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How precision treatments, AI, and genomics are reshaping healthcare by using advanced technologies to deliver tailored treatments, early diagnoses, and better patient outcomes. < 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

< Back A Simple Pad Could Transform Cervical Cancer Screening Population-based study shows menstrual blood testing matches clinic-based HPV detection, with far greater convenience. Cervical cancer screening saves lives, but getting screened requires something many women find understandably difficult: a clinic visit, a speculum exam, and a trained healthcare provider. Now, recent research from China highlights that there might be another way, one that requires nothing more than a sanitary pad and a post box. This is of particular importance in UK, where there are high rates of non-attendance at booked screening appointments, driven in large part by anxiety about the procedure itself. Speaking with a friend of BioFocus, who had recently attended her cervical screening, she described the concerning insight her NHS nurse shared: the low attendance rate, which consumes healthcare resources, is prompting discussions about whether the free screening programme can remain sustainable. The nurse went on to say that there is a genuine risk that cost pressures could push these vital services toward a pay-per-use model, a change that might improve attendance among those who book, but would almost certainly discourage many women from seeking screening at all. For a service that should be free, this would be a travesty. In a study published in The BMJ , researchers led by Zheng Hu at Wuhan University tested whether menstrual blood could be used to detect human papillomavirus (HPV), the virus responsible for nearly all cervical cancers. The results were impressive: a small absorbent strip worn during menstruation detected precancerous cervical lesions just as accurately as samples collected by clinicians during pelvic examinations. The study enrolled over 3,000 women aged 20-54 across urban and rural communities in Hubei Province. Each participant provided three samples: menstrual blood collected at home using a prototype "minipad," plus the standard clinic-based cervical sample and Pap smear. Women who tested positive on any method were referred for biopsy to check for cervical abnormalities. The minipad performed remarkably well. For detecting high-grade precancerous lesions (CIN2+), it showed 94.7% sensitivity compared to 92.1% for clinician-collected samples, statistically identical. The negative predictive value was 99.9% for both methods, meaning a negative result was equally reassuring regardless of collection method. Both approaches required similar numbers of follow-up procedures per diagnosis detected (about 10 colposcopies per case of CIN2+). The minipad did show slightly lower specificity (89.1% vs 90.0%), meaning it flagged a few more women for follow-up who ultimately didn't have disease. But this modest difference seems a reasonable trade-off for the potential benefits: privacy, convenience, and dramatically expanded access to screening. What makes menstrual blood work? As blood flows through the cervix and vaginal canal during menstruation, it picks up shed cells from these tissues, including any HPV-infected cells. The researchers designed their sampling strip to adhere to regular sanitary pads, allowing women to use their preferred menstrual products while ensuring standardised collection. When the strip became sufficiently saturated, participants placed it in preservation solution and either mailed it to the lab or handed it to community health workers. The concordance between methods was impressive. When researchers compared specific HPV genotypes detected, the two collection methods agreed 97.7% of the time. Among the most common high-risk types detected were HPV52, HPV16, and HPV58, the same pattern seen with both collection methods. Perhaps most tellingly, 92% of participants in an earlier pilot study preferred self-collection over clinic visits. In a culture where menstruation has traditionally been considered private or even taboo, the acceptance rate suggests the practical advantages, convenience, privacy, and lack of discomfort, can overcome cultural reservations when the health benefits are clear. This matters because cervical cancer remains a major killer, particularly in low- and middle-income countries where 85% of the roughly 661,000 annual cases occur. Screening programmes face persistent challenges such as insufficient healthcare infrastructure in rural areas, cultural barriers around pelvic examinations, lack of trained providers, fear of pain, and the logistical burden of clinic visits. Minipad product and usage Self-collection methods aren't new. Vaginal swabs and cervicovaginal brushes have been studied for years, with relative sensitivities ranging from 77% to 96% compared to clinician sampling. But these still require vaginal insertion, which some women find uncomfortable or culturally unacceptable. In the earlier pilot work for this study, 22.9% of women declined participation specifically because of discomfort with vaginal swabs. Menstrual blood collection sidesteps this entirely. There's no insertion, no manipulation, no medical procedure at all, just a modified sanitary pad worn during a woman's normal menstrual cycle. The researchers even developed a WeChat mini-program called "Early Test" where participants could track their results, ask questions, and access educational resources about HPV and cervical health. The study does have limitations worth noting. Women with negative results on both tests weren't biopsied (which would be unethical given their extremely low risk), so the researchers had to assume these women truly didn't have disease. This could theoretically introduce bias, though previous large studies suggest fewer than 1 in 1,000 such women would have high-grade lesions. There's also an additional biological consideration - because menstrual blood contacts a broader anatomical area than a cervical swab, including the vagina and vulva, it might detect HPV infections from these other sites. This could explain the slightly higher rate of positive results and might mean some women get referred for colposcopy unnecessarily. Future research testing additional biomarkers could help improve specificity. The researchers are careful not to oversell their findings. They're not suggesting menstrual blood testing should immediately replace current screening protocols. But as an alternative for women who can't or won't access clinic-based screening? The evidence is compelling. What happens next matters enormously. The minipad device used in this study is a research prototype, not yet commercially available. Questions about cost-effectiveness, quality control across different laboratories, and integration into existing healthcare systems all need addressing. The researchers acknowledge that implementation research (tracking real-world uptake, retention, and clinical outcomes) will be crucial. But the core finding stands, which is that for detecting the cervical abnormalities that can progress to cancer, menstrual blood seems to work. It's non-invasive, private, convenient, and accurate. In a world where millions of women lack access to screening, that combination could prove genuinely transformative. As the global health community works toward eliminating cervical cancer as a public health problem, innovations like this, ones that meet women where they are rather than demanding they navigate complex healthcare systems, may prove as important as the science itself. Sometimes the biggest breakthroughs aren't about discovering something entirely new, but about finding a simpler way to do what we already know works. Author BioFocus Newsroom Previous Next

< Back Monkeypox: A Persistent Threat? What makes this outbreak more concerning from previous years, and what is its likelihood of developing into a pandemic? Earlier this summer, the World Health Organisation (WHO) announced a global health emergency. Since then, international headlines have been flooded with news of a new Mpox (monkeypox) outbreak, which has surged in the Democratic Republic of Congo (DRC) and several other countries in Africa, where cases had never been seen before. Mpox is a rare infectious disease caused by the monkeypox virus (MPXV), a species of the genus Orthopoxvirus . Those infected with the virus experience flu-like symptoms (fever, headache, muscle aches, fatigue, and swollen lymph nodes) accompanied by a skin rash or mucosal lesions that last two to four weeks. It can be transmitted by close contact with the bodily fluids or lesions of an infected human or animal, as well as human-to-human transmission via respiratory droplets and sexual contact. The WHO declared the escalating global Mpox outbreak a Public Health Emergency of International Concern (PHEIC) from 2022 to 2023 and again in 2024. What makes this outbreak more concerning from previous years, and what is its likelihood of developing into a pandemic? The ongoing threat of Monkeypox The recent upsurge in Mpox cases has been a significant cause of concern for public health officials worldwide. An increasing number of cases have been reported in the United States , Canada, the United Kingdom , and some European countries . Between January 2022 and August 2024, more than 120 countries have documented cases of Mpox, with over 100,000 laboratory-confirmed cases and more than 220 deaths among those confirmed cases. The ongoing spread of the virus can be attributed to a combination of factors, including increased testing, improved surveillance, and the virus's ability to evade immunity. Studies have found that the monkeypox virus is evolving and mutating, which poses a challenge to acquired immunity and can potentially impact the effectiveness of vaccines. Clade I and II: a complicating factor There are two clades of MPXV: Clade I and Clade II. Clade I is the most severe form of the disease, with subtypes Ia and Ib. It is more contagious than Clade II and leads to severe illness and, in some cases, death. Clade II, a milder form of the disease, caused the 2022 Mpox outbreak. Clade Ib is the most recent cause for concern as it is behind the most recent monkeypox outbreak. This variant behaves differently from other types of the virus—it spreads mainly via human contact and has a faster rate of transmission. Cases with Clade Ib have been identified in the DRC and neighbouring countries in Africa but have not been detected outside the continent. Potential for a pandemic With the emergence of new virus variants, researchers are finding it increasingly important to understand the spread of this virus in order to curb its transmission. However, the current Mpox outbreak cannot be compared to the COVID-19 pandemic as the rate of transmission is significantly lower. The likelihood of it evolving into a pandemic is low. What happens next? The recent surge in Monkeypox cases has posed a significant threat to global public health. While the situation has stabilised in recent months, the potential for future resurgences and the emergence of more contagious variants remains a concern. Notably, the emergence of Clade Ib has raised concerns due to its potential to evade immunity and impact vaccine effectiveness. The continued coordination of international efforts in surveillance, testing, and vaccine development remains crucial in addressing this persistent threat. For more information on the current Mpox outbreak and the potential for vaccine development, read this article. Author Mariam Zaki , freelance contributor Previous Next

< 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

Dive into the leading DNA sequencing technologies driving genomic discovery from Oxford Nanopore, Illumina, PacBio, and 10x Genomics. < Back The Four Powerhouses of DNA Sequencing Dive into the leading DNA sequencing technologies driving genomic discovery from Oxford Nanopore, Illumina, PacBio, and 10x Genomics. DNA sequencing has come a long way in the past two decades, revolutionizing how we explore the complexities of life. From understanding disease pathways to advancing personalized medicine, cutting-edge technologies from Oxford Nanopore , Illumina , PacBio , and 10x Genomics are leading the charge. Each of these companies offer unique tools that cater to different scientific and industrial needs. Dive into what makes each one a standout in this dynamic landscape. Oxford Nanopore Technologies: Real-Time Sequencing On the Go Imagine carrying a sequencing device in your pocket and performing genomic analysis anywhere, from a lab bench to a rainforest. That’s the power of Oxford Nanopore Technologies (ONT). Their platforms, including the MinION, GridION, and PromethION, use nanopore-based sequencing—a method where single-stranded DNA passes through a tiny pore, causing electrical disruptions that identify each nucleotide in real time. ONT delivers ultra-long reads, often exceeding hundreds of kilobases. This capability is a game-changer for resolving structural variants and repetitive genomic regions. The portable MinION device enables on-site sequencing for applications like infectious disease tracking and environmental analysis. Real-time sequencing lets researchers make rapid decisions—ideal for clinical and field-based studies. However, while ONT’s technology is versatile, it’s not without challenges. Accuracy, though improving, historically lags behind competitors, and throughput can be inconsistent depending on sample quality. Illumina: The Gold Standard of Short Reads For years, Illumina has set the benchmark for sequencing accuracy and throughput. Their sequencing by synthesis (SBS) technology powers platforms like the NovaSeq and MiSeq, which are staples in laboratories worldwide. With error rates as low as 0.1%, Illumina’s short-read data is trusted for applications like clinical diagnostics and population genomics. The NovaSeq can churn out terabases of data in a single run, perfect for large-scale projects. While equipment costs are high, per-base sequencing costs are exceptionally low, making Illumina a cost-efficient option for high-volume projects. But, Illumina’s short-read sequencing (150-300 base pairs) has its limits, especially when it comes to tackling complex genomic regions or assembling new genomes. Hybrid approaches often pair Illumina’s accuracy with long-read technologies to bridge this gap. PacBio: Marrying Length and Accuracy When accuracy and long reads are non-negotiable, Pacific Biosciences (PacBio) delivers. Their single-molecule, real-time (SMRT) sequencing technology is celebrated for generating highly accurate long reads (HiFi reads), which are ideal for tackling complex genomes. Spanning up to 15-20 kilobases with >99.9% accuracy, HiFi reads combine the strengths of long-read sequencing with unparalleled precision. PacBio’s ability to detect DNA modifications, like methylation, adds an epigenetic dimension to sequencing projects. Whether it’s structural variants or phased haplotypes, PacBio’s data excels in delivering comprehensive genomic insights. The trade-off? PacBio’s instrumentation and reagents are costlier compared to ONT or Illumina, and achieving high coverage can increase both expense and time. 10x Genomics: A Multidimensional Perspective While not a sequencing platform itself, 10x Genomics transforms how researchers extract value from sequencing data. Their Chromium platform partitions DNA or RNA into droplets, enabling downstream library preparation for platforms like Illumina. By linking short reads to long genomic fragments, 10x Genomics reconstructs haplotypes and resolves structural variants. From immunology to oncology, Chromium’s single-cell capabilities provide unparalleled insights into cellular diversity and function. The ability to integrate genomic, transcriptomic, and epigenomic data is a boon for systems biology. That said, 10x Genomics is reliant on external sequencing platforms, and its linked-read approach has faced competition from emerging long-read technologies. The Final Word Each sequencing technology has carved out a niche, excelling in different areas of genomics. Oxford Nanopore’s portability and real-time sequencing are unmatched for field applications. Illumina remains the go-to for short-read accuracy and large-scale studies. PacBio’s HiFi reads bridge the gap between accuracy and long-read capabilities, while 10x Genomics unlocks new dimensions in single-cell and multi-omics research. The genomics revolution is far from over, and as these technologies continue to evolve, so too will our ability to decode the complexities of life at unprecedented depth and precision. For researchers and biotech professionals, the challenge lies not in choosing the best technology overall, but the best technology for the question at hand. Author BioFocus Newsroom Previous Next

In a phase 1 study, 81% of patients responded to IL18-boosted therapy, with over half achieving complete remission. < Back Breakthrough "Armoured" CAR T-Cell Therapy Shows Promising Results in Tough-to-Treat Lymphomas In a phase 1 study, 81% of patients responded to IL18-boosted therapy, with over half achieving complete remission. CAR T-cell therapy revolutionized cancer treatment by using a patient’s own immune cells to target blood cancers. However, its impact has been limited by relapse and resistance , especially in B-cell lymphomas—where more than half of patients do not achieve lasting remission after receiving current FDA-approved therapies. Researchers at Penn Medicine have developed a new, next-generation version of this therapy, called huCART19-IL18, designed to overcome these challenges. Unlike traditional CAR T cells, this “armoured” version not only targets cancer cells but also secretes interleukin-18 (IL18), a molecule that enhances immune activity and supports the engineered cells within the tumor microenvironment. In a recently published phase 1 study in the New England Journal of Medicine , the results were highly encouraging. Among 21 patients with aggressive, treatment-resistant B-cell lymphomas, most of whom had received multiple prior therapies, including earlier-generation CAR T products, 81% experienced tumor shrinkage, and 52% achieved complete remission. Some patients have remained in remission for over two years, suggesting durable responses may be possible with this approach. Importantly, the therapy did not introduce new or unexpected safety issues. Side effects such as cytokine release syndrome and neurotoxicity remained consistent with those observed in standard CAR T therapies and were manageable using existing treatment protocols. The strategy behind huCART19-IL18 centers on boosting the immune system’s ability to sustain its attack on cancer cells. Like most other CAR T therapies for B-cell lymphoma, the engineered T cells are designed to recognize the CD19 protein found on malignant cells. What sets this therapy apart is its built-in production of IL18, a pro-inflammatory cytokine that helps activate and recruit other immune cells, reinforcing the overall immune response. This addition appears to counteract common barriers to CAR T-cell effectiveness, such as immune suppression within the tumor environment and T-cell exhaustion. Early biological data collected during the study supports the idea that IL18 significantly contributed to the high response rates observed. One of the other major innovations of the Penn team is a streamlined manufacturing process that reduces production time for the CAR T cells from the standard 9–14 days down to just three. For patients with fast-growing cancers, this shorter turnaround can make a critical difference, allowing treatment to begin before the disease progresses further. There’s also evidence that this quicker manufacturing timeline may improve the therapeutic potency of the T cells. This study marks the first time a cytokine-enhanced CAR T-cell therapy has been tested in patients with blood cancer, and the implications extend well beyond lymphoma. The underlying concept, arming CAR T cells with immune-boosting cytokines, could be applied to other cancers, including those where CAR T therapies have historically shown limited success, such as solid tumors. Building on these results, the researchers are preparing additional clinical trials, including studies targeting acute lymphoblastic leukemia and chronic lymphocytic leukemia. A trial for non-Hodgkin’s lymphoma using a similar IL18-enhanced CAR T product is already underway. Efforts are also ongoing to refine and scale the manufacturing process in partnership with a biotechnology spinout, with the goal of expanding access to these advanced therapies. Beyond clinical outcomes, the data generated from this trial is providing valuable insight into how and why CAR T therapies fail in some cases, helping guide future improvements in treatment design and patient selection. This therapy represents a significant step forward in the evolution of personalized cancer immunotherapy, offering new hope for patients with few remaining options, and potentially laying the groundwork for broader applications in oncology. Author BioFocus Newsroom Previous Next

In a significant move to bolster local vaccine production across Africa, South African pharmaceutical company Biovac has entered into strategic agreements with Sanofi and EuBiologics. < Back Biovac Advances Africa's Vaccine Production with Sanofi and EuBiologics Partnerships In a significant move to bolster local vaccine production across Africa, South African pharmaceutical company Biovac has entered into strategic agreements with Sanofi and EuBiologics. In a significant move to bolster local vaccine production across Africa, South African pharmaceutical company Biovac has entered into strategic agreements with Sanofi and EuBiologics. These partnerships aim to enhance the continent's self-sufficiency in vaccine manufacturing and distribution, aligning with Africa's broader goal of increasing local vaccine production to meet 60% of its needs by 2040. Through its collaboration with Sanofi, Biovac will become the first African producer of inactivated polio vaccines (IPV). While Sanofi will continue to manufacture the drug material for the vaccines, Biovac will take on the roles of late-stage formulation, filling, packaging, and delivery of millions of doses to UNICEF for distribution across Gavi-supported countries in Africa. This marks a critical step in reinforcing Africa's capacity to handle future vaccination needs independently. Sanofi, a long-standing contributor to the Global Polio Eradication Initiative, has already delivered over 1.5 billion doses of IPV to UNICEF through Gavi . This initiative has successfully immunized 2.5 billion children over the past 30 years, reducing polio cases by 99% worldwide. However, the COVID-19 pandemic disrupted routine pediatric vaccination programs, highlighting the need for increased local production to prevent a resurgence of polio cases. Thomas Triomphe, Sanofi’s Executive Vice President of Vaccines, emphasized the importance of this partnership in ensuring Africa’s readiness for future vaccination demands. Biovac CEO, Dr. Morena Makhoana, expressed pride in the partnership, underscoring the company's commitment to improving vaccine access and health outcomes across the continent. In another strategic move, Biovac has partnered with South Korean vaccine manufacturer EuBiologics to complete a technology transfer for a fully liquid meningococcal pentavalent conjugate vaccine. This collaboration, initially established through a memorandum of understanding last year, aims to supply the vaccine primarily in the meningitis belt of sub-Saharan Africa, which stretches from Senegal to Ethiopia. EuBiologics plans to produce 10 million doses annually, with the vaccine expected to launch in 2029. This agreement was finalized at the Global Forum for Vaccine Sovereignty and Innovation in Paris, where several initiatives were unveiled to support Africa's vaccine production capabilities. Notably, Gavi, the African Union, and the Africa Centres for Disease Control and Prevention (Africa CDC) launched the African Vaccine Manufacturing Accelerator (AVMA), a financing mechanism designed to provide up to $1 billion over the next decade. The AVMA aims to support commercially viable vaccine manufacturing projects in Africa, offering financial incentives to offset the initial costs of development and production. These partnerships and initiatives are pivotal in achieving the African Union’s objective of producing at least 60% of the continent's vaccines locally by 2040. By fostering local manufacturing capabilities, Africa can enhance its resilience against future pandemics and ensure more reliable access to essential vaccines for its population. The Biovac-Sanofi and Biovac-EuBiologics collaborations represent significant milestones in Africa’s journey towards vaccine sovereignty. For pharmaceutical professionals, these developments highlight the importance of strategic partnerships and innovation in addressing global health challenges and achieving sustainable healthcare solutions. Author BioFocus Newsroom Previous Next

Manchester spinout Cytotrait raises £3 million to develop organelle based genetic engineering that could boost crop yields, resilience and sustainability. < Back Engineering Crops From the Inside Out Manchester spinout Cytotrait raises £3 million to develop organelle based genetic engineering that could boost crop yields, resilience and sustainability. Global agriculture faces a difficult balancing act: feeding a growing population while reducing environmental impact and adapting to climate change. A newly funded biotechnology startup from Manchester believes it has a technological answer to part of that challenge. Cytotrait, a spinout from the University of Manchester, has secured £3 million in seed funding to develop advanced genetic engineering approaches for major crops. The round was led by Northern Gritstone, with participation from the UK Innovation and Science Seed Fund and the Northern Universities Venture Fund. The investment will allow the company to expand development programmes aimed at improving the productivity, resilience and environmental footprint of staple crops such as wheat, maize, potato and canola. While early stage funding announcements are common in the rapidly growing agritech sector, Cytotrait’s platform technology reflects a broader shift in how scientists are approaching crop engineering. Instead of focusing solely on the nuclear genome, the central DNA repository within plant cells, the company is targeting genes in cellular organelles such as chloroplasts and mitochondria. Engineering beyond the nucleus At the centre of Cytotrait’s approach is a technology known as the Mutant Organelle Selection System, or MOSS. The system enables scientists to introduce genes or gene edits into chloroplasts and mitochondria and rapidly achieve “homoplasmy”, meaning every copy of the organelle genome within a plant cell carries the engineered change. This is significant because plant cells can contain hundreds of copies of organelle genomes. In conventional engineering approaches, only a fraction of these copies may initially carry a modification, creating inconsistencies in gene expression and complicating breeding programmes. By ensuring uniform genetic modification across organelles, MOSS could enable high level, localised expression of engineered traits. According to Cytotrait, this offers several practical advantages: reduced toxicity from transgene expression, improved ability to combine multiple traits in a single plant line, and potentially more straightforward regulatory pathways. Engineering traits in chloroplasts can also provide a natural containment mechanism. In many crops, chloroplast DNA is inherited primarily through the maternal line, which can limit the spread of engineered genes through pollen, a long standing concern in agricultural biotechnology. These characteristics could make organelle based engineering particularly attractive for applications where strong expression of specific proteins or metabolic pathways is needed. Applications across major crops With the new funding, Cytotrait plans to initiate research programmes targeting several globally important crops in European and North American markets. These include wheat and maize, two of the world’s most widely cultivated grains, as well as potatoes and canola. The company is exploring a range of potential applications. Some focus on traditional agricultural priorities such as higher yields or increased resilience to environmental stresses. Others aim to introduce entirely new traits that could enhance the nutritional value or functionality of crop products. A third area of interest reflects a growing trend in agricultural biotechnology: designing crops that contribute to climate mitigation. Cytotrait suggests its technology could enable traits that enhance carbon sequestration, potentially allowing crops to capture and store more carbon in plant biomass or soils. If realised, such capabilities would place crop biotechnology squarely within the emerging field of climate-positive agriculture, an area attracting increasing attention from investors and policymakers. Building on earlier research support The company has already received early validation from UK public research funding bodies. In 2025, the Advanced Research and Invention Agency awarded Cytotrait nearly £500,000 to apply its technology to hybrid seed production in wheat. Hybrid seeds, created by crossing genetically distinct parent plants, can dramatically increase crop yields through hybrid vigour. However, producing hybrid wheat reliably has long been a technical challenge. Technologies that enable controlled male sterility or fertility restoration in crops are therefore considered valuable tools for seed companies. Cytotrait’s research aims to use organelle engineering to create more reliable systems for hybrid seed production, potentially unlocking new yield gains in one of the world’s most important staple crops. A growing ecosystem for agritech spinouts The company also reflects the growing role of university spinouts in the biotechnology sector. Cytotrait emerged from research conducted at the University of Manchester and was commercialised with support from the institution’s technology transfer arm, the University of Manchester Innovation Factory. It also participated in NG Studios, a venture building programme run by Northern Gritstone that focuses on deep technology startups originating from northern UK universities. Such programmes aim to bridge a persistent gap between academic discovery and commercial development. This ecosystem is increasingly important for translating advances in synthetic biology and engineering biology into real-world applications. Agricultural biotechnology, in particular, often requires substantial early investment before products reach the field. The broader context: food security and sustainability Cytotrait’s funding announcement comes at a time when global food systems face mounting pressure. The world’s population is projected to approach 10 billion by mid century, while climate change is already affecting crop yields in many regions. At the same time, agriculture itself is responsible for a significant share of global greenhouse gas emissions and environmental degradation. Producing more food with fewer resources, such as land, water and fertiliser, has become a key goal for both governments and the agricultural industry. Biotechnology is widely viewed as one of the tools that could help meet that challenge. Advances in gene editing, synthetic biology and plant engineering are enabling researchers to design crops that are more resilient to drought, heat and disease, while also improving resource efficiency. However, regulatory hurdles and public acceptance remain significant factors shaping the deployment of genetically engineered crops. Technologies that offer improved containment, targeted expression or simplified regulatory pathways could therefore play an important role in the next generation of agricultural biotechnology. Cytotrait’s founders believe their platform could represent a step forward in that effort. As Dr Junwei Ji, Co-Founder and Executive Director of Cytotrait, said: “Food security and sustainability are two of our most pressing global challenges, and issues that we must be prepared to face today to ensure we are ready to meet the needs of tomorrow. We developed MOSS with those challenges in mind – a unique crop engineering solution capable of streamlining regulatory pathways and generating crops with new, enhanced, and more carbon-conscious traits. Thank you to our investors, whose support reaffirms our belief in the potential of MOSS to bring about a new frontier in crop technology.” Investors involved in the funding round also highlighted the significance of the company’s approach. Duncan Johnson, CEO of Northern Gritstone, said: “Cytotrait is a prime example of the world-class innovation from the North of England’s universities and the ambitious founders and teams we see on our venture building program, NG Studios. Northern Gritstone is very pleased to be working with Dr Ji and the team and looks forward to positive results from this first tranche of new development programmes.” Dr Tim Brears, Executive Chair of Cytotrait, emphasised the wider potential of the technology: “MOSS is truly a breakthrough in the field of crop technology, allowing us to precisely engineer characteristics that can not only enhance yield and resilience, but also help to drive a more sustainable future for modern agriculture. We’re extremely proud of everything our team has already accomplished, and thankful to our investors, whose support will enable us to expand our pipeline and explore the applications of MOSS in some of the world’s major crop types.” Hassan Mahmudul, Investment Manager at the UK Innovation and Science Seed Fund, added: “UKI2S invests in companies developing novel engineering biology solutions to tackle large, global challenges. We are delighted to welcome Cytotrait to our growing agritech portfolio, recognising the strength of its platform technology, which has the potential to unlock high-value trait expression at levels significantly beyond what is achievable through conventional nuclear genome engineering.” The coming years will determine whether organelle-based engineering can fulfil its promise in real-world agricultural systems. For now, Cytotrait’s seed funding marks an early step in translating a novel approach to plant biotechnology into tools that could help reshape the future of sustainable farming. Author BioFocus Newsroom Previous Next

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

< Back 1st – 2nd April, 2025 Barcelona, Spain RESI Europe Conference 2025 The RESI Europe Conference 2025 is coming back to Barcelona! Hosted by Life Science Nation (LSN) in collaboration with Biocat, RESI Europe continues its mission to connect healthcare innovators with global investors and partners. This year’s event features an in-person conference on April 1, 2025, at The InterContinental Barcelona, followed by two days of virtual partnering on April 2-3, 2025. Part of the globally recognized Redefining Every Stage Investments (RESI) series, this conference offers unparalleled opportunities for life science companies in drugs, devices, diagnostics, and digital health. With 250+ global investors/in-licensors and 250+ life science innovators expected, RESI Europe is your gateway to securing capital, partnerships, and licensing deals. Previous Register now Next

< Back Impulse Dynamics Secures $158M to Accelerate Heart Failure Pipeline Financing follows major CMS coverage decision for CCM therapy and supports next-generation heart failure devices. Impulse Dynamics has closed a $158 million financing round to support strategic growth, expand commercialization, and advance its clinical and technology pipelines for heart failure therapies. The funding round was anchored by new institutional investors Sands Capital and Braidwell, with continued support from existing investors including Redmile, Perceptive, and Alger, as well as several prominent industry executives. The investment underscores growing confidence in Impulse Dynamics’ vision and its role in addressing unmet needs in heart failure care. The financing follows a major regulatory milestone for the company: a recent Centers for Medicare and Medicaid Services (CMS) National Coverage Determination (NCD) for Cardiac Contractility Modulation (CCM®) therapy. The decision expands access to CCM therapy for more than 66 million Medicare beneficiaries and formally establishes the therapy as no longer experimental or investigational. CCM therapy was also selected as one of only five technologies for inclusion in CMS’s Transitional Coverage for Emerging Technologies (TCET) pathway in 2025. To date, more than 12,000 patients have received CCM therapy worldwide. The NCD removes a significant reimbursement barrier, paving the way for broader adoption among heart failure patients who continue to experience debilitating symptoms despite optimal medical therapy. Impulse Dynamics plans to use the new capital to expand access to CCM therapy while accelerating development of next-generation technologies, including the investigational CCM-D® HF System. The single device is designed to deliver both CCM therapy for heart failure symptom relief and implantable cardioverter defibrillator (ICD) therapy, which provides life-saving protection against sudden cardiac death. The company is also advancing several key clinical trials. These include the INTEGRA-D trial, which is evaluating the CCM-D HF System in patients who already require an ICD, and the AIM HIGHer trial, focused on patients with diastolic heart failure, an area with limited treatment options that represents approximately half of all heart failure cases. With a strengthened balance sheet, expanding reimbursement coverage, and a growing clinical pipeline, Impulse Dynamics is positioning itself as a leader in device-based therapies aimed at improving quality of life for people living with heart failure. Author BioFocus Newsroom Previous Next

Collaboration with SRI demonstrates Nuvec®’s ability to selectively deliver RNA to cancer cells, advancing prospects for targeted, orally delivered RNA therapeutics. < Back N4 Pharma and SRI Advance Targeted RNA Delivery with Nuvec® Platform for Cancer Therapies Collaboration with SRI demonstrates Nuvec®’s ability to selectively deliver RNA to cancer cells, advancing prospects for targeted, orally delivered RNA therapeutics. N4 Pharma plc (AIM: N4P) has released new data from its collaboration with SRI International demonstrating the potential of its proprietary Nuvec® delivery platform to precisely target RNA payloads to cancer cells, a breakthrough that could reshape how RNA therapeutics are developed and delivered. The findings point to a future where common cancers such as lung, breast, and pancreatic could be treated with targeted RNA therapies, potentially in oral tablet form and with fewer side effects than conventional chemotherapy. The collaboration combined SRI’s targeting molecules with Nuvec® particles to deliver small interfering RNA (siRNA) payloads to non-small cell lung cancer cells. Results showed that Nuvec® functionalised with a targeting molecule binding to the αvβ6 adhesion protein, a marker overexpressed in many epithelial cancers, achieved precision delivery and selective uptake. siRNA payloads were active only when carried by targeted Nuvec® particles, confirming their ability to direct therapeutic action to diseased cells while sparing healthy tissue. “These recent data from our collaboration with SRI are particularly exciting because they represent the first example of the use of Nuvec® for the potential treatment of some of the most common and life-threatening cancers,” said Nigel Theobold, CEO of N4 Pharma. The results further validate Nuvec® as a differentiated RNA delivery system, supporting its broader application in oncology and beyond. Alongside its potential for targeted delivery, the platform offers multiple advantages including the ability to carry more than one RNA therapy in a single particle, stability suitable for oral delivery, low immunogenicity, and scalability in manufacturing. “Targeting RNA therapies to particular cell types is highly sought after by companies developing RNA therapeutics,” Theobold added. “We have now demonstrated Nuvec®'s ability to do this in multiple systems, which we believe sets it apart from other RNA delivery methods.” The global RNA therapeutics market, valued at $13.7 billion in 2023 and forecast to reach $18 billion by 2028, is growing rapidly but faces persistent challenges in manufacturing and delivery. Targeting specific tissues and reducing systemic toxicity remain among the biggest hurdles. Nuvec®’s latest data suggest it could help overcome these barriers and support deal-making with RNA therapy developers looking for next-generation delivery solutions. N4 Pharma raised capital earlier this year to generate data supporting Nuvec®’s key claims, including multi-RNA delivery, targeted uptake, oral dosing, and stability, with the aim of advancing both licensing opportunities and its own RNA therapeutics pipeline. Its lead program, N4 101, is an oral anti-inflammatory therapy for inflammatory bowel disease (IBD) designed to showcase the platform’s capabilities. Author BioFocus Newsroom Previous Next