Universitätsklinikum Würzburg

Applications for the independently assessed, expert-led Research Grant Initiative, Find For Rare, are open from today, with experts calling for innovative research to broaden the level of understanding into three lysosomal storage disorders and, ultimately, improve patient care and management. Researchers can apply for grants up to €50,000 – the research grants are designed to support original research projects that aim to advance knowledge in the field of: Fabry disease, alpha-mannosidosis, and cystinosis. All applications will be independently reviewed and assessed by a Steering Committee of leading experts in the lysosomal storage disorders field, who have been identified, contracted and funded by Chiesi, with the successful applicants announced in Q1 2025. PARMA, Italy, Sept. 9, 2024 /PRNewswire/ -- Chiesi Global Rare Diseases, a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases, has today announced a new Research Grant Initiative, Find For Rare. Researchers are invited to apply for grants up to €50,000 to support their research into lysosomal storage disorders (LSDs). LSDs are inborn errors of metabolism, rare genetic disorders characterized by defective functioning of lysosomes.1 Whilst the disorders can be rare individually, their prevalence is significant at a global level with an estimated 1 in 8000 people diagnosed.1 To address current gaps in understanding, Chiesi Global Rare Diseases is organizing and funding the new call for research into three LSDs – Fabry disease, alpha-mannosidosis, and cystinosis. The initiative's ambition is to provide a platform for innovative research into improving the understanding of the factors affecting the diseases as well as patient-tailored clinical management. All submitted research proposals will be evaluated by the Find For Rare Steering Committee, an independent scientific board of 10 leading experts in the field of LSDs, chaired by three of the experts, including Professor Christoph Wanner. "Find For Rare is calling for all principal investigators and scientists with an interest in lysosomal storage disorders to apply for this fantastic research opportunity," said Professor Christoph Wanner, a Chair of the Find For Rare Steering Committee and Professor of Medicine and Chief of the Division of Nephrology and Hypertension at the University Hospital of Würzburg, Germany. "Whilst we have expanded our knowledge into LSDs in the last decade, we still need to generate greater understanding into the factors affecting the disorders from pathogenesis to progression." "Find For Rare represents an opportunity to discover new thinking and ambition to meet our ultimate goal of improved patient care. The Steering Committee is eagerly awaiting the opportunity to review new ideas from across the globe and hopefully identify excellent candidates to receive the grants." Applications will be evaluated by the independent Steering Committee according to defined assessment criteria: strength of the proposed research project, innovation and novelty, relevance and impact, and potential for success. Depending on the number and quality of applications, based on the review by the Steering Committee, the total number of grants and total amount per project awarded may vary. However, the resources set aside by Chiesi Global Rare Diseases for this initiative amount to a total of €150,000, and researchers can apply for grants up to €50,000 per project. Although Chiesi Global Rare Diseases are organizing and funding the Research Grant Initiative, Chiesi will not in any way influence the application, evaluation, nor grant selection process. Proposals are welcome from all global regions other than the Americas. "Find For Rare is an important platform for the generation of new ideas and identification of future clinical leaders in the rare disease landscape. There is a societal obligation to address issues impacting the estimated 400 million living with a rare disease," said Enrico Piccinini, Head of Europe & International, Chiesi Global Rare Diseases. "Similar to all of our activities, the inspiration behind this initiative is the rare disease community. We pride ourselves on listening closely to their needs and delivering what will truly benefit patients – our real hope is this year's Find For Rare initiative will provide future solutions to today's challenging rare conditions." Further information regarding the initiative and the application process can be found at . The application process is simple and at no cost to the researcher. The application deadline is 31 October 2024, and the grants will be awarded in Q1 2025. About Find For Rare The Find For Rare Research Grant Initiative has been designed to support original research projects that advance knowledge in the fields of Fabry disease, alpha-mannosidosis, and cystinosis. The categories of research eligible for funding are projects aimed at improving understanding of the factors affecting diseases, from pathogenesis to progression, and studies of patient tailored clinical management. Research grants will be provided to organizations operating in the health or scientific research sector. Proposals are welcome from all global regions other than the Americas, with application documents required to be in English. For more information and to apply, visit . The application process is simple and free of charge. Simply complete the application form on the website and submit it with your CV and cover letter to [email protected]. The application deadline is 31 October 2024, and the grants will be awarded in Q1 2025. About lysosomal storage disorders LSDs are inborn errors of metabolism that are characterized by an abnormal build-up of substances in the body's cells as a result of enzyme deficiencies.1 The build-up of these substances can affect different parts of the body, including the skeleton, central nervous system (brain), lungs, heart, and eyes. Whilst there has been progress in clinical knowledge, more research in LSDs can be beneficial.1 About Chiesi Global Rare Diseases Chiesi Global Rare Diseases is a business unit of the Chiesi Group, established to deliver innovative therapies and solutions for people living with rare diseases. As a family business, Chiesi Group strives to create a world where it is common to have a therapy for all diseases and acts as a force for good, for society and the planet. The goal of the Global Rare Diseases unit is to ensure equal access to therapy so as many people as possible can experience their most fulfilling life. The unit collaborates with the rare disease community around the globe to bring a voice to underserved people in the healthcare system. About Chiesi Group Chiesi is a research-oriented, international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company's mission is to improve people's quality of life and act responsibly towards both the community and the environment. By changing its legal status to a Benefit Corporation in Italy, the US, and France, Chiesi's commitment to create shared value for society as a whole is legally binding and central to company-wide decision-making. As a certified B Corp since 2019, we're part of a global community of businesses that meet high standards of social and environmental impact. The company aims to reach Net-Zero greenhouse gases (GHG) emissions by 2035. With over 85 years of experience, Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,000 employees. The Group's research and development centre in Parma works alongside 6 other important R&D hubs in France, the US, Canada, China, the UK, and Sweden. Media Contact Chiara Travagin Rare Communication Manager, Chiesi Global Rare Diseases Tel: +39 348 8818985 Email: [email protected] Nathan McGlone Associate Director and Media Lead, Aurora Healthcare Communications Tel: +44 7850 775 669 Email: [email protected] References 1 NCBI. Lysosomal Storage Disease. Available at: (LSDs)%20are,to%20great%20morbidity%20and%20mortality. UK-CHI-2400703 | September 2024 Logo -

Soluble guanylate cyclase (sGC) activator is being studied in patients with chronic kidney disease (CKD), with potential implications for cardiovascular disease treatment CKD is a progressive condition affecting more than 10% of the global population, totaling approximately 850 million individuals Phase II study aims to address the unmet medical needs of chronic kidney disease patients and has potential to improve outcomes in related cardiovascular conditions through innovative sGC modulation BERLIN, Germany I August 22, 2024 I Bayer announced today the start of the ALPINE-1 study, a Phase II clinical trial with BAY3283142, an investigational soluble guanylate cyclase (sGC) activator, in patients with chronic kidney disease (CKD). The results of this study have the potential to provide evidence of the sGC activator as a viable treatment option for cardiovascular diseases. CKD is a progressive condition that affects more than 10% of the global population, totaling approximately 850 million individuals. It is a common and potentially deadly condition that is widely underrecognized. It progresses silently and unpredictably, with many symptoms not appearing until the disease is well-advanced. CKD is one of the most frequent complications arising from diabetes and is also an independent risk factor of cardiovascular disease. “This study marks a significant step forward in our commitment to addressing the high unmet medical need in this patient population and advancing our innovative approach to treat renal disease,” said Dr. Christian Rommel, Head of Research and Development at Bayer’s Pharmaceuticals Division. “In addition, this further strengthens our overall development portfolio. We are dedicated to advancing the development of BAY3283142 to improve outcomes for patients with chronic kidney disease and related cardiovascular conditions.” “The change in demography tells us that we are living longer in the future, and people want to spend this time in good health, with healthy kidneys,” said Prof. Christoph Wanner, Senior Professor of Medicine in the Department of Clinical Studies and Epidemiology, University Hospital of Würzburg and Co-Chair of the ALPINE-1 study. “The prevalence of chronic kidney disease (CKD) as one of the top 10 leading causes of mortality underscores the pressing need for effective treatment options. The evaluation of the sGC activator in patients with CKD holds promise in addressing a critical unmet medical need in patient care.” Following the successful first-in-human trials, the goal of the randomized, double-blind, placebo-controlled, multi-center Phase II ALPINE-1 study is to assess the efficacy and safety of BAY3283142 in patients with chronic kidney disease. BAY3283142 is a soluble guanylate cyclase (sGC) activator that represents an innovative approach to modulating the nitric oxide (NO)-soluble guanylate cyclase (sGC)-cyclic guanosine monophosphate (cGMP) pathway with a broad treatment potential in cardiovascular diseases. About Bayer’s Commitment in Cardiovascular and Kidney Diseases Bayer is an innovation leader in the area of cardiovascular diseases, with a long-standing commitment to delivering science for a better life by advancing a portfolio of innovative treatments. The heart and the kidneys are closely linked in health and disease, and Bayer is working in a wide range of therapeutic areas on new treatment approaches for cardiovascular and kidney diseases with high unmet medical needs. Together, this reflects the company’s approach to research, which prioritizes targets and pathways with the potential to impact the way that cardiovascular diseases are treated. About Bayer Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com. Find more information at https://pharma.bayer.com/ Follow us on Facebook: http://www.facebook.com/bayer SOURCE: Bayer

Multiple myeloma is one of the most common forms of cancer of the immune cells in the bone marrow. It is considered incurable. Even when patients respond to treatment at first, the cancer comes back. To be able to intervene faster and on a more targeted basis, researchers completed a comprehensive study of this disease at the molecular level. Multiple myeloma is one of the most common forms of cancer of the immune cells in the bone marrow. It is considered incurable. Even when patients respond to treatment at first, the cancer comes back. To be able to intervene faster and on a more targeted basis, researchers at Charité -- Universitätsmedizin Berlin, the Berlin Institute of Health at Charité (BIH), and the Max Delbrück Center teamed up with other partners for a comprehensive study of this disease at the molecular level. The team now describes how highly aggressive types of tumors can be detected early on in an article published in the journal Nature Cancer.* They show how changes in genetic material affect the protein pro the tumor cells, and thus the mechanisms involved in the disease. Multiple myeloma is a form of cancer in which the immune cells in the bone marrow, known as plasma cells, mutate and become cancerous. Plasma cells are responsible for producing antibodies. All humans have many different kinds of plasma cells that form large numbers of different antibodies. This allows the body to recognize and fight various pathogens. In multiple myeloma, a single plasma cell mutates into a tumor cell. That cell reproduces unchecked, forming a monoclonal cell population. This means many cells are formed, all of them exactly the same and genetically identical at first. The mutated cells often also produce large volumes of antibodies or fragments of them -- but they do not function properly. Over the course of the disease, most patients develop tumors at various locations in the bone marrow, hence the "multiple" in the disease's name. Immunodeficiency, kidney failure, bone loss, and bone fractures are just some of the consequences of this uncontrolled cell growth. Despite advances in treatment and the introduction of new gene and cell therapies, there is no cure for multiple myeloma at present. With this issue in mind, a team of researchers led by Jan Krönke from the Department of Hematology, Oncology and Cancer Immunology at Charité and Dr. Philipp Mertins, head of the Proteomics technology platform of the Max Delbrück Center and BIH, set out in search of new approaches to diagnosis and treatment. What path does the tumor take? No two cases of cancer are alike, and multiple myeloma is no exception. Tumors develop differently in different individuals, including at different rates. This makes it more difficult to predict how the disease will progress and choose the optimum treatment. While the mutated plasma cells do not spread much in some cases, in others they are extremely aggressive, leading to a poor prognosis. But what causes so much divergence in the course of multiple myeloma? In cooperation with protein analysis experts from the Max Delbrück Center and BIH, the researchers conducted a detailed study of genetic and molecular changes occurring in the tumor cells in a group of more than one hundred patients. The study included data from patients in the German Multiple Myeloma Study Group (DSMM), which is coordinated by the University Hospital of Würzburg. This allowed the researchers to include clinical data on patients who had received standardized treatment over a period of eight years or more following initial diagnosis. Systems medicine and big data While changes in the genome and their effects on the proteome are already well described for other types of cancer, this is the first detailed proteo-genomic study of multiple myeloma. "Genetic data alone is insufficient to explain the mechanisms involved in this disease," Mertins says. "We wanted to know the consequences of genetic changes at the protein level and compare this molecular biology data against the actual course of the disease in patients." The team was supported in collecting and analyzing the large volumes of data by experts at Charité, BIH, and the German Cancer Consortium (DKTK). Cutting-edge mass spectrometry methods made it possible to map the protein pro mutated plasma cells and compare it against that of healthy plasma cells in people without the disease. The researchers found that both genetic changes and changes in signaling pathways lead to uncontrolled activation of cancer cells. Regulatory processes at the protein level had the stronger influence. The researchers identified a protein constellation that suggests the disease will take a particularly aggressive course, regardless of other known risk factors. Unlocking new therapies "Our findings will help subcategorize patients more effectively going forward, personalizing their treatment," Krönke concludes. "We've identified key proteins and signaling pathways that can serve as the basis for even more effective, better tolerated treatments for multiple myeloma, for example for immune therapies such as CAR T-cell therapy." In further steps, the researchers plan to study which of the target structures they have identified are in fact good candidates for new therapeutic approaches. The study is a crucial resource for research and applied development, says Dr. Evelyn Ramberger, first author of the study: "To make the complex data set manageable, we programmed an interactive, freely available online tool." This has given cancer researchers easy access to the results, so they can use the information to develop new therapies and tests to help guide treatment. For example, it may be possible to treat patients with an especially aggressive form of multiple myeloma with more intensive therapies right at the outset. Mass spectrometry Mass spectrometry is a technique for analyzing the mass of molecules and atoms. The substance to be analyzed is ionized and converted to a gas phase. The ions formed are sharply accelerated using an electric field and then sorted by mass-to-charge ratio in the mass spectrometer's analytical unit. The mass spectrum of a substance provides information on its molecular composition. Mass spectrometry is therefore useful for identifying, characterizing, and quantifying a large number of biomolecules, such as proteins, metabolites, sugars, and fats, which may behave differently depending on the disease and the individual organism.