Beta-Thalassemia

2024 Harrington Prize for Innovation in Medicine Recognizes Pioneering Work in Immunology CLEVELAND, March 19, 2024 /PRNewswire/ -- The eleventh annual Harrington Prize for Innovation in Medicine has been awarded to Arlene H. Sharpe, MD, PhD, Kolokotrones University Professor and Chair of the Department of Immunology at Harvard Medical School. The award recognizes her breakthrough discoveries in immune regulation, which have established foundational principles in immunology and led to new cancer therapies that act by boosting the immune response to cancer. The Harrington Prize for Innovation in Medicine, established in 2014 by the Harrington Discovery Institute at University Hospitals and the American Society for Clinical Investigation (ASCI), honors physician-scientists who have moved science forward with achievements notable for innovation, creativity and potential for clinical application. Dr. Sharpe's research in immunology has led to significant advances in medical treatments. Her work helped identify key pathways that restrain the activity of T lymphocytes (immune cells) to fight cancer. Her work defined the PD-1 pathway and its immunoinhibitory functions as well as the inhibitory functions of CTLA-4 to restrain the activity of the immune system. Dr. Sharpe's research thus laid the foundation for the development of 'immune checkpoint inhibitors', drugs that target PD-1 and related pathways. These therapies, including pembrolizumab and nivolumab, have been approved by the FDA for treating numerous types of cancer. They work by preventing inhibition of T cell function, thereby unleashing the body's immune system to fight cancer cells more effectively. This approach has become a cornerstone in cancer treatment strategies. "Dr. Sharpe's body of work has shaped our understanding of the role of the T cell in regulating the immune response. Her innovative scientific discoveries are among the best examples of bench-to-bedside work in the past three decades," said Benjamin D. Humphreys, MD, PhD, Joseph Friedman Professor of Renal Diseases in Medicine and Chief, Division of Nephrology, Washington University in St. Louis and 2023-2024 President of the ASCI. "Immune checkpoint inhibitors are drugs that allow for dramatic remission, and even cures, of cancers that had previously been considered untreatable. Dr. Sharpe's foundational insights have thus been harnessed to transform cancer therapy. Her work provides a beautiful example of physician impact through creative science," said Jonathan S. Stamler, MD, President, Harrington Discovery Institute, Robert S. and Sylvia K. Reitman Family Foundation Distinguished Professor of Cardiovascular Innovation and Professor of Medicine and of Biochemistry at University Hospitals and Case Western Reserve University. A committee composed of members of the ASCI Council and the Harrington Discovery Institute Scientific Advisory Board reviewed nominations from leading academic medical centers globally before selecting the 2024 Harrington Prize recipient. In addition to sharing the Prize's $20,000 honorarium, Dr. Sharpe will deliver the Harrington Prize Lecture at the 2024 AAP/ASCI/APSA Joint Meeting on April 5, will be a featured speaker at the 2024 Harrington Scientific Symposium May 22-23, and will publish an essay in the Journal of Clinical Investigation. The Harrington Prize has recognized outstanding and diverse innovations in medicine: 2014: Harry Dietz, MD, Johns Hopkins University, for his contributions to the understanding of the biology and treatment of Marfan syndrome, a disorder leading to deadly aneurysms in children and adults. 2015: Douglas R. Lowy, MD, The National Cancer Institute, in recognition of his discoveries that led to the development of the Human Papillomavirus vaccine to prevent cervical cancer. 2016: Jeffrey M. Friedman, MD, PhD, The Rockefeller University, for his discovery of leptin, which controls feeding behavior and is used to treat related clinical disorders. 2017: Jointly awarded to Daniel J. Drucker, MD, Mount Sinai Hospital, Canada, Joel F. Habener, MD, Massachusetts General Hospital, and Jens J. Holst, MD, DMSc, University of Copenhagen, Denmark, for their discovery of incretin hormones and for the translation of these findings into transformative therapies for major metabolic diseases such as diabetes. 2018: Helen H. Hobbs, MD, UT Southwestern Medical Center, for the discovery of the link between a gene mutation (PCSK9) and lower levels of LDL, which has improved the treatment of high cholesterol. 2019: Carl H. June, MD, University of Pennsylvania, for advancing the clinical application of CAR T therapy for cancer treatment, and for his sustained contributions to the field of cellular immunology. 2020: Stuart H. Orkin, MD, Harvard University, for breakthrough discoveries on red blood cells that offer new treatments for patients with sickle cell disease and beta-thalassemia, which are among the most common genetic disorders. 2021: Warren J. Leonard, MD, and John J. O'Shea, MD, NIH, for their respective contributions to the field of immunology, from fundamental discovery to therapeutic impact. 2022: James E. Crowe Jr., MD, and Michel C. Nussenzweig, MD, PhD, for their groundbreaking work, which has elucidated fundamental principles of the human immune response and enabled the use of human antibodies to treat COVID-19. 2023: Jean Bennett, MD, PhD, and Albert M. Maguire, MD, for their groundbreaking translational research to restore sight in inherited genetic diseases. SOURCE Harrington Discovery Institute

With the loan, bluebird said its cash runway now extends to the first quarter of 2026. As bluebird bio works to make the most of three gene therapy launches, cash-flow concerns have been looming over the drugmaker.  Now, Hercules Capital has swooped in to offer a five-year loan—worth up to $175 million—that will significantly extend the company's runway. The first $75 million portion of the loan was drawn upon the deal closing, with two $25 million tranches available upon the achievement of certain commercial milestones, bluebird said in a Monday release.  A fourth $50 million pot “may be available at the sole discretion Hercules,” bluebird added. Excluding that tranche, the $125 million in loan funding should support the company's operations through the first quarter of 2026. “This financing underscores the value bluebird offers as a standalone gene therapy leader and meaningfully extends our runway, bolstering our ability to bring transformative treatments to patients and their families,” bluebird's chief financial officer, Chris Krawtschuk, said in the release. Bluebird in December scored FDA approval for its sickle cell disease (SCD) gene therapy Lyfgenia, albeit with a black-box warning. Bluebird set its wholesale acquisition cost at $3.1 million, about 40% higher than the price competitor Vertex put on its CRISPR Therapeutics-partnered SCD therapy Casgevy.  Still, bluebird CEO Andrew Obenshain has said there's much more to consider than just the starting price for the therapies. Bluebird has offered a “very innovative contract” for its drug that has been well received by payers, Obenshain said in January. Outside of Lyfgenia, bluebird also markets its one-time beta thalassemia treatment Zynteglo, which comes with a $2.8 million price tag. The company also sells cerebral adrenoleukodystrophy gene therapy Skysona, which is priced at $3 million. Those drugs won back-to-back approvals in 2022. Amid its regulatory advances, bluebird has sought new cash sources to support its commercial future. Other than the latest loan, the company has reached an agreement with Alterna Capital Solutions to hand over future revenues in exchange for upfront funding in a deal that covers a maximum credit of $100 million. It also announced in December that it was working with Goldman Sachs and J.P. Morgan Securities to sell $150 million in new shares.  Before all of those fundraising efforts, bluebird estimated that its cash and certain other assets could fund operations through the second quarter of 2024.

NEW YORK, March 18, 2024 /PRNewswire/ -- Technavio categorizes the global gene therapy market size as estimated to increase by USD 4,613.9 million between 2022 and 2027. The market's growth momentum will be progressing at a CAGR of 19.91% during the forecast period. During the forecast period, North America is anticipated to drive 39% of global market growth, as per Technavio's analysts. The US, particularly, emerges as a promising investment hub within the region, supported by substantial funding from entities like the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA) to emerging players in the market. This influx of government and private sector investment is propelling the development of innovative gene therapies, with 18 clinical trials and 17 biotech startups underway. Such financial backing is poised to significantly stimulate market demand in the coming years. Continue Reading Technavio has announced its latest market research report titled Global Gene Therapy Market 2023-2027 Discover some insights on market size historic data (2017 to 2021) and Forecast (2023 to 2027) before buying the full report - Request a sample report Technavio has segmented the market based on therapy areas (oncology, CNS, ophthalmology, rare diseases, and others), and delivery mode (in vivo and ex vivo). The market growth in the oncology segment will be significant during the forecast period. The global oncology drugs market has a huge unmet demand for the treatment of various indications. Also, the increasing prevalence of cancer indications has led pharmaceutical companies and research institutions to conduct extensive research to develop drugs for these indications. The lack of efficacy of small molecules and biomolecules has led companies to move towards effective therapeutic options such as gene therapy. Such factors will increase the segment growth during the forecast period. For insights on global, regional, and country-level parameters with growth opportunities from 2017 to 2027 - Download a Sample Report Key Companies The global gene therapy market is fragmented, with the presence of several global as well as regional vendors. A few prominent vendors that offer gene therapy in the market are Abeona Therapeutics Inc., Adaptimmune Therapeutics plc, Adverum Biotechnologies Inc., Amgen Inc., Astellas Pharma Inc., Biogen Inc., bluebird bio Inc., Editas Medicine Inc., ElevateBio, F. Hoffmann La Roche Ltd., Generation Bio Co., Gilead Sciences Inc., Novartis AG, Orchard Therapeutics Plc, Poseida Therapeutics Inc., Sangamo Therapeutics Inc., Sibiono GeneTech Co. Ltd., Syncona Ltd., uniQure NV, and Voyager Therapeutics Inc. and others. Abeona Therapeutics Inc.: The company offers gene therapy solutions such as Phase 3 VIITAL Clinical Trial of EB 101. Amgen Inc.: The company offers gene therapy solutions such as CAR T cell immunotherapies. Astellas Pharma Inc.: The company offers gene therapy solutions such as AT132, AT845, AT466. Biogen Inc.: The company offers gene therapy solutions such as GTxAU. The growth of the market is driven by the increase in special drug designations. Most gene therapy programs have been granted orphan drug designation, fast track designation, or breakthrough designation by the U.S. FDA, EMA, and other regulatory bodies for the treatment of certain diseases. In addition, orphan drug status will also help the company with tax incentives in its next research. Such advantages of specialty drug designations will drive the growth of the market during the forecast period. Growing research in gene therapy for CVDs and orphan diseases is one of the key market trends that is contributing to the market growth. High treatment cost is one of the factors hindering the gene therapy market growth. Driver, Trend & Challenges are the factor of market dynamics which states about consequences & sustainability of the businesses, find some insights from a sample report! Analyst Review Gene therapy stands at the forefront of medical innovation, offering promising solutions for a myriad of genetic disorders ranging from atrophy to muscular dystrophy. As the world grapples with health challenges like the COVID-19 pandemic, companies like Applied Genetic Technologies Corporation and Benitec Biopharma in countries like Australia and Belgium are actively engaged in pioneering gene therapy research to combat such conditions. Beta thalassemia, sickle cell disease, and spinal muscular atrophy represent just a fraction of the genetic issues that gene therapy aims to address. With the advent of breakthrough treatments like betibeglogene autotemcel and onasemnogene abeparvovec, patients suffering from these debilitating conditions now have newfound hope. The gene therapy market, bolstered by advancements in CRISPR Therapeutics and lentivirus technology, is witnessing exponential growth. As companies like Pfizer, Cellectis, and Novartis Gene Therapies invest in research and development, the market is projected to expand further, especially in regions like California and Massachusetts, known for their biotechnology hubs. However, this growth isn't without challenges. Regulatory bodies like ClinicalTrials.gov, the FDA, and the International Organization for Standardization (ISO 9000) play pivotal roles in ensuring the safety and efficacy of gene therapies. Striking a balance between innovation and compliance with regulations such as the California Consumer Privacy Act and the General Data Protection Regulation remains paramount. One of the critical factors driving the gene therapy market is the increasing prevalence of genetic disorders worldwide. From hereditary illnesses to congenital diseases, the demand for innovative treatments continues to rise. Moreover, with advancements in gene editing and therapeutic gene delivery systems, the potential to target specific cells and protein molecules offers new avenues for treatment. Venture capital financing and partnerships between academia and industry players further fuel research efforts. Companies like Lonza Group and Oxford Biomedica contribute to the manufacturing and logistics aspects of gene therapy, ensuring efficient drug delivery and scalability. In conclusion, the gene therapy market holds immense promise in addressing genetic disorders and improving patient outcomes. As the field continues to evolve, collaboration, adherence to regulations, and a focus on patient-centric approaches will be essential in unlocking the full potential of gene therapy to tackle the most challenging medical conditions of our time. Download a Sample Report Market Overview The Gene Therapy Market is witnessing exponential growth globally, with companies like Applied Genetic Technologies Corporation and Benitec Biopharma leading the charge. This revolutionary approach offers hope for conditions such as atrophy and beta thalassemia, providing targeted solutions to genetic disorders. Recent advancements include Betibeglogene autotemcel, a promising treatment for beta-thalassemia pioneered by CRISPR Therapeutics. Biomedicine is at the forefront of this transformative field, driving innovation and clinical trials. Amidst the COVID-19 pandemic, gene therapy research continues unabated, with countries like Australia and Belgium actively contributing to the global effort. As the market expands, gene therapy emerges as a beacon of hope for patients worldwide, offering personalized treatments and potential cures. Download a Sample Report Related Reports: The cell and gene therapy market size is expected to increase by USD 9.97 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 20.15%. The cell therapy market size is expected to increase by USD 21.061 billion from 2021 to 2026, and the market's growth momentum will accelerate at a CAGR of 56.79%. TOC Executive Summary Market Landscape Market Sizing Historic Market Size Five Forces Analysis Market Segmentation by Therapy Areas market Segmentation by Delivery Mode Customer Landscape Geographic Landscape Drivers, Challenges, and Trends Company Landscape Company Analysis Appendix About US Technavio is a leading global technology research and advisory company. Their research and analysis focuses on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios. Contact Technavio Research Jesse Maida Media & Marketing Executive US: +1 844 364 1100 UK: +44 203 893 3200 Email: [email protected] Website: SOURCE Technavio