Editor’s note: Since this webinar, Waskyra has also received approval from the U.S. Food and Drug Administration (FDA), expanding access beyond Europe. One of the hardest challenges in cell & gene therapy (CGT) is determining how programs for ultra-rare diseases move forward when there’s no clear commercial path. Many promising efforts stop well before reaching […]
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Access to life-saving cell and gene therapies depends not only on scientific breakthroughs but also on the systems that bring those breakthroughs to patients. Regulations, infrastructure, and collaboration across borders determine whether a therapy moves from the lab to the bedside, or remains out of reach This urgent reality set the stage for the November […]
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The last decade has seen a revolution in the way cell and gene therapies are developed, delivered, and accessed. What began as pioneering work in bone marrow transplantation has evolved into a global ecosystem advancing engineered cell therapies that offer new hope to patients facing devastating diseases. This transformation—rooted in science, collaboration, and compassion—continues to […]
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Access to advanced therapies is often framed as a challenge of resources and infrastructure. But in Europe, a bold new model is reshaping how patients receive life-saving CAR T-cell therapy. Instead of relying solely on commercial manufacturing and centralized approval processes, the Hospital Clínic de Barcelona has pioneered a hospital-based pathway that delivers CAR-T directly […]
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Providing access to transformative cell and gene therapies is one of the most urgent challenges in modern healthcare. These treatments offer the possibility of curing conditions once thought untreatable—yet in the United States, only a fraction of eligible patients are receiving them. The barriers are complex: financial risk for payers, short-term budgeting in a long-term […]
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Access to life-saving cell and gene therapies is one of the most critical challenges facing healthcare today. While these therapies offer remarkable hope for patients battling life-threatening conditions like cancer and rare genetic diseases, the reality is that only a fraction of eligible patients are actually receiving them. The reasons are rarely scientific. More often, […]
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In the rapidly advancing world of cell and gene therapy, a single treatment can alter a patient’s life trajectory. But what happens after the initial success? For many, the journey is just beginning. Long-term follow-up (LTFU) is not just a regulatory requirement—it’s a vital, ethical commitment to patients who become pioneers of new treatments. Yet, […]
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Access to advanced therapies that can fundamentally alter disease trajectories is one of the most pressing frontiers in healthcare. Among them, cell and gene therapies offer unprecedented hope for millions, especially those grappling with rare, chronic, or previously untreatable conditions. Yet, realizing the full potential of these therapies requires more than scientific innovation—it demands cross-sector […]
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Across the globe, access to life-saving cell and gene therapies like CAR T-cell therapy remains largely limited to wealthier nations and well-resourced health systems. While the clinical outcomes of these therapies can be extraordinary—offering cures for certain blood cancers—their high cost and complexity of production present a barrier that many countries cannot overcome. But a […]
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Mesenchymal stem cells (MSCs) have long shown promise in regenerative medicine, with the potential to treat everything from heart disease to autoimmune conditions. But for decades, MSC therapies struggled to move beyond early-phase clinical trials and regional approvals. That changed in December 2024 when the U.S. Food and Drug Administration (FDA) approved RYONCIL—and it became […]
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