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- Lundbeck Reports Seizure Reductions in Rare Childhood-Onset Epilepsies | BioFocus
< 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
- Sartorius and McMaster University Open Biomanufacturing Lab in Canada
State-of-the-art facility with advanced bioprocessing technology aims to accelerate therapeutic development, improve efficiency, and train the next generation of biomanufacturing talent for Canada’s growing life sciences sector. < Back Sartorius and McMaster University Open Biomanufacturing Lab in Canada State-of-the-art facility with advanced bioprocessing technology aims to accelerate therapeutic development, improve efficiency, and train the next generation of biomanufacturing talent for Canada’s growing life sciences sector. Sartorius and McMaster University have expanded their partnership by inaugurating a state-of-the-art bioprocessing automation laboratory in Hamilton, Ontario. Located within McMaster's Faculty of Engineering, the 1,600-square-foot facility is equipped with advanced biomanufacturing equipment, much of which is supplied by Sartorius. The lab will serve as a training and development hub for students, Sartorius employees, and industry partners, focusing on the advancement of bioprocess modeling, simulation, and control. This initiative builds upon a collaboration that began in 2019, aiming to optimize the manufacturing processes of antibody and virus-based therapeutics for diseases such as COVID-19, cancers, and genetic disorders. The partnership has also provided valuable training opportunities for students, fostering the next generation of talent in Canada's biomanufacturing sector. The establishment of the lab was made possible through a grant from the Biosciences Research Infrastructure Fund (BRIF), a Canadian government initiative designed to enhance the nation's biomanufacturing and life sciences capabilities. Notably, this is the first facility fully funded by BRIF to open in Canada. Dr. Heather Sheardown, Dean of the Faculty of Engineering at McMaster University, emphasized the lab's significance: "The opening of the Sartorius Bioprocess Automation Lab marks a milestone in McMaster’s commitment to advancing biomanufacturing capabilities in Canada." She highlighted that the facility will support technological innovations in large-scale biotherapeutics manufacturing, enhancing production efficiency and expanding access to life-saving treatments for chronic illnesses such as autoimmune disorders and cancers. Prof. Dr. Oscar-Werner Reif, Chief Technology Officer of Sartorius, added, "This partnership enables McMaster University and Sartorius to explore and industrialize innovative bioprocessing solutions together with partners from the biopharmaceutical industry." He noted that young researchers from academia and industry will jointly develop innovative modeling and predictive control solutions in the facility, ultimately driving faster development of improved therapies accessible to patients worldwide. Further strengthening this collaboration, a team of McMaster researchers has secured additional Alliance Grant funding from the Natural Sciences and Engineering Research Council of Canada (NSERC) to launch an extensive four-year project with Sartorius. This partnership underscores a shared commitment to advancing biomanufacturing processes and training the next generation of innovators in the field. Author BioFocus Newsroom Previous Next
- Fibromyalgia Research: A New Focus | BioFocus
< Back Fibromyalgia Research: A New Focus Fibromyalgia research has long failed to uncover a cause to the mysterious yet debilitating condition. A breakthrough study shows antibodies may be crucial. Introduction to Fibromyalgia Research Fibromyalgia research has long failed to uncover a cause of the mysterious yet debilitating condition. A breakthrough study shows antibodies may be crucial. Fibromyalgia syndrome (FMS) is a complex and debilitating condition that affects millions of people worldwide. Characterized by widespread pain, tenderness, and a range of other symptoms, FMS has long remained a medical enigma with fibromyalgia research efforts failing to determine a definitive cure. A groundbreaking study by A. Goebel and E. Krock et al. from the Walton Center NHS Foundation Trust in Liverpool, UK, reveals a significant breakthrough in understanding the pathophysiology of FMS. The findings not only uncover more about the underlying mechanisms of the condition, but also offer an avenue for future therapeutic interventions. From Humans to Mice: Insights Into The Importance of Antibodies The study demonstrated that antibodies obtained from FMS patients had a significant impact on the sensitization of nociceptive neurons in mice. Mice treated with antibodies isolated from FMS patients displayed heightened sensitivity to noxious mechanical and cold stimulation. This increased responsiveness was also observed in nociceptive fibers of skin-nerve preparations. These observations suggest that antibodies from FMS patients may play a crucial role in the development of FMS hypersensitivity. In addition to heightened sensory sensitivity, the researchers observed that mice treated with FMS IgG exhibited reduced movement and paw grip strength, mirroring some of the functional impairments seen in human FMS patients. Crucially, the study demonstrated that antibody-depleted serum from FMS patients or antibodies from healthy control subjects did not produce the same sensory hypersensitivity, thus suggesting the symptoms seen were specific to FMS patient-derived antibodies. Mechanisms of Action Antibodies derived from FMS patients did not directly activate sensory neurons, but rather were found to bind to various cellular components. Importantly, FMS antibodies also exhibited binding to human dorsal root ganglion, suggesting potential cross-species relevance. Implications and Future Directions The findings of this work shed light on the pathophysiology of FMS; by demonstrating the role of antibodies isolated from patients in contributing to the symptoms characteristic to FMS, a novel target for therapeutic approaches has been identified. Treatments aimed at reducing the levels of antibodies may prove efficacious. Going forward, more work must be done to further substantiate these results, but it is certainly a critical step towards decoding the mystery of fibromyalgia. You can learn more about the organisation behind this research here . Author BioFocus Newsroom Previous Next
- Immuno-Oncology 360º | BioFocus
< Back World Health Summit 11th - 13th October, 2026 Berlin, Germany From Crisis to Resilience: Innovating for Health ! Widget Didn’t Load Check your internet and refresh this page. If that doesn’t work, contact us. Previous Next
- Kandu Health and Neurolutions Merge to Transform Stroke Recovery | BioFocus
< Back Kandu Health and Neurolutions Merge to Transform Stroke Recovery Kandu, Inc. merges Kandu Health and Neurolutions to create an innovative, integrated approach to stroke recovery, combining advanced technology with personalized, at-home care. Kandu Health and Neurolutions have announced their merger, creating a new company—Kandu, Inc.—aimed at transforming stroke rehabilitation. The merger combines Neurolutions’ innovative brain-computer interface (BCI) technology with Kandu’s AI-driven telehealth services to provide a more integrated, accessible solution for stroke recovery. This move is expected to significantly improve care for stroke survivors, offering continuous, at-home rehabilitation and support. The newly formed Kandu, Inc. has also secured $30 million in financing, co-led by Ally Bridge Group and AMED Ventures, which will help expand its services. The company will use this funding to further enhance its telehealth platform, provide better caregiver support, and continue developing its groundbreaking technology for motor recovery. Neurolutions’ FDA-cleared IpsiHand® System, a BCI device that helps stroke survivors regain motor function, has already proven effective in improving limb movement for patients in the chronic phase of recovery. By merging with Kandu Health, the companies now offer a more comprehensive care model that combines cutting-edge technology with personalized rehabilitation at home. Kandu, Inc. aims to fill a gap in stroke recovery care, where patients often face fragmented treatment and high readmission rates. The merger brings together telehealth rehabilitation, therapy monitoring, and support for both patients and caregivers, helping to improve patient outcomes and quality of life. “This merger positions us to lead in stroke recovery,” said Leo Petrossian, CEO of Neurolutions, who will head the new company. “We now have the opportunity to offer stroke survivors the tools they need to enhance their recovery and regain independence.” With the merger, Kandu, Inc. is already seeing positive results. Early reports show that many patients who received its services achieved independent living within 90 days, with reduced hospital readmissions compared to traditional rehabilitation models. The company’s approach focuses on making stroke recovery more effective and accessible, offering a holistic and personalized care plan that goes beyond just medical treatments. Kandu, Inc. is set to redefine stroke rehabilitation by combining cutting-edge technology with patient-centered care, improving the recovery process for survivors and providing crucial support for their families. Author BioFocus Newsroom Previous Next
- 9th Annual CAR-TCR Summit Europe | BioFocus
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- 5th Risk-Based Quality Management Summit | BioFocus
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- 3D Printing of Bioreactors: a New Horizon for Bioprocess Development
Researchers develop a 3D-printed small-scale, single-use bioreactor that features real-time monitoring of cell growth. < Back 3D Printing of Bioreactors: a New Horizon for Bioprocess Development Researchers develop a 3D-printed small-scale, single-use bioreactor that features real-time monitoring of cell growth. The continuous development of upstream bioprocesses requires cost-effective and customizable bioreactors for optimizing production processes. Leveraging the recent advances in additive manufacturing, this research introduces a small-scale 3D printed bioreactor designed for both mammalian and microbial cultivations. The bioreactor boasts a 90 mL working volume and incorporates inline pH and dissolved oxygen probes, along with a levitating magnetic stirrer. A unique feature is the integration of aeration channels and a sampling port directly into the vessel walls. Additionally, a 3D printed customizable optical biomass sensor enhances the bioreactor's functionality. The study evaluated the bioreactor's performance through technical characterization and proof-of-concept cultivations. Results indicated that the mixing time and oxygen mass transfer were adequate for cultivating both mammalian and microbial cells at high densities. For instance, an Escherichia coli fed-batch cultivation achieved an impressive maximum OD600 of 204. In another demonstration, a fed-batch cultivation of a Chinese hamster ovary cell line producing IgG antibodies achieved a peak viable cell density of 10.2 × 106 cells mL−1 and a maximum product titer of 2.75 g L−1. A notable aspect of the bioreactor is its 3D printed customizable optical biomass sensor, allowing real-time monitoring of cell growth. By employing a three-parameter fit, the inline biomass signal was successfully correlated to corresponding offline values with satisfactory accuracy. This innovation holds promise for advancing the efficiency of upstream bioprocessing by providing a cost-effective and flexible tool for investigating and optimizing production processes. Author BioFocus Newsroom Previous Next
- Healthcare Policy: Myeloma UK's Call for Equitable NHS Access to CAR-T Cell Treatment | BioFocus
< Back Healthcare Policy: Myeloma UK's Call for Equitable NHS Access to CAR-T Cell Treatment Myeloma UK is campaigning to ensure life-changing CAR-T cell therapy is accessible to all myeloma patients, regardless of their ability to pay. In recent weeks, several hospitals in the UK have begun treating myeloma patients privately with CAR-T cell treatment. Myeloma UK , the only organisation in the UK exclusively dedicated to myeloma and related conditions, firmly believes that everyone should be able to access treatment, regardless of the ability to pay. They are committed to working with everyone involved to make CAR-T cell treatment available for people with myeloma on the NHS. Lifted from their recent blog post published in March 2025, Myeloma UK’s Senior Policy Officer, Amy Capper, explains what CAR-T cell treatment is, and what they’re doing to campaign for equal access. What is CAR-T cell treatment? CAR-T cell (chimeric antigen receptor T cell) treatment uses the body’s immune system to kill myeloma cells. The patient’s own T cells are collected and genetically modified in a laboratory so that they can recognise myeloma cells. The modified T cells are then infused back into the patient and can attack myeloma cells. What is Myeloma UK doing and why? While the treatment is not yet available for people with myeloma in the UK on the NHS – although there are some clinical trials running – Myeloma UK is fighting hard for access to new life-changing treatments like CAR-T cell treatment. We’ve joined together with our supporter Jason, who was fortunate to receive CAR-T cell treatment through a clinical trial, to call for the treatment to be made available on the NHS. As part of our work, Jason’s story was featured on the BBC last week. Jason was diagnosed with myeloma in 2014. Over the next nine years, Jason’s cancer returned three times and by spring 2023 he was told he had reached the end of the line. Thankfully, he was one of 11 people selected for a clinical trial in the UK to receive the pioneering new treatment known as cilta-cel (ciltacabtagene autoleucel) (Carvykti™), a type of CAR-T cell treatment. 15 months on, Jason is in remission for the very first time: “Until this treatment, I had never been in remission. Now they’re saying it’s undetectable. I never thought when I was diagnosed more than 10 years ago that I would ever get to this point. It feels surreal after all this time. “There were 11 people in the trial in the whole of the UK and I know other people weren’t as fortunate. “I want to give people hope and put pressure on the system to get this treatment where it needs to be. I am doing really well. It is fair to say I am at my best place since I was diagnosed, I have more energy and no side effects. I’m getting married next year. “It’s a bit of a gamble, you don’t know which treatment is going to work for you. That’s why we need more treatments like this available to people.” Shelagh McKinlay, Director of Research and Advocacy said: “Jason’s story shows just how important it is to have access to innovative treatments for people with myeloma. However, access to these treatments should never be on the ability to pay. That’s why we have joined together with Jason to make the call for CAR-T cell treatments to be made available on the NHS. Until we have a cure, Myeloma UK will continue fighting for all patients to have as many options as possible to keep their cancer at bay.” The specific CAR-T cell treatment that Jason was able to access through the clinical trial, cilta-cel, was initially put forward to be assessed by the National Institute of Health and Care Excellence (NICE) in a bid to make it available on the NHS. But the appraisal was terminated in March 2023 after the NICE drug approval process was halted. The case for approving CAR-T cell treatment in myeloma is challenging because the treatments are complex and difficult to manufacture and the trial data, while very promising, has not been as strong as in some other cancers. Since CAR-T cell treatment trials were first introduced, the UK has had other landmark drug approvals, particularly two new bi-specific antibodies, elranatamab (Elrexfio®) and teclistamab (Tecvayli®). This is great news and means that people living with myeloma have more options to treat their cancer. CAR-T cell treatment still can play a vital role in the treatment pathway and access to it should not be based on ability to pay. What’s next? Over the next few weeks Myeloma UK will be getting in touch with politicians as part of our ongoing campaigns and advocacy work. We will be making the case for improving access to CAR-T cell treatment for everyone that needs it. If you are interested in hearing more then please email policy@myeloma.org.uk and we will keep you updated on this work. FAQs Can I access CAR-T cell treatments now? There are some new clinical trials that are taking place for these types of treatment. You can use our Myeloma Trial Finder to see which clinical trials are open to new patients. We would always recommend that you speak to your healthcare team to discuss the best options for your treatment. Are there other similar types of treatment available for people living with myeloma? CAR-T cell treatment is a type of immunotherapy. Immunotherapies are a group of treatments which harness the patient’s immune system to kill myeloma cells. Although CAR-T cell treatments are only available on the NHS through clinical trials for now, we are now seeing more and more emerging immunotherapy treatments for myeloma. Read more about the different immunotherapies here. In recent years, we have seen approval of monoclonal antibodies like daratumumab (Darzalex®) and isatuximab (Sarclisa®), and last year we helped to make the first bispecific antibodies, elranatamab and teclistamab, available on the NHS. Help get more treatments approved on the NHS - for more information, click here . Author Amy Capper , Senior Policy Offer at Myeloma UK Previous Next
- Meet the Workhorses Behind Biologics' Next Growth Wave
The cell culture vessels industry is projected to grow from USD 5.10 billion in 2025 to USD 8.03 billion by 2030. < Back Meet the Workhorses Behind Biologics' Next Growth Wave The cell culture vessels industry is projected to grow from USD 5.10 billion in 2025 to USD 8.03 billion by 2030. Cell culture vessels are rarely the focus of conference keynotes or investor calls, but they’re an essential story in nearly every breakthrough in modern life sciences. Monoclonal antibodies, vaccines, viral vectors, cell and gene therapies; none of these advances exist without reliable ways to grow and maintain living cells. At the most basic level, cell culture vessels are the physical environments in which cells live outside the body. Flasks, Petri dishes, multi-well plates, roller bottles, and large-scale cell factory systems all serve the same fundamental purpose: providing cells with the right surface, gas exchange, and protection from contamination so they can grow predictably. What sounds simple becomes increasingly complex as processes scale up and regulatory expectations tighten. A vessel’s material, geometry, surface treatment, and sterility assurance can directly affect cell behaviour, productivity, and ultimately product quality. Indeed, as biologics and advanced therapies move from niche pipelines to mainstream medicine, the vessels that support cell culture are stepping into a much more strategic role. Decisions around cell culture vessels are becoming pivotal to success. Now, material science matters more than ever; with single-use bioreactors growing from handling small batches to 2,000L+ systems, the surfaces that contact cells, which directly affect yield, product quality, and process consistency, are an important consideration. Companies are investing heavily in surface modifications and polymer science to optimise cell attachment and growth. There are also scalability considerations when thinking about cell culture vessel strategy - moving from research-scale flasks to commercial-scale bioreactors isn't straightforward for advanced therapies. Autologous cell therapies (personalised to each patient) require rethinking the entire vessel strategy - for example, you may need many smaller, parallel systems rather than one large bioreactor. This is fundamentally different from traditional pharmaceutical manufacturing. In trying to intensify the process, there's a push toward perfusion systems and continuous manufacturing; here, vessel design directly impacts productivity. The geometry, mixing characteristics, and monitoring capabilities of vessels are now critical process parameters, not afterthoughts. As these therapies reach more patients, regulators are scrutinising manufacturing consistency more carefully. Vessels and their qualification become part of the validated manufacturing process, making vendor selection and design choices strategic business decisions. All of these strategic decisions ultimately determine whether biologics can reach patients reliably and affordably. Companies like Cytiva, Sartorius, and Thermo Fisher are positioning bioreactors as integrated systems with sensors, AI-driven process control, and data analytics - not just steel tanks. The vessel has become a platform technology. This is why cell culture vessels matter far beyond the lab bench. Cell culture itself underpins much of modern medicine , enabling drug discovery, safety testing, and the manufacture of biologics that treat cancer, autoimmune disease, and rare genetic disorders. It is also central to vaccine production and to newer approaches that rely on living cells as the therapy. As society demands more targeted, effective, and personalised treatments, the pressure on cell culture systems to deliver consistency at scale continues to grow. That pressure is reshaping the cell culture vessels industry. According to recent analysis from MarketsandMarkets , demand is being driven by the expansion of biologics pipelines, increased vaccine development, and rapid growth in cell and gene therapy programs. These applications require vessels that support higher cell densities, scale efficiently from research to production, and reduce the risk of contamination. As a result, manufacturers are moving away from improvised or legacy solutions toward more standardised, purpose-built culture systems. Large-scale cell factory systems and cell stacks have become central to adherent cell production in biopharmaceutical manufacturing, while single-use designs are now the default in many facilities. The appeal is fewer cleaning steps, faster turnaround times, and lower cross-contamination risk. At the same time, traditional formats such as cell culture flasks remain indispensable. Their flexibility and ease of use keep them firmly embedded in early-stage research, process development, and routine laboratory work across both academic and industrial settings. Pharmaceutical and biotechnology companies are the primary force behind this growth. As more organisations bring biologics development in-house, they need cell culture systems that can be used consistently across discovery, development, and manufacturing. Research institutes, hospitals, and contract research organisations are also contributing, particularly as advanced in vitro models and cell-based assays become more important in drug screening and toxicity testing. Geographically, the picture reflects broader shifts in the life sciences landscape. North America continues to set the pace in terms of established biopharmaceutical infrastructure, while Asia Pacific is emerging as a key growth engine. Expanding biotech hubs, notably in Singapore and China , increased research funding, and rising local manufacturing capacity are fueling demand for modern cell culture vessels, especially those aligned with global quality and regulatory standards. Despite its momentum, the industry faces familiar challenges. Standardisation across vessels and workflows remains difficult, particularly when processes are transferred between sites or scaled rapidly. Sustainability is also becoming a concern as single-use plastics proliferate. Suppliers are under growing pressure to balance performance, cost, and environmental responsibility while maintaining the reliability that regulated manufacturing demands. Leading companies such as Thermo Fisher Scientific, Danaher, Sartorius, Corning, Eppendorf, Lonza, and Getinge are responding by treating cell culture vessels not as commodities, but as integral components of bioprocess design. Investments in surface chemistry, scalable formats, and closer collaboration with biopharma companies and CDMOs point to a future where vessels are tailored to specific workflows rather than chosen off the shelf. The growth of the cell culture vessels market is not a story of flashy innovation, but of quiet necessity. Cell-based science continues to reshape medicine, and these vessels are becoming foundational infrastructure, unassuming in appearance, but essential to the development of our therapies. Author BioFocus Newsroom Previous Next
- The Current and Future State of Personalised Medicine | BioFocus
< Back The Current and Future State of Personalised Medicine How precision treatments, AI, and genomics are reshaping healthcare by using advanced technologies to deliver tailored treatments, early diagnoses, and better patient outcomes. Personalised medicine is transforming healthcare by harnessing molecular genetics, big data, screening, and early diagnosis to deliver targeted treatments. Unlike traditional one-size-fits-all approaches, it tailors therapies to a patient’s unique genetic and biological profile. Beyond treating illness , it enables proactive, preventative care by identifying potential health risks early and bringing awareness to individuals. By integrating collaborative healthcare solutions that ensure the right treatment is delivered to the right patient at the right time, personalised medicine is redefining medical standards. With a well-established connection between high-quality care, patient engagement, and improved health outcomes, this rapidly evolving field presents significant opportunities for both healthcare providers and businesses. Personalised medicine shifts the role of patients from passive to active, as patients are increasingly engaged in pursuing medical insights and making decisions about their treatments with healthcare professionals rather than simply following a doctor’s orders. The traditional top-down approach in healthcare is transitioning into a more collaborative model, where patients and healthcare providers work as partners. This shift represents a transfer from paternalistic medicine where doctors make decisions on behalf of patients, to participatory medicine where patients play an active role in their treatment plans. There are numerous benefits to personalised medicine including improved diagnostic accuracy, optimised treatment selection, increased patient engagement, reduced side effects, and ultimately, better health outcomes. Key technology advances driving personalised medicine The progress of personalised medicine has been advanced by key technologies, including developments in AI, CRISPR-Cas9, mRNA, and biomarkers. These innovations are crucial for achieving cost-effective healthcare solutions and improving overall patient outcomes. AI has transformed medical data analysis through integrating enormous amounts of patient-specific information to identify mutations, prescribe drugs, and predict responses to treatments. With tools like Tempus and Foundation Medicine using AI to analyse cancer mutations and recommend targeted therapies, and generative AI aiding in drug discovery by designing novel drug candidates, patient diagnoses are becoming more accurate and accelerated. Machine learning is also transforming oncology, with machine learning classifiers like OncoNPC being trained on more than 36,000 tumours to identify cancers of unknown origin and guide treatment strategies. Similarly, AI-driven models like eDICE are revolutionising the field of epigenetics by predicting variations that influence disease risk and treatment response. By leveraging epigenomic data to train algorithms, the model has successfully identified individual epigenetic differences, demonstrating its potential to enhance the role of epigenomics in personalised medicine. Additionally, AI is being utilised to personalise therapies based on an individual’s microbiome. Researchers have developed a model trained on over 41,000 microbiome-drug interactions, enabling the prediction of drug-induced disruptions to the microbiome and identifying which microbial taxa are most affected. This is an important advancement, as the microbiome plays a crucial role in drug metabolism, influencing both the efficacy of treatments and the likelihood of adverse drug reactions. CRISPR-Cas9 is a major advancement in personalised medicine, allowing precise modifications to DNA and genetic-level changes. The FDA's recent approval of Casgevy , a CRISPR-based therapy for sickle cell disease, marks a significant milestone in genetic medicine and is projected to achieve global sales of $593 million by 2029. With its ability to silence or modify genes, CRISPR-Cas9 has been employed in one of the most notable applications of personalized cancer therapy, CAR T-cell therapy. This approach involves extracting and genetically modifying a patient’s own T-cells to recognise and eliminate tumours before reinfusing them into the body. It has demonstrated remarkable success in treating leukaemia and lymphoma, offering hope to patients with cancers that relapse after chemotherapy. Meanwhile, mRNA therapies have made significant strides beyond their success in COVID-19 vaccines, with Pfizer’s Comirnaty being the first mRNA-based COVID-19 vaccine. The exploration of mRNA’s potential has continued to expand , as evidenced by Moderna’s development of mRESVIA, an mRNA vaccine targeting RSV. These advancements mark a broader shift toward utilising mRNA-based treatments for a wide range of conditions, including infectious diseases, cancer, and genetic disorders. Biomarkers and molecular diagnostics have become fundamental to personalised medicine, playing a crucial role in identifying genetic variations that influence disease progression and treatment response. Biomarker testing, including companion diagnostics and next-generation sequencing (NGS), is essential for matching patients with the most effective therapies. NGS stands out for its speed, accuracy, and reduced cost and time required for DNA sequencing. Moreover, there has been increasing interest in isothermal nucleic acid detection methods, with loop-mediated isothermal amplification emerging as a faster, more stable alternative to traditional PCR-based diagnostics. Despite these advancements, biomarkers still face challenges related to their instability and low concentrations, which can hamper reliable detection. To address these limitations, synthetic biosensors such as DNA barcoding are being employed to amplify and detect biomarkers, with recent studies also exploring the potential of CRISPR-Cas technology for enhanced precision and sensitivity towards biomarker detection. Current market and key players The global personalised medicine market was valued at $529.28 billion in 2023 and is projected to grow at a compound annual growth rate (CAGR) of 8.2% from 2024 to 2030. This growth is driven by the increasing demand for novel drug discovery, particularly for cancer and rare diseases. The widespread adoption of NGS has also accelerated the development of personalised treatments. Additionally, strategic collaborations between pharmaceutical companies and research institutions have fuelled innovation in this field. Leading companies in the personalised medicine market include GE Healthcare, Illumina, Inc., Abbott, Danaher Corporation (Cepheid, Inc.), QIAGEN, and Exact Sciences Corporation. These organisations are at the forefront of developing cutting-edge diagnostic solutions and investing in research to advance personalised treatment options. Their efforts continue to shape the future of precision medicine, making it more accessible and effective for patients worldwide. Barriers to personalised medicine Despite its promise, personalised medicine faces several challenges. One of the most critical issues is the lack of diversity in genomic research. Over 90% of genome-wide association studies have focused on individuals of European descent, limiting the applicability of personalised medicine for other populations. This lack of representation poses a significant barrier to equitable healthcare. Additionally, socioeconomic disparities impact access to advanced genomic testing and personalised treatments, further raising health inequalities. Data management is another critical issue in personalised medicine. The enormous amount of genetic and health data generated through personalised medicine requires secure storage, accurate interpretation, and strict protection. Healthcare institutions must invest in infrastructure capable of handling large-scale genomic data while complying with strict data protection regulations. Many organisations, particularly smaller healthcare facilities, struggle with the high costs and complexity of integrating AI and big data analytics into their systems. Regulatory and ethical concerns further complicate the adoption of personalised medicine. Informed consent is essential, as patients must fully understand how their genetic data is used and who has access to it. As regulatory approval pathways are not well established for personalised medicine, this creates further complexity. The risk of genetic discrimination is another issue, with potential implications for insurance, employment, and healthcare access. Comprehensive policies must be established to prevent discrimination and safeguard patient confidentiality. Furthermore, workforce training is crucial, as healthcare professionals require specialised skills to interpret genetic data and incorporate personalised medicine into clinical practice. Lastly, costs are a major barrier for personalised medicine market. Research and development costs for companies investing in precision medicine is more expensive than traditional medicines due to the requirement of companion diagnostics and genetic testing. The heavy requirement for biomarkers results in the need for larger patient groups and higher costs, both of which are significant barriers. In 2022, the cost of precision medicine treatment in North America averaged to around USD $300,000. Whilst by 2027 it is projected to drop below USD $260,000, it is still an extremely expensive mode of patient care. Future outlook Personalised medicine is ready to reshape healthcare by offering targeted, efficient, and patient-centric solutions. As AI, genomics, and biotechnology continue to evolve, medicine will increasingly be tailored to the individual, improving treatment efficacy and patient outcomes. However, addressing key challenges such as improving accessibility, ensuring data security, and establishing clear regulatory frameworks, will be essential to fully realising the potential of precision medicine. Despite these challenges, personalised medicine is widely regarded as the most promising approach for treating and potentially curing significant diseases. To make this vision a reality, key stakeholders including pharmaceutical and biotech companies, diagnostic firms, regulatory agencies, payers, and policymakers must work together to remove obstacles and provide incentives for continued innovation. By fostering collaboration and investing in cutting-edge research, the healthcare industry can ensure that personalised medicine becomes a standard of care, transforming lives and setting a new benchmark for medical treatment. Author Amrithavarshini Omprakash , freelance contributor Previous Next
- Scottish Brain Sciences Opens New Alzheimer’s Clinical Research Centre at ONE BioHub | BioFocus
< Back Scottish Brain Sciences Opens New Alzheimer’s Clinical Research Centre at ONE BioHub Aberdeen expansion widens access to early-stage trials and strengthens Scotland’s position in global brain health innovation. Scottish Brain Sciences (SBS) has opened a major new Alzheimer’s clinical research centre at ONE BioHub in Aberdeen, expanding access to cutting-edge clinical trials for communities across the north-east of Scotland and accelerating national efforts to detect and treat neurodegenerative conditions earlier. The state-of-the-art site, officially opened on 14 November, becomes SBS’s third research facility, joining its St Andrews hub and Edinburgh headquarters. The Aberdeen centre will support advanced clinical studies in early diagnosis, intervention and precision medicine, bringing world-class research closer to patients who have historically faced long waits and long journeys to participate in trials. By locating within ONE BioHub, a flagship launchpad for high-potential life sciences companies, SBS becomes part of a growing cluster of research-driven organisations, including NovaBiotics and Genomes.io. The move links neurological research directly with clinical, commercial, and translational pathways, reinforcing Scotland’s ambition to position itself as a global leader in brain health innovation. For Professor Craig Ritchie, CEO and Founder of Scottish Brain Sciences, the expansion is as much about equity of access as scientific ambition. “The people of the north-east deserve early access to breakthroughs in brain health and dementia research,” he said. “This new site will help ensure that people here can take part in trials, receive advanced assessments, and contribute to discoveries that could change the future of Alzheimer’s disease. Inviting one of our research participants to officially open this centre reflects our belief that progress begins and ends with the people who volunteer to take part. They are the beating heart of discovery.” That participant was Lynne Carroll, an Aberdeen-based volunteer who spoke candidly about the challenges of obtaining a diagnosis and the importance of local opportunities to engage in research. “It took several years to receive my Alzheimer’s diagnosis, and that is the reality for so many people,” Carroll said. “By being part of research that aims to detect and treat Alzheimer’s earlier, I hope I can help make the path a little clearer for others in the future… When [SBS] shared plans to open a research site in Aberdeen, I was thrilled, as it means I will be able to take part in trials that may require regular visits. It is a privilege to be involved in today’s opening, and I would encourage anyone locally affected by Alzheimer’s to connect with the team.” The opening was also attended by Richard Lochhead MSP, Minister for Business and Employment, who highlighted the significance of the investment for Scotland’s innovation economy and public health landscape. As ONE BioHub’s first tenant, Scottish Brain Sciences’ continued expansion underscores the momentum of Aberdeen’s emerging life sciences cluster. Dr Deborah O’Neil OBE FRSE, Chair of ONE Life Sciences and BioAberdeen Ltd, said: “We are delighted that Scottish Brain Science is expanding at ONE BioHub. Their focus on advancing understanding and treatment of neurological conditions adds important strengths to our growing life sciences cluster. The north east has a vibrant community of life science innovators and it is wonderful to see our first tenant growing and scaling within ONE BioHub.” With Alzheimer’s disease affecting millions worldwide and early-intervention strategies gaining urgency, the new centre positions Scotland at the forefront of developing more precise, earlier, and more accessible approaches to treatment. For patients in the north-east, it represents something more immediate: a chance to contribute directly to discoveries that could change the trajectory of dementia care for generations. Author BioFocus Newsroom Previous Next










