis, monitoring, treatment and appropriate avenues of research. The book covers the disease from basic biology and epidemiology, to clinical impact, and includes the understanding of the natural history of the disease and the significant advances that have been made in the last 20 years, including the three-dimensional structure of the molecule, its broad biological activity and improved therapeutic options, including replacement therapy and gene therapy. The editors have recruited international experts in the field to contribute evidence-based chapters and insights on future developments in the understanding of this disease. - Provides documentation of the variations in clinical presentation and pathology in a single reference - Presents new insights by pulling together the advances in the understanding of the structure and function of alpha1-antitrypsin deficiency with the genetic variants that cause the disease - Allows for easy reference for the diagnosis of AATD to lead to better therapeutics
  csl behring gene therapy: Raising a Child with Hemophilia Laureen Kelley, 2016-04-01 A parenting book covering medical, consumer, emotional and practical child-rearing aspects of raising a child with hemophilia.
  csl behring gene therapy: Nonmalignant Hematology Syed A. Abutalib, Jean M. Connors, Margaret V. Ragni, 2016-08-24 This book, in Q&A format, addresses a wide range of clinically relevant topics and issues in Nonmalignant Hematology, or “Benign Hematology,” with a view to offering a robust, engaging tool that will assist every hematologist and oncologist (pediatric and adult equally) in making decisions during day-to-day practice. The entire spectrum of the specialty is covered in more than 60 exceptional chapters written by acknowledged authorities in the field. The content is organized into well-designed broad sections on red cell disorders, platelet and coagulation disorders, coagulopathy in systemic diseases, thromboembolic disease and its management (including surgical), immune system and related disorders, hemostasis and thrombosis during pregnancy and in the newborn and elderly, and Transfusion Medicine. A key aspect of the book is the opportunity it affords expert physicians to express well-reasoned opinions regarding complex issues in Nonmalignant Hematology. Readers will find that it provides a practical and immediately applicable compendium of answers to often complex and vexing questions. It will appeal to residents, fellows, house officers and more experienced practitioners around the globe.
  csl behring gene therapy: Rossi's Principles of Transfusion Medicine Toby L. Simon, Eric A. Gehrie, Jeffrey McCullough, John D. Roback, Edward L. Snyder, 2022-08-15 ROSSI’S PRINCIPLES OF TRANSFUSION MEDICINE Transfusion Medicine impacts patients with hematologic, oncologic, and surgical conditions as well as all areas of critical care medicine and multiple areas of chronic care. This book aims to be the single best source for information related to any aspect or application of Transfusion Medicine. Contributors for the sixth edition have once again been drawn from various scientific, medical, and surgical disciplines. Thus, this book ranges from encouraging and managing donors, to collecting and preserving the blood, to matching it to the appropriate recipient, all the way to its clinical uses. It also extends these concepts to implantable tissue and regenerative medicine. Other sample topics covered within the work include: Contemporary issues in donation and transfusion: patient blood management, clinical and technical aspects of blood administration, and donor and patient Hemovigilance Blood components and derivatives: red blood cell metabolism, preservation and oxygen delivery, blood groups, and composition of plasma Apheresis, transplantation, and new therapies: hematopoietic growth factors, therapeutic phlebotomy and cellular apheresis, HLA antigens, alleles, and antibodies How Transfusion Medicine has been affected by the coronavirus pandemic, the role of pathogen reduction and other modern trends This book serves as a complete and comprehensive resource on Transfusion Medicine for clinicians who prescribe blood, students who expect to enter clinical practice, and for the scientists, physicians, nurses, technologists, and others who assure the quality and availability of blood services.
  csl behring gene therapy: The Catalyst: RNA and the Quest to Unlock Life's Deepest Secrets Thomas R. Cech, 2024-06-04 One of Literary Hub's Most Anticipated Books of 2024 Exploring the most transformative breakthroughs in biology since the discovery of the double helix, a Nobel Prize–winning scientist unveils the RNA age. For over half a century, DNA has dominated science and the popular imagination as the “secret of life.” But over the last several decades, a quiet revolution has taken place. In a series of breathtaking discoveries, the biochemist Thomas R. Cech and a diverse cast of brilliant scientists have revealed that RNA—long overlooked as the passive servant of DNA—sits at the center of biology’s greatest mysteries: How did life begin? What makes us human? Why do we get sick and grow old? In The Catalyst, Cech finally brings together years of research to demonstrate that RNA is the true key to understanding life on Earth, from its very origins to our future in the twenty-first century. A gripping journey of discovery, The Catalyst moves from the early experiments that first hinted at RNA’s spectacular powers, to Cech’s own paradigm-shifting finding that it can catalyze cellular reactions, to the cutting-edge biotechnologies poised to reshape our health. We learn how RNA—once thought merely to transmit DNA’s genetic instructions to the cell’s protein-making machinery—may have jump-started life itself, and how, at the same time, it can cut our individual lives short through viral diseases and cancer. We see how RNA is implicated in the aging process and explore the darker depths of the supposed fountain of youth, telomerase. And we catch a thrilling glimpse into how RNA-powered therapies—from CRISPR, the revolutionary tool that uses RNA to rewrite the code of life, to the groundbreaking mRNA vaccines that have saved millions during the pandemic, and more—may enable us to improve and even extend life beyond nature’s current limits. Written by one of our foremost scientists, The Catalyst is a must-read guide to the present and future of biology and medicine.
  csl behring 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
  csl behring 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.
  csl behring gene therapy: The Management of the Haemophilc Arthropathy Christian Carulli, 2017-06-16 Frontiers in Arthritis is an ebook series devoted to publishing the latest advances in arthritis medicine and research. Each volume brings forth contributions on topics relevant to the diagnosis, management and treatment of arthritis. The ebook series is essential reading for rheumatologists and orthopedic surgeons involved in clinical research and practice. This volume presents comprehensive information about the pathology, diagnosis and treatment of haemophilic arthropathy. Readers will find information about knee, hip, elbow, foot and ankle surgery in patients affected by haemophilia as well as special topics (microsurgery and postoperative rehabilitation and health risks). The broad range of information presented in this volume makes it the definitive handbook on arthritis in haemophiliac cases and the management of related complications.
  csl behring gene therapy: Hemostasis and Thrombosis Thomas G. DeLoughery, 2019-08-10 The latest edition of this practical handbook provides a concise yet comprehensive overview of common and rare problems with hemostasis and thrombosis. Through thoroughly updated and revised chapters, the text features practical information on diagnosing and managing troublesome hematologic conditions often found in clinical practice. The handbook also spotlights advances in the field since the publication of the last edition, including multiple novel coagulation factors for hemophilia, the increasing use of novel anticoagulants and their reversal agents, and the increasing use of genetics for diagnosis. Written by experts in the field, Hemostasis and Thrombosis, Fourth Edition is an invaluable resource for healthcare professionals who treat patients afflicted with these conditions.
  csl behring gene therapy: High Value Fermentation Products, Volume 1 Saurabh Saran, Vikash Babu, Asha Chaubey, 2019-04-09 Green technologies are no longer the “future” of science, but the present. With more and more mature industries, such as the process industries, making large strides seemingly every single day, and more consumers demanding products created from green technologies, it is essential for any business in any industry to be familiar with the latest processes and technologies. It is all part of a global effort to “go greener,” and this is nowhere more apparent than in fermentation technology. This book describes relevant aspects of industrial-scale fermentation, an expanding area of activity, which already generates commercial values of over one third of a trillion US dollars annually, and which will most likely radically change the way we produce chemicals in the long-term future. From biofuels and bulk amino acids to monoclonal antibodies and stem cells, they all rely on mass suspension cultivation of cells in stirred bioreactors, which is the most widely used and versatile way to produce. Today, a wide array of cells can be cultivated in this way, and for most of them genetic engineering tools are also available. Examples of products, operating procedures, engineering and design aspects, economic drivers and cost, and regulatory issues are addressed. In addition, there will be a discussion of how we got to where we are today, and of the real world in industrial fermentation. This chapter is exclusively dedicated to large-scale production used in industrial settings.
  csl behring gene therapy: Plunkett's Health Care Industry Almanac 2006 Jack W. Plunbett, 2005-11 Plunketts Health Care Industry Almanac is the only complete reference to the American Health Care Industry and its leading corporations. Whatever your purpose for researching the health care field, youll find this massive reference book to be a valuable guide. No other source provides this books easy-to-understand comparisons of national health expenditures, emerging technologies, patient populations, hospitals, clinics, corporations, research, Medicare, Medicaid, managed care, and many other areas of vital importance. Included in the market research sections are dozens of statistical tables covering every aspect of the industry, from Medicare expenditures to hospital utilization, from insured and uninsured populations to revenues to health care expenditures as a percent of GDP. A special area covers vital statistics and health status of the U.S. population. The corporate analysis section features in-depth profiles of the 500 major for-profit firms (which we call The Health Care 500) within the many industry sectors that make up the health care system, from the leading companies in pharmaceuticals to the major managed care companies. Details for each corporation include executives by title, phone, fax, website, address, growth plans, divisions, subsidiaries, brand names, competitive advantage and financial results. Purchasers of either the book or PDF version can receive a free copy of the company profiles database on CD-ROM, enabling key word search and export of key information, addresses, phone numbers and executive names with titles for every company profiled.
  csl behring gene therapy: Advances in thrombin generation Romy De Laat-Kremers, Stéphane Zuily, Bas De Laat, 2023-04-26
  csl behring gene therapy: Neonatal Hematology Pedro A. de Alarcón, Eric J. Werner, Robert D. Christensen, Martha C. Sola-Visner, 2021-02-18 Neonatal hematology is a fast-growing field, and the majority of sick neonates will develop hematological problems. This is an essential guide to the pathogenesis, diagnosis and management of hematologic problems in the neonate. Guidance is practical, including blood test interpretation, advice on transfusions and reference ranges for hematological values. Chapters have been thoroughly revised according to the latest advances in the field for this updated third edition. Topics discussed include erythrocyte disorders, platelet disorders, leukocyte disorders, immunologic disorders and hemostatic disorders. Coverage of oncological issues has been expanded to two separate chapters on leukemia and solid tumors, making information more easily accessible. Approaches to identifying the cause of anemia in a neonate are explained, with detailed algorithms provided to aid clinicians in practice. Covering an important hematologic niche with an ever increasing amount of specialized knowledge, this book is a valuable resource for hematologists, neonatologists and pediatricians.
  csl behring gene therapy: Allergy and Immunology, An Issue of Medical Clinics of North America Andrew Lutzkanin, Kristen M. Lutzkanin, 2024-06-03 In this issue of Primary Care: Clinics in Office Practice, guest editors Drs. Andrew Lutzkanin and Kristen M. Lutzkanin bring their considerable expertise to the topic of Allergy and Immunology. Top experts in the field cover updated treatment strategies for common allergies encountered in primary care, including asthma, food and drug allergies, immunodeficiency, eosinophilic esophagitis, and more. - Contains 12 practice-oriented topics including atopic dermatitis; drug allergy; acute and chronic urticaria and angioedema; venom hypersensitivity; contact dermatitis; and more. - Provides in-depth clinical reviews on allergy and immunology, 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.
  csl behring gene therapy: Optimized Gene-Engineering and Combination Therapies to Boost γδT Cell Immunotherapeutic Performance Jonathan Fisher, Daniel Abate-Daga, Marta Barisa, 2024-04-25
  csl behring gene therapy: BREAKTHROUGHS IN MEDICINE- A COMPENDIUM Prof. (Dr). K. Prathapan, Prof. (Dr.) Ullas Mony, Mr. Yogesh V. Chimate, Mrs. Shweta S. Pakale, Mr. Yogesh B. Thakare, 2024-09-19 This book, edited by Prof. (Dr.) K. Prathapan, Prof. (Dr.) Ullas Mony, Mr. Yogesh V. Chimate, Mrs. Shweta S. Pakale, and Mr. Yogesh B. Thakare, offers a comprehensive exploration of key concepts, developments, and breakthroughs in the field of medicine. As rapid advancements in science and medicine continue to shape healthcare, this compendium provides an essential resource for students, researchers, and healthcare professionals aiming to stay informed. Covering a broad spectrum of topics, including anatomy, physiology, pharmacology, and pathology, the book serves as a valuable tool for deepening understanding of the complex relationship between science and medicine, ultimately contributing to improved healthcare practices and patient outcomes.
  csl behring gene therapy: Conn's Current Therapy 2022 Rick D. Kellerman, David P. Rakel, KUSM-W Medical Practice Association, 2021-12-21 Trusted by clinicians for nearly 75 years, Conn’s Current Therapy presents today’s evidence-based information along with the personal experience and discernment of expert physicians. The 2022 edition is an excellent resource for a wide range of healthcare providers, including primary care, subspecialists, and allied health, providing current treatment information in a concise yet in-depth format. More than 300 topics have been carefully reviewed and updated to bring you state-of-the-art content in even the most rapidly changing areas of medicine. Offers personal approaches from recognized leaders in the field, covering common complaints, acute diseases, and chronic illnesses along with the most current evidence-based clinical management options. Follows a consistent, easy-to-use format throughout, with diagnosis, therapy, drug protocols, and treatment pearls presented in quick-reference boxes and tables for point-of-care answers to common clinical questions. Includes new and significantly revised chapters on COVID-19 and post-COVID syndrome, pyoderma gangrenosum, mitochondrial disease, gender affirming care, stem cell therapy, and artificial intelligence. Incorporates more electronic links throughout the text that connect the reader to apps and clinical prediction tools that can easily be accessed in practice. Features thoroughly reviewed and updated information from many new authors who offer a fresh perspective and their unique personal experience and judgment. Provides current drug information thoroughly reviewed by PharmDs. Features nearly 300 images, including algorithms, anatomical illustrations, and photographs, that provide useful information for diagnosis.
  csl behring gene therapy: The Challenge of CMC Regulatory Compliance for Biopharmaceuticals John Geigert, 2014-07-08 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.)
  csl behring gene therapy: Neonatal Hematology Pedro de Alarcón, Eric Werner, Robert D. Christensen, 2013-01-10 Fully updated new edition of a successful practical guide to the pathogenesis, recognition and management of hematologic problems in neonates.
  csl behring gene therapy: Pediatric Oncology Nursing Deborah Tomlinson, Nancy E. Kline, 2010-02-05 Pediatric Oncology Nursing: Advanced Clinical Hand- signs, diagnostic and laboratory procedures, tre- book is a joint effort between nurses in Canada, the ment, prognosis, and follow up care are included for United Kingdom, and the United States. This is the each of the disorders. second edition, and has been another wonderful Part III covers cancer treatment, including c- opportunity to bring together the expertise of hema- motherapy, radiation therapy, hematopoetic stem cell tology and oncology nurses from two continents. The transplantation, surgery, gene therapy, biotherapy, c- book is designed to be a comprehensive clinical hand- plimentary and alternative medicine, and clinical trials. book for nurses in advanced practice working with The principles and description of treatment, method pediatric hematology/oncology patients. Speci? c of treatment delivery, potential side effects, and special issues related to young children and adolescents with considerations for each type of treatment are discussed. cancer and hematologic disorders are discussed. Part IV focuses on the side effects of cancer Thirty-one contributors and two editors partici- treatment in relation to metabolic processes and pated in the writing of this text. Individuals in advanced gastrointestinal, hematologic, respiratory, renal, c- practice and academic roles – nurse practitioners, clin- diovascular, neurologic, musculoskeletal, integum- ical nurse specialists, nutritionists, clinical instructors, tary, endocrine, and auditory systems. The incidence, lecturers, academicians, and educators – were involved. etiology, treatment, prevention, and prognosis are One of the most appealing features of this text is the included for each side effect reviewed.
Global Biotechnology Company | CSL
CSL Behring is a global leader in developing and delivering high-quality medicines that treat people with rare and serious diseases.

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As a global biotech leader we are a diverse collective of dedicated people exploring every avenue, every day, to achieve one shared goal—to reimagine, reinvent and be ever inspired to find …

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Biotech leader CSL has manufacturing centers, sales offices, and locations to serve patients and public health around the globe.

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CSL consists of CSL Behring, CSL Seqirus and CSL Vifor. Together they help ensure people everywhere get the treatments they need.

Financial Results and Information | Global Biotechnology Company …
CSL consists of CSL Behring, CSL Seqirus and CSL Vifor. Together they help ensure people everywhere get the treatments they need.

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CSL's product portfolio focuses on innovation in new products, improved products and manufacturing expertise thereby ensuring our continued growth.

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CSL Plasma operates one of the world’s largest and most sophisticated plasma collection networks.

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CSL conducts clinical trials on all of its products. See if one of our current clinical trials might be right for you.

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Investment in R&D is an important driver for CSL’s future growth. New and exciting opportunities allow us to address previously unmet patient needs and these continue to drive us each day. …

Driven by Our Promise - CSL
protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet medical needs and help patients lead full …

Product Monograph Including Patient Medication Information …
Recombinant adeno-associated virus 5 vector-based gene therapy (rAAV5-hFIXco-Padua) 1 × 1013 genome copies/mL, suspension for intravenous infusion ATC Code: B02BD16 …

CSL Limited
Oct 17, 2018 · 20 CSL Behring 28 Seqirus 32 Research and Development OUR COMPANY 36 Directors 38 Global Leadership Group 40 Share Information ... Acquisition of Calimmune …

Half year reported NPATA US$2 billion1,2 - CSL Limited
“Our transformational gene therapy product for haemophilia B patients, HEMGENIX ®, is attracting significant interest from patients and health care professionals and patient referrals …

Gene therapies and genetically modified cellular therapies
Gene therapies and genetically modified cellular therapies 6. Gene editing: A trend to watch. An innovative technique called . gene editing is already changing . the field of gene therapy. Gene …

CSL Shareholder Briefings - CSL Limited
CSL Behring •Ig franchise growing strongly •Record levels of plasma collections CSL Seqirus •Strong sales growth driven by differentiated products •Licence agreement for late-stage self …

HEMGENIX demonstrates long-term durability, safety, and …
©2023 CSL Behring LLC 1020 First Avenue, PO Box 61501, King of Prussia, PA 19406-0901 USA www.CSLBehring.com www.HEMGENIX.com USA-HGX-0558-DEC23 ... gene therapy …

PRODUCT PIPELINE GENE THERAPY (March 8, 2023)
uniQure/CSL Behring Gene therapy for hemophilia B CSL Behring reported the latest results from the Phase 3 trial of Etranacogene Dezaparvovec at the February 2023 EAHAD conference. …

CSL Highlights Progress Across Strong R&D Pipeline - CSL …
Sep 13, 2022 · • Cell & Gene Therapy • Preventative Vaccines • Iron Therapy. 6. R&D Highlights – FY22 • Garadacimab(Anti-FXIIa) HAE • Phase III study enrolment completed (Last ...

Driven by Our Promise - CSL Behring
CSL Behring’s subcutaneous C1-INH therapy is used prophylactically to treat symptoms of HAE, which is characterized by acute attacks of edema (swelling) of the face, larynx (airways), ...

CSL’s Statement on Modern Slavery
of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation and continually refine ways in which products can address unmet …

CSL Behring Corporate Brochure - Updated Nov. 2021
Gene Therapy Respiratory Cardiovascular and Metabolic Immunology Hematology Transplant For more than a century, CSL Behring has linked its strong foundation of science to discovering, …

PRODUCT PIPELINE GENE THERAPY (November 2, 2023)
uniQure/CSL Behring Gene therapy for hemophilia B CSL Behring reported the latest results from the Phase 3 trial of etranacogene dezaparvovec at the February 2023 EAHAD conference. …

CSL Limited 2018 Full Year Results
gene therapy platform •Differentiating technology: - Cytegrity - Select+ •Phase 3 study commenced: - ~17,400 patients - 40 countries •First patient enrolled March 2018 •Futility #1, …

Results Announcement for the Half year ended 31 December …
Dec 31, 2024 · IDELVION®, CSL Behring’s novel long-acting recombinant factor IX product achieved growth of 6%3 and continues to be the market leader in key markets. Uptake of …

2020 JP MORGAN HEALTHCARE CONFERENCE …
Today, CSL — including our two businesses, CSL Behring and Seqirus - provides life-saving products to more than 70 countries and employs 25,000 people. Our unique combination of …

Medical Services Advisory Committee (MSAC) Public …
Applicant: CSL Behring (Australia) Pty Ltd. Date of MSAC consideration: 1-2 August 2024 . ... Hemgenix is a viral-based gene therapy – this means that an inactive virus (that can’t …

A red blood cell disease meets a company with a plasma …
CSL Behring has earned a reputation as an ... transplants and gene therapy. SCD is an important haematologic disease that presents an opportunity to apply the historical strengths

PRODUCT PIPELINE GENE THERAPY (March 13, 2024)
CSL Behring/uniQure Gene therapy for hemophilia B CSL Behring reported the latest results from the Phase 3 trial of etranacogene dezaparvovec at the December 2023 ASH conference. 96% …

Half Year Profit of US$1.62 Billion1,2 - CSL Limited
CSL Vifor progressing well CSL delivered a net profit after tax of $1.62 billion2 for the first half of financial year 2023, steady 3at CC . This included one-off costs associated with the acquisition …

News in brief FDA approves first haemophilia B gene therapy …
B gene therapy The US FDA approved CSL Behring and uniQure’s etra­ nacogene dezaparvovec for haemophilia B. It is the fifth gene therapy for a rare genetic disease to secure FDA …

CSL Limited Tax Transparency Report 2020/21
CSL also entered the cell and gene therapy field in 2017 with the acquisition of Calimmune, and more recently in 2020/21 expanded our ... CSL Behring meets patients needs using the latest …

Pharmacy Focus Hemgenix and Beqvez Gene Therapies - HMIG
Hemgenix, produced by CSL Behring, was approved November 22, 2022, and Beqvez, produced by Pfizer, was approved April 24, 2024. Both are approved for use in adult patients with …

For immediate release 11 February 2025 - investors.csl.com
IDELVION®, CSL Behring’s novel long-acting recombinant factor IX product achieved growth of 6%3 and continues to be the market leader in key markets. Uptake of HEMGENIX®, CSL's …

Immunogenicity - cslbehringevents.com
Gene therapy, specifically gene transfer, takes advantage of the ability of viruses to deliver genetic material to host cells. A functional gene is inserted into the viral vector and is …

Approved & Investigational Cell and Gene Therapies Pipeline
Below is a brief and limited overview of several investigational cell and gene therapy clinical trials that Emerging Therapy Solutions® (ETS) is monitoring in the United States. For combined …

Guidelines for the following medications - Banner Health
Feb 12, 2025 · 2. Has not received prior treatment with any gene therapy for hemophilia B AND 3. AAV5 Neutralizing Antibody test processing conducted with CSL Behring and documentation …

CSL Limited
20 CSL Behring 28 Seqirus 32 Research and Development OUR COMPANY 36 Directors 38 Global Leadership Group 40 Share Information ... Acquisition of Calimmune provides CSL with …

Hemgenix as First Gene Therapy for Treatment of …
Etranacogene dezaparvovec (Hemgenix, CSL Behring) is an adeno-associated virus vector-based gene therapy that carries a gene for clotting factor IX and is given as a one-time IV infusion. …

CSL Limited
Institute to develop stem cell gene therapies for PID –WAS and XLA • CSL200 in SCD Phase I study initiated • CSL889 Hemopexin Phase I SCD study initiated • FDA granted CSL200 fast …

2018 Half Year Results - csl.com
North America 46% EU 27% Asia Pac 17% EM 8% 8% 11% 21% CSL Behring Revenue – 1H18 4 | Driven by Our Promise™ Therapy PCP 1 1H18 vs 1H17 TP 1H18 vs 2H17 …

etranacogene dezaparvovec concentrate for solution for …
Etranacogene dezaparvovec is an advanced therapy medicinal product. It is a gene therapy that employs a non-replicating, recombinant adeno-associated virus-based vector serotype 5 …

Etranacogene Dezaparvovec: First Approval - Springer
replacement therapy, gene therapy is being investigated as a potential new treatment paradigm for haemophilia B [1 –3]. Etranacogene dezaparvovec (etranacogene dezaparvovec-drlb; …

Prescription Form Instructions - Accredo
HEMGENIX is an adeno-associated virus vector-based gene therapy indicated for the treatment of adults with Hemophilia B (congenital Fact or . IX deficiency) who: ... programs (if one or …

CSL LIMITED ANNUAL GENERAL MEETING 2024
CSL Limited P: +61 455 022 740 E: chris.cooper@csl.com.au Bernard Ronchi Director, Investor Relations CSL Limited P: +61 3 9389 3470 E: bernard.ronchi@csl.com.au Stephen McKeon …

Optimize Cell Line Selection, Unveil Complex
perspectives from CSL Behring and LNP-mRNA insights from Sanofi. Discover how these innovative delivery mechanisms can enhance integration, optimize efficacy, and apply these …

CSL Behring Moves Forward to Ensure Patients Receive their …
CSL Behring is a global biotherapeutics leader driven by our promise to save lives. Focused on serving patients’ needs by using the latest technologies, we discover, develop and deliver …

Driven by Our Promise Modern Slavery Statement 2023/24
therapeutic areas. CSL Behring uses three strategic scientific platforms of plasma fractionation, recombinant protein technology, and cell and gene therapy to support continued innovation …

Half Year Profit of US$1.62 Billion Ig franchise growing …
CSL Vifor progressing well CSL delivered a net profit after tax of $1.62 billion2 for the first half of financial year 2023, steady 3at CC . This included one-off costs associated with the acquisition …

Bill Mezzanotte MD - CSL
clinical science, statistics and clinical operations across CSL Behring's portfolio. Most recently he served as Senior Vice President and Head of Development, where he oversaw regulatory, …

SECTOR SNAPSHOT - Alliance for Regenerative Medicine
HEMGENIX (GENE THERAPY) CSL Behring and UniQure Hemophilia B CTX001 (GENE EDITING THERAPY) CRISPR Therapeutics & Vertex Pharmaceuticals Sickle cell disease, ß …

An optimised protocol for quality control of gene therapy
Dec 3, 2023 · 2CSL Behring, Research, Bern, Switzerland 3Swiss Institute for Translational Medicine, sitem-insel, Bern, Switzerland 4Research Data Science Group, R&D, CSL, …

Hemophilia - Gene Therapy - Hemgenix UM Medical Policy
Mar 26, 2025 · POLICY: Hemophilia – Gene Therapy – Hemgenix Utilization Management Medical Policy • Hemgenix® (etranacogene dezaparvovec- drlb intravenous infusion − CSL …

Regenerative Medicine: 2022 The Pipeline Momentum Builds
on a new gene therapy to treat a rare disease — EtranaDez from uniQure and CSL Behring to treat Hemophilia B — is expected by the end of 2022, perhaps in both the US and Europe. In …

Gene Therapy with Etranacogene Dezaparvovec for …
ed by uniQure and CSL Behring; HOPE-B ClinicalTrials.gov number, NCT03569891.) ... gene therapy for hemophilia B. 25,26 A single infu-sion of AMT-060 (5×1012 or 2×1013 genome copies

PATIENT SERVICES ENROLLMENT FORM
(6) evaluating the effectiveness of CSL Behring’s support program(s) (7) providing any other related support, education, and assistance services to me related to my treatment with CSL …

FY23 reported NPATA 1US$2.61 billion Up 20% at constant
the FDA approved the first and only gene therapy for the treatment of adults with haemophilia B earlier in the year”, Dr McKenzie concluded. PERFORMANCE CSL Behring Total revenue …

2024 State of the Industry Briefing - Alliance for Regenerative …
Jan 8, 2024 · Expediting the Development of Cell and Gene Therapy Peter Marks, M.D., Ph.D., Director, ... Hemgenix (UniQure and CSL Behring) Condition: Hemophilia B. 9 V W K W …

Hemophilia - Gene Therapy - Hemgenix - Cigna
b. On-demand use of Factor IX therapy was required for this life-threatening hemorrhage; OR iii. Patient meets BOTH of the following (a and b): a. Patient has a history of repeated, serious …

CSL LIMITED ANNUAL GENERAL MEETING 2024
CSL Limited P: +61 455 022 740 E: chris.cooper@csl.com.au Bernard Ronchi Director, Investor Relations CSL Limited P: +61 3 9389 3470 E: bernard.ronchi@csl.com.au Stephen McKeon …