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< Back 23rd March, 2026 Lisbon, Portugal BIO-Europe Spring Europe’s premier springtime partnering event for the life sciences. BIO-Europe Spring is Europe’s largest springtime biotech partnering conference, uniting thousands of leaders from biotech, pharmaceutical, academic, and investment communities in strategic collaboration and deal-making. Over multiple days, the event facilitates thousands of one-to-one partnering meetings, expert panel discussions, innovative company presentations, and dynamic networking opportunities designed to accelerate licensing, business development, and cross-sector partnerships. With both in-person and digital components, BIO-Europe Spring empowers attendees to build global connections and drive innovation across the life sciences ecosystem. Previous Register now Next

VYNE Therapeutics has paused its Phase 1b trial of VYN202 for psoriasis after the FDA imposed a clinical hold due to testicular toxicity observed in non-clinical studies. < Back VYNE Therapeutics Faces Clinical Hold on VYN202 Program Following Toxicity Findings VYNE Therapeutics has paused its Phase 1b trial of VYN202 for psoriasis after the FDA imposed a clinical hold due to testicular toxicity observed in non-clinical studies. VYNE Therapeutics Inc. (Nasdaq: VYNE) has announced a temporary halt in its Phase 1b clinical trial of VYN202, a novel treatment for moderate-to-severe plaque psoriasis, following a clinical hold imposed by the U.S. Food and Drug Administration (FDA). The FDA’s decision came after the company observed testicular toxicity in dogs during a non-clinical toxicology study of VYN202. As a result, VYNE has suspended all screening, patient enrollment, and dosing in the ongoing Phase 1b trial. The company, however, emphasized that no serious adverse events have been reported in the patients already enrolled in the study. FDA Clinical Hold: A Step Toward Resolution VYNE has expressed its commitment to working closely with the FDA to resolve the clinical hold swiftly. "While we are disappointed by this unexpected development, the safety and well-being of patients in our studies is our top priority,” said David Domzalski, President and CEO of VYNE Therapeutics. "We intend to work closely with the FDA to address the clinical hold as expeditiously as possible and we plan to provide additional updates pending continued engagement with the FDA.” Despite the setback with VYN202, the company emphasized that the clinical hold does not affect its ongoing Phase 2b trial of repibresib gel, an entirely different compound designed for the treatment of nonsegmental vitiligo. VYNE is still on track to release top-line results from the 24-week, double-blind, vehicle-controlled portion of the Phase 2b study in mid-2025. About VYNE Therapeutics VYNE Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing differentiated therapies aimed at treating chronic inflammatory and immune-mediated conditions with significant unmet medical needs. The company's proprietary BET inhibitors, part of its InhiBET™ platform, are designed to overcome limitations seen in early-generation BET inhibitors, offering enhanced selectivity and alternative administration routes. Investors and stakeholders can follow VYNE’s progress and updates through its website and official filings, as the company continues to navigate its clinical trials and regulatory challenges. Author BioFocus Newsroom Previous Next

The pharma giant will close its cell therapy unit, cutting 250 jobs and ending work on Type 1 diabetes and Parkinson's programs, as new CEO shifts focus to obesity, diabetes, and liver disease treatments. < Back Novo Nordisk Pulls Plug on Cell Therapy in $1.3B Restructuring Push The pharma giant will close its cell therapy unit, cutting 250 jobs and ending work on Type 1 diabetes and Parkinson's programs, as new CEO shifts focus to obesity, diabetes, and liver disease treatments. In a major shift under new CEO Maziar Mike Doustdar, Novo Nordisk has announced the complete closure of its cell therapy operations, a move that will eliminate nearly all 250 positions within the division and mark the end of its long-running efforts to develop regenerative treatments for Type 1 diabetes, Parkinson’s disease, and heart failure. A company spokesperson confirmed that “we have decided to discontinue our cell therapy R&D efforts,” adding that Novo Nordisk is “in the process of identifying partners with the right capabilities and manufacturing capacity to further develop our innovations.” “Out of respect for the employees involved, we will not share additional details about individual sites or areas,” the spokesperson said. The decision, first reported by Danish outlet Børsen , comes as part of Doustdar’s broader restructuring campaign to streamline operations and redirect investment into Novo’s highest-growth areas - particularly obesity and diabetes, where demand for GLP-1 drugs such as Wegovy and Ozempic continues to surge globally. The plan is expected to generate annual savings of roughly $1.3 billion by the end of 2026 through a global headcount reduction of about 9,000 employees, or 11% of the total workforce. “As part of this change, we are assessing all business areas and regions to simplify structures, reduce duplication and sharpen focus,” the spokesperson said. The discontinuation marks a symbolic end to Novo’s decade-long ambitions in cell therapy, once a promising avenue for achieving functional cures for chronic diseases. Novo had invested heavily in stem cell–based programs targeting insulin-producing beta cell replacement for Type 1 diabetes, alongside exploratory work in Parkinson’s disease and chronic heart failure. But recent recalibration appears to prioritise near-term commercial impact over longer-horizon regenerative bets. Less than two weeks ago, Novo terminated a $598 million cell therapy collaboration in cardiovascular disease, signalling that the retreat from the modality was already underway. For observers, the exit underscores the mounting financial and manufacturing challenges that have constrained the scalability of cell therapies beyond oncology. Takeda Pharmaceuticals announced a similar withdrawal from cell therapy R&D just last week, citing similar operational hurdles. Novo’s retrenchment in cell therapy coincides with an aggressive expansion into other high-value therapeutic areas. The company revealed plans to acquire U.S.-based Akero Therapeutics for up to $5.2 billion, gaining control of its FGF21 analogue, a promising liver disease drug currently in phase 3 development. Data from the pivotal program are expected in the first half of 2026. Analysts view the Akero acquisition as a continuation of Novo’s strategy to consolidate its leadership in metabolic and endocrine disorders, leveraging its GLP-1 franchise to diversify into adjacent spaces such as NASH and broader cardiometabolic disease. For pharmaceutical professionals, the move represents a growing industry consensus: capital-intensive modalities like cell therapy may be taking a back seat to more commercially viable biologics and small molecules in the current funding climate. With the company’s weight-loss and diabetes drugs continuing to dominate global demand, and supply chains stretched to meet that demand, Novo's latest restructuring signals a clear focus on efficiency, profitability, and market leadership in metabolic health. Author BioFocus Newsroom Previous Next

< Back NHS Delivers First CAR-T Treatment for Adult B-cell ALL Patient Pioneering ‘living drug’ offers new hope for adults with aggressive leukaemia. The first patient to receive a pioneering form of CAR-T therapy for aggressive leukaemia on the NHS has described the experience as both “fantastic” and “very sci-fi”. Oscar Murphy, 28, from Bury, became the inaugural recipient of the treatment for B-cell acute lymphoblastic leukaemia (B-cell ALL) at Manchester Royal Infirmary on 2 January, when he began the first of two infusions of his genetically modified immune cells. The immunotherapy, often referred to as a “living drug”, is designed to recognise and kill cancerous cells and will now be funded across several NHS centres in England. Around 50 patients a year are expected to qualify, though clinicians believe the number could rise. Murphy was diagnosed with B-cell ALL in March 2025 and initially underwent chemotherapy followed by a donor stem cell transplant in July. His cancer relapsed just months later. “The leukaemia I've got is so fast-acting,” he said. “It needs an even quicker response to stop it. And we've now got an answer for that.” Clinical trial data underpinning the NHS rollout showed 77% of patients entered remission after treatment, with half displaying no detectable cancer three and a half years later. On average, patients gained an additional 15.6 months of life. According to Murphy’s haematologist, Dr Eleni Tholouli, the new iteration of CAR-T therapy is both safer and more effective than existing options. “Usually, this type of leukaemia is very aggressive and adult patients don't live beyond six to eight months. With this therapy, we are able to offer them years and potentially a cure. It's very significant and is revolutionising the way we tackle this cancer.” CAR-T therapy has been available on the NHS for several years for certain leukaemias and lymphomas, but this marks the first time adults with B-cell ALL have had access to the treatment. The breakthrough also comes amid ongoing conversations about who can access CAR-T therapies and when. In our earlier feature on Myeloma UK’s campaigning efforts, stakeholders warned that while the science is advancing quickly, equitable access across the NHS is yet to catch up. Murphy’s personalised therapy required T-cells to be extracted and sent to a manufacturing site in Stevenage, where they were engineered using a viral vector to introduce a genetic sequence that enables them to recognise malignant cells. Newly expressed surface receptors act like a lock-and-key system to identify the cancer, transforming the cells into chimeric antigen receptor T-cells, or CAR-T cells, which are expanded into the millions before being cryopreserved. The first infusion delivered around 100 million CAR-T cells contained in just a few teaspoons of fluid. A second infusion of 300 million cells followed. As a living therapy, the reprogrammed T-cells are expected to persist in the body, continue dividing and maintain anti-cancer activity over time. Murphy admitted he was astonished that such a small volume could deliver such a potent intervention. “It's very sci-fi, but if it means it gets rid of the cancer permanently and my own cells can do it it's just fantastic.” The treatment is manufactured by Autolus , a University College London spin-out . During earlier clinical studies, patient cells had to be shipped to laboratories in the US, underscoring the significance of onshore manufacturing capacity. The therapy carries a list price of £372,000 per infusion, though the NHS has negotiated confidential discounts. Eligible patients over 26 whose B-cell ALL has relapsed or failed to respond to treatment will be able to access the therapy at centres in Manchester, Cambridge, Newcastle, Sheffield, Plymouth and London. Patients from Wales and Northern Ireland will need to travel to England, and the treatment is not yet approved in Scotland. NHS England estimates that around 50 patients a year may benefit, but Tholouli told the BBC she believed more candidates will emerge, and predicted the therapy could ultimately replace stem cell transplantation as a first-line approach. Prof Peter Johnson, NHS National Clinical Director for Cancer, described the development as a “landmark moment” for people with aggressive blood cancers, adding that it was “remarkable” that a treatment conceived through UK research was now being delivered across the health service. “It will help more people like Oscar live longer and healthier lives.” The potential of the therapy is already evident in earlier patients. Chris Williams, 29, from Belfast, who was treated in Manchester while the therapy was still experimental, has now been in remission for nearly three years. “A few years ago I was very unwell and now I'm able to live a full life. I was able to go back to work. I also met Chloe and now we're engaged. I have fantastic support from my family and they are over the moon.” Murphy married his fiancée Lauren at Manchester Royal Infirmary last month, conscious of the uncertainties surrounding his condition, though the couple have planned a second ceremony for October. Looking ahead, his ambitions are disarmingly ordinary. “I want children and the white picket fence with my amazing wife, I just want that normality. This is my gateway to doing it and I can't wait.” Author BioFocus Newsroom Previous Next

< Back Lundbeck Reports Seizure Reductions in Rare Childhood-Onset Epilepsies AES 2025 data highlight continued seizure improvement in patients treated with bexicaserin through long-term follow-up. Lundbeck has announced new long-term Phase 2 follow-up data showing that patients with Developmental and Epileptic Encephalopathies (DEEs) who experienced early seizure reduction on bexicaserin maintained those improvements for up to two years. The results were presented at the 2025 American Epilepsy Society Annual Meeting in Atlanta. Bexicaserin is an investigational, centrally acting 5-HT2C receptor superagonist being developed for seizures associated with a broad range of DEEs, a group of rare and severe childhood-onset epilepsies marked by drug-resistant seizures, developmental impairment, and lifelong care needs. In the new data, patients who had completed the Phase 1b/2a PACIFIC trial, then participated in a 12-month open-label extension, continued treatment through expanded access for a total of up to two years. During this period, patients experienced a median reduction in countable motor seizures of 60.2 percent at roughly 18 months and 53.7 percent at around 24 months, with consistent results across diverse DEE subtypes. No new safety concerns were identified. Highlighting the impact on families, Johan Luthman, EVP and Head of Research and Development at Lundbeck, said: “The constant management of DEEs place a heavy emotional and financial burden on families, underscoring the urgent need for better seizure control. We are increasingly hopeful that bexicaserin can address this need. The data so far show durable seizure reductions, an encouraging safety profile and minimal risk of drug-drug interactions, reinforcing bexicaserin's potential as a first-in-class therapy across a broad range of DEEs.” DEEs represent some of the most challenging epilepsies to treat, with many patients resistant to available anti-seizure medications and few options spanning the full spectrum of DEE subtypes. The two-year findings suggest that patients who achieve an initial meaningful reduction may sustain that benefit long term, an important consideration in conditions where effective therapies are limited. The full results of the PACIFIC trial were recently published in Epilepsia , marking a milestone for DEE research and supporting the continued development of bexicaserin, which is currently being evaluated in a global Phase 3 program. Lundbeck presented seven additional scientific updates at AES 2025, emphasizing the company’s expanding commitment to rare childhood epilepsies and its broader neuroscience research portfolio. Bexicaserin remains investigational and is not yet approved by any regulatory authority. Author BioFocus Newsroom Previous Next

The pharmaceutical giant invests in AviadoBio, giving them an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). < Back Astellas Pharma Invests $50 million in AviadoBio to Bolster Gene Therapy Offering The pharmaceutical giant invests in AviadoBio, giving them an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). Astellas Pharma has invested $50 million in AviadoBio, giving the pharmaceutical giant an option to acquire the London-based biotech's gene therapy aimed at frontotemporal dementia (FTD). AviadoBio's innovative approach targets a specific protein deficiency associated with the disease, aiming to deliver long-term therapeutic benefits. Specifically, Astellas is interested in AviadoBio's single dose AAV-based gene therapy, AVB-101, currently in phase 1/2 clinical trials. The partnership also includes potential milestone payments if development progresses successfully, showcasing Astellas' commitment to gene therapy as a major focus for treating neurodegenerative diseases. The deal is part of Astellas’ broader strategy to expand its gene therapy pipeline. AviadoBio's CEO, Lisa Deschamps , commented “As we complete dosing of the first cohort of patients in our phase 1/2 ASPIRE-FTD trial of AVB-101, we are excited about the potential of this collaboration to help address the unmet need that exists today in frontotemporal dementia". Of the $50 million total sum being paid by Astellas Pharma, $30 million constitutes an upfront payment while the remaining $20 million comes in the form of an equity investment. In return, Astellas will be granted the option of being the therapy's exclusive global license holder. Astellas Pharma is a Japanese multinational pharmaceutical company focused on developing innovative therapies in various areas, including oncology, immunology, and gene therapy. It is heavily investing in cutting-edge medical technologies, such as gene therapies, to address complex diseases. AviadoBio , a London-based biotech, specializes in gene therapies for neurodegenerative diseases. The company is working on treatments that target specific genetic causes of these conditions, with a particular focus on frontotemporal dementia (FTD), aiming to deliver durable therapeutic benefits. Author BioFocus Newsroom Previous Next

< Back The Effect of Paternal Microbiome on Mice Offspring New study suggests a link between the paternal microbiome and offspring health. The Hackett group at the European Molecular Biology Laboratory (EMBL) have uncovered a link between paternal gut microbiota and offspring health. Results from their study show perturbations (slightly negative alterations) to paternal mice gut microbiota result in low birth weight, growth restriction and premature mortality in their offspring. The microbiota In your gut alone, there are trillions of bacteria, archaea, and eukarya. In fact, the number of these microorganisms in the body has been estimated to be approximately equal to the number of human cells (while some other estimates suggest ten times the number of microorganisms). A majority of this vast population exists in the gastrointestinal tract and is known as the gut microbiota. The gut microbiota are responsible for a range of internal functions, such as helping with digestion, protecting against harmful bacteria, and controlling your immune system. Studying gut microbiota offers insights into how these microbes influence digestion, immune function, and even mental health. Research has shown that an imbalanced gut microbiome is linked to various conditions, from obesity to depression. By understanding the gut microbiome, scientists can develop targeted therapies and probiotics to restore balance, improve health outcomes, and potentially prevent or treat a range of diseases. This burgeoning field underscores the intricate connection between our microbial inhabitants and overall well-being. Dysbiosis, having an altered gut microbiota, has also been associated with the development of inflammatory diseases and infections; previous studies have found that the maternal microbiome can have an effect on offspring health. However, the effect of paternal microbiome perturbations on the germline and mammalian offspring health is unclear. EMBL’s recent study published in Nature sheds some light on the link between the paternal environment and his offspring using mice. Epigenetic experiments Sperm carry information to the next generation through both genetic (DNA) and epigenetic (non-DNA sequence-based) material. Researchers have hypothesised that the epigenetic information carried on to offspring has the potential to be modified by the preconception environment, and therefore could influence offspring phenotype. Such epigenetic material can be affected by the gut microbiome. Given the modern diet and the plausible reduction in human gut microbiota diversity, richness, or abundance as a result of food modifications or a less varied diet, these potential changes to our microbiota may impact the health of future generations. By altering the gut bacterial composition of paternal mice, Jamie Hackett and colleagues at EMBL have explored the impact of the paternal gut microbiome on the health of successive generations, and so the effect of the germline on the next generation. Using antibiotics which reduce abundance, diversity and richness of mice gut microbiota, they found the offspring of these mice are smaller, less healthy, and show increased premature mortality compared to the offspring of mice whose gut microbiota has not been antibiotically ‘perturbed’. Results also showed that leptin, a hormone with a key role in energy homeostasis and reproduction, was especially ‘dysregulated’ as a result of nABX (non-absorbable antibiotics) medication. Perturbation to paternal leptin before conception—as a result of dysbiosis—has an intergenerational effect on offspring gene expression programmes. Fortunately, the study showed that restoration of gut microbiota (stopping antibiotic supplements and allowing 8 weeks recovery time) rescued emergent F1 (dominant) phenotypes—the effect of nABX-induced dysbiosis is reversible and treatable. Using IVF the Hackett group showed that these F1 phenotypes were transmitted specifically through paternal gametes and copurifying molecules—dysbiotic sperm donors produced offspring with similarly significantly reduced neonatal birth weight, postnatal growth and serious growth restriction. Comparing mice and men Human and mice microbiota are largely distinct: despite a 62% overlap of mouse microbiota genome and human gastrointestinal genomes at the genus, there is only 10% overlap at the species level. So, there are likely to be variations in the effect of reduced microbiota richness, abundance and diversity in humans compared to mice. This 90% discrepancy may mean drawing a link between the data observed in mice and applying it to human heritable epigenetic material is not applicable. The group plans to investigate the relevant inherited phenotypes further, and how these results may be applied beyond mice. However, this microbiota germline link may prove to be of interest in mitigating against unwanted pregnancy outcomes. Author Frances Briggs , freelance contributor Previous Next

We assess the intricate manufacturing landscapes of cell and gene therapies, highlighting the distinct capabilities, regulatory environments, and market dynamics of North America, Europe, the Middle East, Africa, and the Asia-Pacific region. < Back CGT Manufacturing: A Comparative Analysis of APAC, EMEA, and NA Markets We assess the intricate manufacturing landscapes of cell and gene therapies, highlighting the distinct capabilities, regulatory environments, and market dynamics of North America, Europe, the Middle East, Africa, and the Asia-Pacific region. Cell and gene therapy (CGT) represents one of the most advanced and rapidly evolving fields in medicine, promising cures for diseases that were previously considered untreatable. However, the manufacturing process behind these therapies is highly complex, involving advanced biotechnological tools, stringent regulatory oversight, and significant logistical coordination. As the demand for CGT grows globally, manufacturing capabilities in different regions have become an essential focus. Here we explore the cell and gene therapy manufacturing landscape across three key markets: Asia-Pacific (APAC), Europe, the Middle East, and Africa (EMEA), and North America (NA), outlining the regional differences in capabilities, regulatory landscapes, and market dynamics. The Manufacturing Landscape in North America (NA) North America, specifically the United States, is the global leader in cell and gene therapy development and manufacturing. Home to some of the largest biopharmaceutical companies (such as Bluebird Bio and Thermo Fisher Scientific) and academic institutions pioneering CGT research (such as UCLA’s Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell and Stanford University’s Center for Definitive and Curative Medicine), North America’s dominance stems from its strong innovation ecosystem and robust funding mechanisms. Manufacturing Infrastructure : North America benefits from a mature biopharmaceutical manufacturing industry, with state-of-the-art facilities capable of producing both autologous and allogeneic therapies. Companies such as Bluebird Bio and Kite Pharma (a Gilead company) are pioneers in producing CAR-T cell therapies, gene-modified cell therapies, and gene therapies. The region has also seen a surge in contract development and manufacturing organizations (CDMOs), supporting scaling up production to meet rising demand. Regulatory Environment : The U.S. Food and Drug Administration (FDA) has established clear regulatory pathways for CGT products, such as the Breakthrough Therapy designation and the Regenerative Medicine Advanced Therapy (RMAT) designation. These pathways expedite the development and review of CGT products, fostering innovation while maintaining safety and efficacy standards. Challenges : Despite North America’s lead, the region still faces challenges in scaling up manufacturing due to the complexity of CGT production. Ensuring consistent product quality, controlling costs, and managing the supply chain, particularly for autologous therapies, remain significant hurdles. Furthermore, skilled labor shortages and high operating costs create bottlenecks in manufacturing capacity expansion. The Manufacturing Landscape in Europe, the Middle East, and Africa (EMEA) The EMEA region, specifically Europe, has also established itself as a major hub for cell and gene therapy innovation, with countries such as the United Kingdom, Germany, and Belgium leading in manufacturing capabilities. However, the market dynamics and regulatory environment in the EMEA region differ significantly from those in North America. Manufacturing Infrastructure : While Europe houses world-class manufacturing facilities, the region has historically lagged behind North America in terms of production capacity. Nonetheless, recent years have seen significant investment in expanding CGT manufacturing in Europe. For example, companies such as Lonza and Oxford Biomedica have established advanced facilities focused on viral vector production and cell therapy manufacturing. The European market is also seeing increasing participation from CDMOs, which are key in scaling production for smaller biotech firms. Regulatory Environment : The European Medicines Agency (EMA) has its own distinct regulatory pathways for cell and gene therapies, such as the PRIME (PRIority MEdicines) scheme. The EMA’s regulatory framework is harmonized across the European Union, simplifying market access for CGT manufacturers. However, the complex national-level pricing and reimbursement systems across different EU member states can pose challenges for companies in navigating market access and achieving commercial success. Challenges : One of the primary challenges in the EMEA region is the fragmented nature of the market. While there is regulatory harmonization, there are still discrepancies in national healthcare systems, pricing, and reimbursement policies. Moreover, Europe faces a similar issue as North America in terms of scaling up manufacturing, particularly with respect to maintaining cost efficiencies in a highly regulated environment. The Manufacturing Landscape in Asia-Pacific (APAC) The APAC region, particularly China, Japan, and South Korea, is emerging as a key player in the global CGT market. The region’s growing biotech sector, increasing government support, and large patient population make it a strategic market for cell and gene therapy development and manufacturing. Manufacturing Infrastructure : While APAC's CGT manufacturing infrastructure is still developing, it is rapidly expanding. Countries such as China and Japan have made significant strides in building advanced manufacturing capabilities. China, in particular, has seen a boom in the construction of CGT manufacturing facilities, with both domestic companies like WuXi AppTec and foreign companies expanding their presence in the region. Japan, with its focus on regenerative medicine, has also developed specialized manufacturing hubs, supported by initiatives such as the Japanese Regenerative Medicine Promotion Act. Regulatory Environment : One of the distinctive features of the APAC market is its relatively fast regulatory approvals for CGT products. China’s National Medical Products Administration (NMPA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) have implemented expedited regulatory pathways for regenerative medicine products. In Japan, for example, the “conditional time-limited approval” system allows early market access for promising therapies with provisional approval based on limited clinical data. Challenges : Despite its rapid growth, the APAC region faces several challenges in CGT manufacturing. A lack of standardized regulations across the region creates difficulties for multinational companies seeking to enter multiple APAC markets. Additionally, the high cost of manufacturing and ensuring supply chain integrity remain significant issues. While China and Japan have made considerable strides, other APAC countries still face infrastructure gaps in terms of manufacturing capacity and expertise. Key Differences and Comparative Insights Regulatory Frameworks : The regulatory landscape is one of the most distinct differences between these regions. While North America has a well-defined and streamlined regulatory process, EMEA’s market is more fragmented (despite efforts aimed at harmonization). In contrast, APAC has shown remarkable flexibility in expediting approvals, though regulatory standards vary widely across countries, making cross-border commercial strategies complex. Manufacturing Capacity and Expertise : North America leads in terms of established manufacturing infrastructure and expertise. However, Europe is quickly catching up, particularly with the increase in CDMO activities. APAC, while growing rapidly, still faces a gap in manufacturing infrastructure, especially outside of major markets like China and Japan. Market Dynamics : North America remains the largest market for CGT products, driven by a strong investment landscape and extensive healthcare reimbursement systems. Europe, while advanced in scientific innovation, struggles with market access due to complex pricing and reimbursement processes. The APAC region, with its large patient population and increasing government support, offers significant growth potential but remains fragmented in terms of market access and regulatory consistency. Market leaders Based on 2023 end of year figures, the top cell and gene therapy companies as judged by single therapy revenue is as follows: 1. Kite, a Gilead Company (USA) Yescarta (axicabtagene ciloleucel) is a CAR-T cell therapy developed by Kite Pharma, a subsidiary of Gilead Sciences. It was one of the first CAR-T therapies to gain approval and represents a significant advancement in cancer treatment, specifically for certain types of blood cancers. 2023 revenue: $1.5 billion 2. Novartis (Switzerland) Zolgensma (onasemnogene abeparvovec) is a groundbreaking gene therapy developed by Novartis for the treatment of spinal muscular atrophy (SMA), a rare genetic disorder that affects motor neurons, leading to muscle weakness and loss of movement. It is the first and only gene therapy approved to treat this condition. 2023 revenue: $1.2 billion 3. Novartis (Switzerland) Kymriah (tisagenlecleucel) is a pioneering CAR-T cell therapy developed by Novartis for the treatment of certain blood cancers. It was the first CAR-T therapy to receive FDA approval and has since been a landmark in the field of personalized cancer treatments. 2023 revenue: $508 million 4. Janssen Biotech, Johnson & Johnson (USA), and Legend Biotech (USA) Carvykti (ciltacabtagene autoleucel) is a CAR-T cell therapy co-developed by Legend Biotech and Janssen Pharmaceuticals (a subsidiary of Johnson & Johnson) for the treatment of relapsed or refractory multiple myeloma. It is an innovative therapy that offers a personalized treatment approach for patients with advanced stages of this blood cancer. 2023 revenue: $500 million 5. Bristol Myers Squibb (USA) and 2seventy bio (USA) Abecma (idecabtagene vicleucel) is a CAR-T cell therapy developed by Bristol Myers Squibb and 2seventy bio for the treatment of relapsed or refractory multiple myeloma. It is the first FDA-approved CAR-T therapy specifically targeting this form of cancer, providing a new treatment option for patients who have exhausted other therapies. 2023 revenue: $472 million Conclusion Cell and gene therapy manufacturing is a complex and evolving field, with significant regional differences in terms of infrastructure, regulatory oversight, and market dynamics. While North America currently dominates CGT manufacturing, EMEA and APAC are quickly advancing, each with unique strengths and challenges. As the global CGT market continues to expand, manufacturers will need to navigate these regional distinctions to optimize production and market access strategies, ensuring that life-saving therapies reach patients worldwide. Author BioFocus Newsroom Previous Next

SPT Labtech and Semarion have partnered to create flexible, automated, and miniaturized workflows for advanced cell-based assays. < Back SPT Labtech and Semarion Join Forces to Advance Automated Cell-Based Assays SPT Labtech and Semarion have partnered to create flexible, automated, and miniaturized workflows for advanced cell-based assays. SPT Labtech, a global leader in laboratory automation, has announced a new collaboration with Semarion, a University of Cambridge spin-out focused on microcarrier technology for cell biology. The partnership will combine SPT Labtech’s firefly® liquid handling platform with Semarion’s SemaCyte® microcarriers to develop flexible, miniaturized, and automated workflows for cell-based assays. Cell-based assays play a critical role in drug discovery and biological research, but their automation and miniaturization have remained challenging. By integrating SPT Labtech’s adaptable liquid handling system with Semarion’s microcarrier technology, the two companies aim to simplify and speed up key steps in adherent cell assay development, including applications like high-content imaging and cell painting. Maryia Karpiyevich, Product Development Scientist at SPT Labtech, said, “This collaboration gives us the chance to bring together innovative microcarrier technology and flexible automation in one platform. Our goal is to provide scientists with better tools to advance cellular research.” Jeroen Verheyen, CEO and Co-Founder of Semarion, added, “Partnering with SPT Labtech allows us to combine our microcarrier technology with their automation expertise. This opens the door to new, streamlined workflows that are both miniaturized and scalable, helping researchers develop assays more efficiently.” Morten Frost, Chief Commercial Officer at SPT Labtech, commented, “We’re committed to supporting technologies that make lab work easier and more adaptable. This partnership reflects that commitment, offering solutions that can scale and adjust as research demands change.” The companies will work together on application protocols, proof-of-concept studies, and workflow optimization to deliver practical, automated solutions for cell-based research. Author BioFocus Newsroom Previous Next

< Back Strategic Partnership Between UK Government and Oxford Nanopore Set to Advance Genomics-Driven Healthcare Oxford Nanopore x UK Biobank x Genomics England x NHS England x UK Government: a landmark collaboration for UK healthcare In a bold move that aligns with the UK Government's vision of creating a healthcare system "fit for the future," a strategic partnership has been forged between Oxford Nanopore, UK Biobank, Genomics England, and NHS England. This collaboration marks a new chapter in biomedical research, leveraging cutting-edge genomic and epigenomic technologies to improve patient outcomes and bolster national biosecurity. The partnership aims to revolutionise healthcare, not only through the development of advanced diagnostic tools but also by enhancing the UK's pandemic preparedness, making it a global leader in genomic innovation. Advancing Patient Care through Genomic Research At the heart of this partnership lies the ambition to transform the diagnosis and treatment of diseases, particularly cancer, rare genetic disorders, and infectious diseases. Genomic insights, which involve the study of DNA to understand genetic diseases and predispositions, have already shown significant promise in personalizing treatments. The integration of Oxford Nanopore’s high-performance nanopore sequencing technology into clinical practice will enable faster, more affordable, and more accessible genomic sequencing, ensuring that healthcare providers can make more precise treatment decisions. Oxford Nanopore's technology excels in providing rich, high-resolution genomic data, capable of uncovering both genetic and epigenetic alterations. These insights are crucial in understanding how changes in DNA contribute to disease. For cancer, this means more accurate identification of mutations that can guide personalized treatment plans, potentially leading to earlier diagnoses and better survival rates. For rare genetic diseases, the ability to pinpoint even the smallest genetic mutations could drastically improve diagnostic accuracy, reducing diagnostic odysseys, and speeding up intervention. The partnership’s focus on pharmacogenomics, where genetic information is used to tailor medications to an individual’s genetic profile, has the potential to significantly improve patient safety and treatment efficacy. This would address a long-standing challenge in medicine, where trial-and-error approaches to prescribing medications often lead to adverse reactions and inefficiencies in care. Preparing for Future Pandemics: Real-Time Pathogen Surveillance Perhaps one of the most ambitious and timely aspects of the partnership is the establishment of a real-time, pathogen-agnostic biosurveillance system, which will span up to 30 NHS sites. This initiative is a response to the lessons learned during the COVID-19 pandemic, where the UK’s life sciences sector demonstrated the power of genomics in tracking viral evolution and identifying emerging threats. By creating a rapid pathogen identification system, this partnership aims to ensure that the NHS can respond quickly to future pandemics and other biological threats. The project will build on successful pilots, particularly in respiratory metagenomics, a program led by Guy's and St Thomas' NHS Foundation Trust. The integration of Oxford Nanopore’s sequencing technology into this system will allow for the rapid characterization of respiratory diseases, including identifying drug-resistant pathogens. By reducing the time it takes to diagnose such diseases from days to just six hours, this program has the potential to transform patient care, allowing for faster, more targeted treatments. This biosurveillance system will not only improve patient outcomes by ensuring timely diagnosis and treatment but also enhance the UK's biosecurity, aligning with the UK Biological Security Strategy . By providing real-time data to the UK Health Security Agency (UKHSA), this partnership aims to place UK scientists and decision-makers ahead of emerging infectious diseases, providing them with critical information to make informed decisions on public health measures and interventions. Strengthening the UK’s Life Sciences Sector Beyond the immediate healthcare benefits, the partnership represents a strategic investment in the UK's life sciences sector, which is poised to become a global leader in genomic and biotechnological innovation. The collaboration should help catalyze economic growth, supporting the development of high-value jobs and fostering a skilled workforce trained in genomics and personalized medicine. With access to cutting-edge technology and training, NHS staff and researchers will be better equipped to navigate the complexities of genomics in clinical practice. It is hoped the initiative will also create opportunities for collaboration between the private and public sectors, further positioning the UK as a biotechnology hub. By accelerating the adoption of innovative genomic technologies into the NHS, this partnership will bridge the gap between scientific discovery and its translation into real-world applications. Author BioFocus Newsroom Previous Next

< Back 31st May – 4th June, 2024 Chicago, IL 2024 ASCO Annual Meeting The ASCO Annual Meeting is one of the most significant global events for bringing together oncology professionals. This year, it is hosted in Chicago, Illinois & Online. Previous Register now Next

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