Christian Medical College

TORONTO, April 11, 2024 (GLOBE NEWSWIRE) -- The Gairdner Foundation is pleased to announce the 2024 Canada Gairdner Award laureates, recognizing some of the world’s most significant biomedical and global health research and discoveries. “Canada is a world leader in science and innovation, and it is crucial to recognize the hard work done by its scientific community to make the world around us better. The 2024 Canada Gairdner Awards recipients’ contributions to science and research are truly exemplary, standing out on the global stage. The work of these laureates will most definitely improve the health of people in Canada and around the world. Congratulations to this year’s recipients!” - The Honourable François-Philippe Champagne, Minister of Innovation, Science and Industry “Congratulations to the recipients of the 2024 Canada Gairdner Awards who are being recognized for scientific discoveries that have transformed biomedical science, medicine, and global health. I’d like to give special mention to the Canadian recipients of this year’s Canada Gairdner Momentum Awards, Dr. Meghan Azad and Dr. Christian Landry. The outstanding work of these researchers will have a profound and lasting impact on the health of people in Canada and around the world.” – The Honourable Mark Holland, Minister of Health “The Gairdner Awardees showcase the world’s best science and innovation to improve human health. This year is no exception. Join us to get to know these exceptional individuals and their passions.” - Rémi Quirion, Scientifique en chef du Québec Président des CA des Fonds de recherche du Québec, partenaires de la Fondation Gairdner 2024 Canada Gairdner International Award The five 2024 Canada Gairdner International Award laureates are recognized for seminal discoveries or contributions to biomedical science: Zelig Eshhar, PhDWeizmann Institute of Science, Rehovot, Israel Michel Sadelain, MD, PhDDirector, Center for Cell Engineering, Stephen and Barbara Friedman Chair, Sloan Kettering Institute, Memorial Sloan Kettering Cancer Center, New York, NY, USA Awarded “For seminal contributions to the conception, development and application of CD19-CAR T cell therapy for cancer.” The Work: Chimeric antigen receptor (CAR) T cell therapy is a type of cancer immunotherapy. It uses immune cells, called T cells, that are genetically altered in the lab to enable them to locate and destroy cancer cells. Both Eshhar’s and Sadelain’s trailblazing research into CAR T cell engineering spans many decades. Their combined efforts defined the pathway for the future of cellular immunotherapy to treat cancer. Eshhar has focused his career around the genetic engineering of T cells. These studies led him to predict that combining the killing machinery of cytotoxic T cells with an antibody’s specificity and high affinity to a target, would lead to new T cell-based therapies. He developed the first chimeric antigen receptor system in the 1980s and introduced it into T cells armed with an anti-tumor specificity. Eshhar went on to demonstrate the ability of such CAR T cells to successfully treat cancer in animal models. Sadelain began investigating what would eventually be referred to as CAR T cell therapy in the late 1980s, publishing his first abstract on successful T-cell engineering in 1992. Over the next decade, Sadelain and his team improved upon Eshhar’s design and by 2002 built the first truly effective CAR T cells, paving the way for their clinical use. Key to this breakthrough was the demonstration that CD19, a molecule on white blood cells, could serve as an effective target for CAR T cells in blood-borne cancers. These second-generation CAR T cells were able to survive, proliferate and kill cancer cells in the lab, which established the feasibility and proof of concept for producing genetically instructed, targeted immune responses. In 2003, Sadelain and his colleagues published a seminal research paper showing that human CD19-directed CAR T cells could eradicate leukemia cells in a mouse model. Soon afterwards, clinical trials led by different groups, including Dr. Sadelain’s, demonstrated that the tumor burden in refractory chronic lymphocytic leukemia and adult and pediatric acute lymphoblastic leukemia could be successfully reduced and eventually become undetectable. The first CAR T cell therapies, targeting CD19, were approved by the U.S. Food and Drug Administration in 2017 for use in children and young adults with refractory acute leukemias and certain refractory lymphomas. The Impact: Currently, CAR T cell products have been approved worldwide for multiple indications, including large B-cell lymphoma, B-cell acute lymphoblastic leukemia, mantle cell lymphoma and follicular lymphoma. CAR T cell therapy has revolutionized the treatment landscape of relapsed and refractory B-cell malignancies. In contrast to conventional cytotoxic chemotherapy or immunotherapy with monoclonal antibodies, CAR T-cell therapy can induce durable and complete responses after a single treatment course. Such responses stem from the ability of CAR T cells to expand in vivo and persist for several months or years, which leads to continuous therapeutic efficacy and tumor control. Efforts are underway to expand the repertoire of cancers that can be treated by CAR T cells through identifying new target antigens in solid tumors. The success of CAR T cells has also fueled interest in expanding their application beyond cancer. CAR T cells are demonstrating high response rates in autoimmune diseases and are being investigated as potential treatments for additional disorders, including fibrosis, senescence-associated pathologies and infectious diseases. The next set of 2024 Canada Gairdner International Award laureates are: Shankar Balasubramanian, FRSHerchel Smith Professor of Medicinal Chemistry, Yusuf Hamied Department of Chemistry, University of Cambridge David Klenerman, FRS, FmedSciProfessor of Biophysical Chemistry, Yusuf Hamied Department of Chemistry, University of Cambridge Pascal Mayer, PhDCEO and Co-founder of Alphanosos; Honorary Chair at the Institute for Advanced Study of the University of Strasbourg (USIAS) Awarded “For the fundamental and applied research that led to a revolutionary and affordable method to sequence DNA on a massive scale, which has dramatically accelerated discoveries in the life sciences and medicine” The Work: Balasubramanian, Klenerman and Mayer are recognized for developing the underlying methodologies that led to Solexa-Illumina Next Generation DNA Sequencing (NGS), a technology that has enhanced our basic understanding of life, converting biosciences into ‘big science’ by enabling fast, accurate, low-cost and large-scale genome sequencing – the process of determining the complete DNA sequence of an organism. Balasubramanian and Klenerman’s original work involved methods to observe a single molecule of DNA polymerase incorporating fluorescently labelled DNA monomers onto a growing strand of DNA hybridised to an immobilised template. They had the vision to see how this science could be extended and applied to sequencing a vast array of immobilised DNA molecules in a way that would bring huge benefits of scale, speed and cost. They co-founded the company Solexa, later acquired by Illumina, to make the technology available to the world. Mayer conceived and performed the initial development of 'DNA colony-based massively parallel sequencing by synthesis', initially at Glaxo-Wellcome’s and Serono’s research institutes in Geneva, and finally at a spin-off company, Manteia Predictive Medicine, where he was Chief Scientific Officer. This cluster technology is a key component of the Solexa-Illumina massively parallel sequencing technology. Manteia and its intellectual property were acquired in 2004 by Solexa. NGS has had – and continues to have – a transformative impact in the fields of genomics, biology and medicine. It has allowed a million-fold improvement in speed and cost when compared to the first sequencing of the human genome. In 2000, sequencing of one human genome took over 10 years and cost more than a billion dollars; today, the entire genome of multiple humans can be sequenced in a single day at a cost of less than $1,000. More than a million human genomes have been sequenced, and continue to be sequenced at scale each year, as well as genomes of animals, plants, bacteria and viruses, providing unprecedented insights into genome variation across the planet. The Impact: NGS has had an enormous impact on life sciences. Many aspects of the basic research into mechanisms in living systems now routinely involve high-throughput sequencing of DNA or RNA as the primary readout. Clinical research is undergoing a revolution via the application of genome sequencing, and genomic applications more generally, to discover the underlying causes and markers of diseases, along with substantial new knowledge on the genetic causes and signatures of cancers. There is a wave of new clinical diagnostics emerging for cancers, rare genetic diseases and infectious diseases enabled by NGS of patient samples that now includes minimally invasive blood sampling. During the COVID-19 pandemic, NGS was essential to the rapid understanding of the viral cause of the disease, and then in identifying and monitoring the spread of new variants. Without this technique, progress towards developing vaccines and other interventions would have been slow. It is difficult to overstate the importance and impact of NGS. It has initiated a revolution in biology, enabling the revelation of unsuspected genetic diversity in humans and their pathogens, with major implications, from cell and microbiome biology to ecology, forensics and personalized medicine. 2024 John Dirks Canada Gairdner Global Health Award The 2024 John Dirks Canada Gairdner Global Health Award laureate is recognized for outstanding achievements in global health research: Gagandeep Kang, FRSAdjunct Professor, Christian Medical College, Vellore, India; Director, Enteric, Diagnostics, Genomics and Epidemiology, Bill and Melinda Gates Foundation Awarded “For extensive cohort-based epidemiological, environmental and clinical trial research on enteric diseases in children and their effects on life course, with significant impact on vaccine development and health policy in India and internationally.” The Work: Gagandeep Kang received her training in medicine and microbiology at the Christian Medical College (CMC), Vellore, in southern India. She is Professor of Microbiology at CMC, where her research group has established substantial community-based birth cohort studies addressing childhood enteric infections, growth, and development for over 20 years. Using data and insights from these studies, alongside immunological, mechanistic, epidemiological and implementation research, Prof. Kang has generated extensive information on the complex interactions between the environment and infections in children, and the influence of prior infections on subsequent responses to vaccination. Through extensive, cross disciplinary work drawing on field epidemiology, molecular understanding of infectious agents and infections, clinical trials and policy implementation, she explored the complex relationships between enteric diseases, gut function, and physical and cognitive development in affected children. Prof. Kang conducted the largest single birth cohort study on rotaviral infections in the world, demonstrating that protection after a natural infection is lower in India than in developed countries, which has important implications for the control of diseases by vaccination. As a physician-scientist, Prof. Kang has led key studies that contributed to the development and introduction of two Indian rotavirus vaccines into the national immunization program. These vaccines are now WHO prequalified and their introduction has begun in countries beyond India. She has expanded her research and collaborations to work on other enteric pathogens, particularly cholera and typhoid, with a view to further vaccine introductions; on nutritional and pharmacological interventions aimed at improving oral vaccine efficacy; and on the transmission and impact of, and vaccine effectiveness for COVID-19 in the Indian context. The Impact: Gagandeep Kang has built an internationally recognized and competitive research program centred in India that prioritizes Indian needs while providing valuable and translatable insights to other regions affected by enteric infections. In addition to understanding the mechanism of infection and its effects on individuals, Kang’s group has used geographic information systems to connect virus prevalence at the community level to cost of illness studies, resulting in India becoming the only low-income country with comprehensive disease burden estimates for which cost-effectiveness of vaccination for multiple clinical outcomes could be predicted, as well as new vaccine roll-out programs. By 2019, all 26 million children born annually in India were eligible to receive the rotavirus vaccine, which has the potential to reduce infections, associated complications and death of vulnerable children. In addition to excellent scientific research, Prof. Kang is an exemplary leader. Her approach to clinical and public health research uses a community base to identify problems that need to be addressed through laboratory science in India, and as needed, through international collaborations. In addition to her work in Vellore, India, she took on the challenge of leading the Department of Biotechnology’s Translational Health Science and Technology Institute (THSTI) from 2016 to 2020 to establish the translational science systems needed in the Indian medical research ecosystem. The programs initiated at the Institute have been critical to supporting the multiple vaccine development efforts of Indian vaccine manufacturers and have received global recognition. 2024 Canada Gairdner Momentum Award The 2024 Canada Gairdner Momentum Award laureates are mid-career investigators recognized for exceptional scientific research contributions with continued potential for impact on human health. Meghan Azad, PhDProfessor, Pediatrics and Child Health, University of Manitoba; Research Scientist, Children’s Hospital Research Institute of Manitoba; Director of Science & Knowledge Mobilization, THRiVE Discovery Lab; Co-Director, Manitoba Interdisciplinary Lactation Centre (MILC); Canada Research Chair, Early Nutrition and the Developmental Origins of Health and Disease Awarded “For research on understanding how human breast milk contributes to shaping the infant microbiome and lifelong health.” The Work: Dr. Azad is leading innovative research about breastfeeding, human breast milk and the microbiome. While previous studies have often been underpowered, narrowly focused on single milk components, inconsistent in their definition of “breastfeeding”, and limited by confounding (e.g. the socioeconomic status) issues, Dr. Azad’s research is addressing these nuances and shortcomings to provide new insights on how infant feeding practices and hundreds of different breast milk components influence health and disease throughout the life cycle. Studying over 3,000 children, Dr. Azad’s team has shown that longer and more exclusive breastfeeding is associated with healthier body composition and reduced risk of asthma. Further, their studies have provided novel evidence that the method of feeding (i.e. pumping vs. nursing at the breast) matters – possibly because bioactive milk compounds degrade in storage. Indeed, her team found that pump extraction affects the human milk microbiome and bacteria sharing between mothers and infants. The Impact: Dr. Azad is a renowned research leader whose groundbreaking work is pushing the boundaries of knowledge generation and translation in the important areas of infant nutrition, lactation, maternal-child health, and the developmental origins of disease. Moreover, she is highly dedicated to enriching the scientific community in Canada through her commitment to recruiting and training new researchers, supporting science literacy among Canadian children and the general public, and engaging Canadian policymakers to foster support for science and evidence-based policy-making. Meghan Azad is Professor of Pediatrics and Child Health at the University of Manitoba. She holds a Canada Research Chair in Early Nutrition and the Developmental Origins of Health and Disease, and she co-directs the THRiVE Discovery Lab, where her team studies how early life nutrition shapes the infant microbiome and child health. She co-founded the Manitoba Interdisciplinary Lactation Centre (MILC) and leads several interdisciplinary research teams, including the International Milk Composition (IMiC) Consortium (7 countries, 18 investigators, 1200 mother-infant pairs). In addition, she is Deputy Director of the CHILD Cohort Study, an ongoing longitudinal Canadian birth cohort of 3500 families. In 2022, she received the Steacie Prize for early career researchers. Christian Landry, Prof. Professor of biology and biochemistry, Laval University; Canada Research Chair in Cellular Systems and Synthetic Biology Awarded “For the development of novel approaches that combine synthetic biology, experimental evolution, and systems biology to address fundamental questions about gene function relevant to health and human disease." The Work: Prof. Landry’s research group is integrating cutting-edge approaches in genetics, genomics, systems biology and bioinformatics to answer one of the most long-lasting questions in biology and biomedical research: how mutations change the way cells work and alter their performance. Understanding the principles by which mutations translate into phenotypes is at the core of human genetics and other fields such as evolutionary biology and biotechnology. The overarching goal of Prof. Landry’s team is to probe the robustness and function of cellular networks, with the long-term objective of better understanding how individual variation such as mutations cause changes in complex traits and, ultimately, diseases. Over the past six years, it has developed numerous high-throughput tools that allow the investigation of entire protein interaction networks, how these networks can be rewired by mutations, and how their past evolution may influence the outcomes of disease mutations. The Impact: Christian Landry has brought an entirely new perspective to systems biology, in which networks, such as protein-protein interactions, and cellular signaling, are investigated in the context of historical evolutionary forces in order to better understand their fundamental properties. His work is of critical importance to health and human disease since all organisms evolve through the same principles, including pathogens impacting humans. His recent research has ranged from investigations of the evolution of resistance to antifungal drugs, host-pathogen arms races, and evolutionary trade-offs in cellular processes and networks, to systems biology studies of microbial interactions. In the long term, systematic analyses of mutations causing variations in complex phenotypes such as drug resistance will enable the development of drugs that are less likely to lead to resistance, which is key in the context of limited treatment options. Prof. Landry was elected in the inaugural cohort of the College of the Royal Society of Canada; he received the NSERC EWR Steacie Memorial Award for early career researchers in 2017 and the SMBE Mid-career Award for outstanding contributions in 2020. In 2021, he received Laval University's Excellence Prize for teaching and mentoring to graduate students and postdocs. He is also contributing to changing the way science is published and communicated through his work as Senior Editor for eLife and editorial advisor for EMBO Review Commons, two organizations actively involved in remodelling the scientific publication landscape to make it more transparent and equitable. About the Gairdner Foundation Established in 1957, the Gairdner Foundation is dedicated to fulfilling James A. Gairdner’s vision to recognize major research contributions to the treatment of disease and alleviation of human suffering. Through the prestigious annual Canada Gairdner Awards, the Foundation celebrates the world’s most creative and accomplished researchers whose work is improving the health and wellbeing of people around the world. Since its inception, 418 awards have been bestowed on laureates from over 40 countries, and of those awardees, 98 have gone on to receive Nobel Prizes. The Gairdner Foundation believes in coming together to openly discuss science in order to better engage the public, understand the problems we face, and work together to find solutions. Since its founding, a number of outreach events and programs have been developed with the goal of inspiring the next generation of scientific innovators and fostering an informed society. https://www.gairdner.org/ For further information, please contact:Sommer WedlockExecutive Vice PresidentGairdner Foundationsommer@gairdner.org

LACHEN, Switzerland, June 6, 2023 /PRNewswire/ -- Octapharma to present new clinical and scientific findings for wilate®, Nuwiq®, octaplex® and atenativ® at the upcoming ISTH 2023 Congress New data and clinical study developments will be presented in nine posters and during two Supported Symposia The most recent developments from Octapharma's Haematology and Critical Care portfolios will be presented at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH) on June 24–28, 2023, in Montréal, Canada. The data will be featured in nine poster presentations and two Supported Symposia during this key international meeting. Octapharma is a proud Silver Supporter of the ISTH 2023 Congress. There will also be a promotional booth focusing on modern approaches to bleeding and coagulation management. People with inherited bleeding disorders, such as haemophilia A and von Willebrand disease, are at risk of recurrent and prolonged bleeds placing a heavy burden on patients' lives. In addition to the risk of potentially life-threatening bleeds, increased bleeding is associated with long-term consequences that severely impair patients' quality of life. Patients with a deficiency of the prothrombin complex of coagulation factors are also at increased risk of bleeding. Such deficiencies may be inherited but can also be acquired and may be induced by the use of vitamin K antagonists (VKA) for anticoagulation therapy. During urgent surgery, rapid correction of the deficiency is required and can be achieved through the use of four-factor prothrombin complex concentrates (4F-PCC). 4F-PCC can also be used to control major bleeding associated with the use of direct oral anticoagulant (DOAC) therapy in patients with factor Xa inhibitors. The presentations at ISTH 2023 reflect Octapharma's long-term commitment to improving patient care and addressing unmet clinical needs. "We are proud to showcase our new data and provide updates of studies from across Octapharma's haematology and critical care portfolios at ISTH 2023," said Olaf Walter, Board Member at Octapharma. "Despite advances in the field, people with bleeding and coagulation disorders still face considerable challenges surrounding bleed management. At Octapharma, we strive to address such challenges head-on with the goal to improve the day-to-day lives of patients." Poster Presentations The WIL-31 study investigated the efficacy and safety of prophylaxis with wilate® in patients with von Willebrand disease (VWD) of any gender with VWD type 1, type 2 (except 2N) or type 3. The results of the study show that wilate® prophylaxis is highly effective at reducing the number of bleeds in children and adults, and provides compelling evidence to support the use of regular prophylaxis in patients with severe VWD. Further data on the efficacy and safety of wilate® prophylaxis is being collected in the WIL-33 study in children under six years of age. The goals and design of this study will be presented. Prophylaxis is already the standard of care for people with haemophilia A. The results of an indirect comparison show that fewer patients experienced bleeds when receiving personalised prophylaxis with Nuwiq® rather than prophylaxis with a comparator factor VIII (FVIII) product. New data exploring differences in the binding of different recombinant FVIII products to platelets in vitro will be shared in a separate poster. Although haemophilia A mainly affects males, female carriers may also have reduced levels of FVIII and be at increased risk of bleeding, especially during traumatic events such as surgery. Data from a retrospective case series of female haemophilia A carriers who were prescribed Nuwiq® during surgery at the Bonn Haemophilia Centre will provide an insight into the effective treatment of females. MOTIVATE is an ongoing, international investigator-initiated study made possible through funding from Octapharma AG, evaluating the management of haemophilia A patients with inhibitors. The Coordinating investigators, Drs Carmen Escuriola Ettingshausen and Robert F. Sidonio Jr., will provide an update on the study status as a poster at the Congress. The LEX-210 study will evaluate the haemostatic efficacy of octaplex® in patients with acute major bleeding receiving factor Xa inhibitors. As anti-factor Xa (anti-Xa) activity levels will be measured in these patients, the correlation between heparin-calibrated and specific factor Xa inhibitor assays has been analysed and will be presented. The results from the first head-to-head, phase 3, randomised, controlled trial (LEX-209) comparing two 4F-PCCs in patients on VKA anticoagulation who need urgent surgery will be presented, with a focus on dosing and haemostatic efficacy results. The results show that octaplex® was non-inferior to control 4F-PCC for rapid vitamin K antagonist reversal in patients undergoing urgent surgeries with significant bleeding risk. The posters will be presented at the following times: Sunday June 25, 18:30–19:30 ET PB0163. Differential binding of recombinant FVIII products to platelets, and impact of FVIII on VWF expression and platelet contractility in vitro. Presenting author: Viola Vogel. PB0235. Phase 3 study of the efficacy, pharmacokinetics, immunogenicity and safety of von Willebrand factor/factor VIII concentrate in patients with severe von Willebrand disease under 6 years of age. Presenting author: Akshat Jain. PB0238. Prospective, phase III study of the efficacy, safety, and pharmacokinetics of a human antithrombin III concentrate in congenital antithrombin deficiency during surgery or childbirth. Presenting author: Craig Kessler. Monday June 26, 18:30–19:30 ET PB0840. Efficacy of prophylaxis with a plasma-derived von Willebrand factor/factor VIII concentrate in patients with von Willebrand disease: Subgroup analysis by age and VWD type. Presenting Author: Robert F. Sidonio Jr. PB0681. German experience with simoctocog alfa during surgery in female haemophilia A carriers. Presenting author: Natascha Marquart. PB0584. Using heparin-calibrated assays to assess anti-factor Xa activity of factor Xa inhibitors (FXaI): a literature correlation analysis. Presenting author: Ravindra Sarode. PB1032. Four-factor prothrombin complex concentrates for vitamin K antagonist reversal before urgent surgery: dosing and hemostatic efficacy in a double-blind randomized study. Presenting author: Ravindra Sarode. Tuesday June 27, 18:30–19:30 ET PB1261. Matching adjusted indirect comparisons between personalized prophylaxis with simoctocog alfa versus standard prophylaxis with turoctocog alfa in adults with severe haemophilia A. Presenting author: Fernando F. Corrales-Medina. PB1288. Immune tolerance induction (ITI) strategies in haemophilia A patients with inhibitors – data from the MOTIVATE study. Presenting author: Carmen Escuriola Ettingshausen. Wednesday June 28, 13:00–14:00 ET PO11. Impact of simoctocog alfa prophylaxis on bleeding and joint health in Spanish patients with severe haemophilia A. Presenting author: María Teresa Alvarez-Román. Supported Symposia New clinical and scientific data, as well as updates on ongoing and new studies, will be shared during two Supported Symposia during the Congress. Final Results of the WIL-31 Study – A New Treatment Standard for von Willebrand Disease Patients with Severe Bleeding? Monday June 26, 13:15–14:30 ET, Room 511 Chair: Alok Srivastava, Christian Medical College, Vellore, India The symposium will focus on new data regarding the use of wilate® prophylaxis in children and adults to improve the lives of people with VWD. Topics presented will include the importance of prophylaxis, including current guidelines on the management of VWD and how to identify patients who may benefit from prophylactic treatment. Key learnings from the WIL-31 VWD prophylaxis study will be shared and case presentations of select WIL-31 patient cases from across the globe will be discussed. Bringing Together Two Worlds – Scientific and Clinical Insights for All-Round Bleed Protection in Haemophilia A Tuesday June 27, 13:15–14:30 ET, Room 511 Chair: Davide Matino, McMaster University, Hamilton, Ontario, Canada Octapharma's symposium on haemophilia A this year is dedicated to both, scientists and clinicians. The speakers will present data from the NuPreviq trial and the matching adjusted indirect comparisons (MAIC) analyses of Nuwiq® vs extended half-life (EHL) products as well as introduce the PROVE study. Topics discussed during this symposium will also include the role of platelets in FVIII binding, impact of prophylaxis on microRNA expression (biomarkers of joint and bone health) and how to utilise the power of RNASeq to understand inhibitor development. Both symposia will be open to congress participants in Montréal and will be streamed live for participants not attending the Congress. The opportunity to ask questions will be available at both symposia. Larisa Belyanskaya, Head of IBU Haematology at Octapharma commented: "We are delighted to showcase new data from WIL-31, which supports the importance of prophylaxis in people with von Willebrand disease, and to discuss scientific and clinical questions that need to be address to advance the management of people with haemophilia A." About Octapharma Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines. Octapharma employs more than11,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Immunotherapy, Haematology and Critical Care. Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 190 plasma donation centres across Europe and the US. Octapharma has 40 years of experience in patient care. About Nuwiq® Nuwiq® (simoctocog alfa) is a 4th generation recombinant factor VIII (rFVIII) protein, produced in a human cell line without chemical modification or fusion with any other protein[1]. It is cultured without additives of human or animal origin, is devoid of antigenic non-human protein epitopes and has a high affinity for von Willebrand factor[1]. Nuwiq® treatment has been assessed in nine[1-3] completed clinical trials which included 201 previously treated patients (190 individuals)[1] and 108 previously untreated patients[2] with severe haemophilia A. Nuwiq® is available in 250 IU, 500 IU, 1,000 IU, 1,500 IU, 2,000 IU, 2,500 IU, 3,000 IU and 4,000 IU presentations[4]. Nuwiq® is approved for use in the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital FVIII deficiency) across all age groups[4]. About wilate ® wilate® is a high-purity human von Willebrand factor/factor VIII (VWF/FVIII) concentrate, that undergoes two virus inactivation steps during its production[5]. No albumin is added as a stabiliser[5]. The purification processes result in a 1:1 ratio of VWF to FVIII that is similar to normal plasma[5]. wilate® contains a VWF triplet structure and content of large high molecular weight multimers similar to normal human plasma[5]. wilate® is exclusively derived from large pools of human plasma collected in approved plasma donation centres[6]. wilate® is available in 500 IU and 1,000 IU presentations. wilate® is indicated for the prevention and treatment of haemorrhage or surgical bleeding in von Willebrand disease (VWD), when desmopressin (DDAVP) alone is ineffective or contra-indicated, and for the treatment and prophylaxis of bleeding in patients with haemophilia A (congenital factor VIII deficiency)[6]. About octaplex® octaplex® is a human four factor prothrombin complex concentrate (4F-PCC) containing Factors II, VII, IX and X, as well as inhibitor protein C and its cofactor protein S. octaplex® is indicated for the treatment of bleeding and perioperative prophylaxis of bleeding in acquired deficiency of the prothrombin complex coagulation factors, such as deficiency caused by treatment with vitamin K antagonists, or in case of overdose of vitamin K antagonists, when rapid correction of the deficiency is required. It can also be used in the treatment of bleeding and perioperative prophylaxis in congenital deficiency of the vitamin K dependent coagulation factors II and X when purified specific coagulation factor product is not available. octaplex® is presented in vials containing 500 IU and 1000 IU of human prothrombin complex. The total protein content per vial is 260–820 mg (500 IU vial)/ 520–1640 mg (1000 IU vial). The specific activity of the product is ≥ 0.6 IU/mg proteins, expressed as factor IX activity. Do not store above 25°C, do not freeze. Store in the original package in order to protect from light. The product shelf life is three years.[7] About atenativ® atenativ® is a human antithrombin III concentrate (AT III), which is presented in vials containing 500 IU and 1000 IU of human antithrombin III as lyophilised powder for infusion. After reconstitution with 10 mL water for injection (WFI), the solution provides 50 IU/mL or 100 IU/mL of human antithrombin III. Store in the refrigerator (2-8 °C) Keep the vials in the outer carton in order to protect from light. The shelf life is three years. It has been indicated for treatment of patients with congenital and acquired antithrombin deficiency including prophylaxis for the prevention of deep vein thrombosis and thromboembolism in clinical risk situations (especially in surgery, pregnancy and childbirth), in combination with heparin, if indicated, as well as prevention of the progression of deep venous thrombosis and thromboembolism associated with heparin as indicated.[8] atenativ® is currently not registered in Canada. 1. Lissitchkov T et al. Ther Adv Hematol 2019; 10:2040620719858471. 2. Liesner RJ et al. Thromb Haemost 2021; 121:1400-8. 3. Octapharma AG; Data on file. 4. Nuwiq® Summary of Product Characteristics. 5. Stadler M et al. Biologicals 2006; 34:281-8. 6. wilate® Summary of Product Characteristics. 7. octaplex® Summary of Product Characteristics. 8. atenativ® Summary of Product Characteristics. Octapharma press releases are specifically for health specialist/medical media and are not for consumer press. 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FRIDAY, March 31, 2023 -- An experimental injectable drug appears effective in reducing bleeds in patients with hemophilia A and B, according to a pair of new clinical trials. Two-thirds of people with treatment-resistant hemophilia who were treated with the drug fitusiran had no bleeds at all after nine months, versus just 5% of people treated with drugs that enhance clotting, according to a trial published online March 29 in The Lancet . Fitusiran also outperformed other drugs in a second trial that involved people with hemophilia. In that trial, 51% of patients experienced no bleeds compared to 5% of the control group. That report appears in The Lancet Hematology . The drug is the first treatment that can be given in advance to prevent bleeds in people with both types of hemophilia, regardless of whether their disease is resistant to treatment, the researchers reported. Hemophilia is an inherited bleeding disorder. People with hemophilia A or B are missing natural proteins that help form blood clots to stop bleeding. Without those proteins, these patients bleed spontaneously into their joints and muscles, and can take much longer to stop bleeding after an injury. Fitusiran is a type of drug called a small interfering RNA therapy. These drugs work by interfering with the production of specific proteins in the body. Fitusiran targets antithrombin, a protein that reduces blood clotting. As a result, patients are better able to form clots that stop dangerous bleeding. The drug is delivered via a monthly injection. The first clinical trial was conducted at 26 hospitals in 12 countries. It included 56 male patients, aged 12 and older, with severe treatment-resistant hemophilia. Two-thirds were treated with fitusiran, while the others (the "control" group) were given drugs that enhance clotting as needed. The average annual bleeding rate among fitusiran patients was 0, compared to nearly 17 bleeds on average in the control group. “The data is encouraging and suggests it may be the first prophylactic treatment — meaning it can be given to prevent bleeds rather than to treat them after they have already occurred — that works for both hemophilia A and B patients with inhibitors (treatment resistance),” lead researcher Guy Young said in a journal news release. “Hemophilia B patients’ treatment options are currently limited to on-demand treatments, which treat bleeds after they have occurred,” added Young, a professor at the University of Southern California Keck School of Medicine, in Los Angeles. The second trial involved 45 hospitals in 17 countries, with 120 male patients, aged 12 or older, who had hemophilia but no treatment resistance. Again, two-thirds were treated with monthly fitusiran, while the others were given clotting drugs on demand. The average annual bleeding rate among fitusiran patients in this study was 0, compared to nearly 22 bleeds on average in the control group. The main side effect in both studies was an increase in levels of alanine aminotransferase, an enzyme released into the blood when the liver is damaged. About 32% of fitusiran patients experienced an increase in this enzyme in the first trial, and 23% in the second trial. No deaths were reported in either trial. “Our study looks at the use of fitusiran in patients with hemophilia A or B without inhibitors and complements the findings from the study looking at fitusiran with inhibitors, also finding that it is very effective in preventing bleeds,” said lead researcher Alok Srivastava of Christian Medical College in Vellore, India. “Fitusiran is administered by under-the-skin injections, which can be easily taken at home. With this drug being administered just once a month or even less frequently, there is marked reduction of treatment burden,” Srivastava continued. “This means patients with hemophilia could manage their condition with fewer trips to [the] hospital, which can cause worry and be disruptive to daily life. This would lead to an improved quality of life as documented in the study.” Both clinical trials were funded by Sanofi, the pharmaceutical company that created fitusiran. Sources The Lancet , news release, March 29, 2023 Posted March 2023