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The BioFocus Editorial Team recap key developments from 2024 and highlight some of the most promising clinical trials and emerging therapies to keep an eye on. < Back 2025: A Year of Promise in Pharma and Biotech — Key Trials and Drug Candidates to Watch The BioFocus Editorial Team recap key developments from 2024 and highlight some of the most promising clinical trials and emerging therapies to keep an eye on. As the new year begins, the life sciences sector is buzzing with anticipation about the clinical trials and drug candidates that could reshape the landscape of medicine in 2025. With advancements in technology, new biologics, and novel drug delivery systems, 2025 promises to be a year of breakthrough discoveries. In this article, we’ll highlight some of the most promising clinical trials and emerging therapies to keep an eye on, as well as recap some of the key developments from 2024 that set the stage for what’s to come. Key Drug Development Advancements in 2024 Before we look ahead to 2025, let’s first acknowledge some of the landmark moments in drug development from 2024. This year saw substantial progress in several therapeutic areas, including oncology, neurology, and gene therapy: mRNA Therapeutics Beyond COVID-19 : Following the success of mRNA COVID-19 vaccines, 2024 marked a pivotal year for mRNA technology, with several candidates entering late-stage trials for infectious diseases like flu and RSV (Respiratory Syncytial Virus). Pfizer and Moderna are leading the charge in developing mRNA vaccines for a variety of conditions, including cancer, autoimmune diseases, and rare genetic disorders. Gene Editing Advancements : CRISPR and other gene-editing technologies gained significant traction in 2024, with companies like CRISPR Therapeutics and Editas Medicine advancing clinical trials targeting genetic disorders such as sickle cell disease and beta-thalassemia. Early results have shown promise in permanently correcting gene mutations with minimal side effects. Immuno-Oncology Breakthroughs : The immuno-oncology space continued to evolve, with multiple trials showing significant progress in treating cancers that were once considered resistant to immune therapies. The approval of Roche’s Vabysmo and early results from Bristol-Myers Squibb's LAG-3 inhibitor in melanoma and other cancers have raised hopes for novel immune checkpoint inhibitors. Neurodegenerative Disease Innovation : In Alzheimer's and Parkinson’s disease, 2024 saw promising data from Biogen’s Leqembi (lecanemab) and Eli Lilly’s Donanemab . These therapies represent a step forward in targeting amyloid plaques, though the debate over efficacy and safety continues to shape the regulatory landscape. Top Clinical Trials and Drug Candidates to Watch in 2025 As we look forward to 2025, the industry is turning its attention to several promising drug candidates and clinical trials that could change the way we treat a wide range of diseases. Below are the trials and candidates we’re most excited about: 1. AstraZeneca's Enhertu (Trastuzumab Deruxtecan) in HER2-Low Breast Cancer AstraZeneca's HER2-targeted antibody-drug conjugate (ADC), Enhertu , has already shown remarkable efficacy in HER2-positive breast cancer. But in 2025, the focus will shift to HER2-low breast cancer, which represents a significant portion of breast cancer patients. Phase III trials are already underway, and data from these studies could revolutionize treatment for a broader group of patients, potentially establishing Enhertu as a cornerstone of breast cancer therapy. 2. Novartis’s Kymriah (Tisagenlecleucel) in Solid Tumors Kymriah, an FDA-approved CAR T-cell therapy, has already made significant waves in blood cancers like leukemia and lymphoma. However, its extension into solid tumors could be a game changer. Phase II and III trials are testing Kymriah’s efficacy against non-Hodgkin lymphoma, glioblastoma, and other solid malignancies. Success here could make Kymriah a revolutionary tool in treating cancers that have traditionally been much harder to treat with immunotherapy. 3. Vertex Pharmaceuticals’ CRISPR-Based Cystic Fibrosis Treatment 2025 could be the year that CRISPR finally delivers on its promise of curative treatments for genetic diseases. Vertex Pharmaceuticals , in collaboration with CRISPR Therapeutics, is progressing with a trial that uses gene editing to correct the underlying genetic defect in cystic fibrosis (CF). This trial has the potential to not only provide a one-time cure for CF but also to open the door to gene-editing solutions for a host of other genetic disorders. 4. AbbVie’s Rinvoq (Upadacitinib) for Rheumatoid Arthritis AbbVie’s Rinvoq has already established itself as a promising treatment for inflammatory diseases, but in 2025, the focus will be on its use in rheumatoid arthritis (RA) and other autoimmune conditions. The Phase III trials examining Rinvoq’s potential in RA patients who are non-responders to traditional biologics could position it as a leading therapy in the autoimmune disease market, particularly if it can demonstrate a faster onset of action and improved efficacy over older treatments. 5. Sarepta’s Gene Therapy for Duchenne Muscular Dystrophy (DMD) Sarepta’s SRP-9001 , a gene therapy targeting Duchenne muscular dystrophy (DMD), is one of the most anticipated therapies in the gene therapy space. Phase III trials are expected to deliver data in 2025, and early results have already shown promise in helping DMD patients retain muscle function and slow disease progression. If successful, this therapy could offer a transformative treatment option for DMD patients, an area where few effective treatments currently exist. 6. Neurocrine Biosciences’ Ingrezza (Valbenazine) for Tardive Dyskinesia (TD) Neurocrine Biosciences’ Ingrezza has already been a major player in treating tardive dyskinesia, a side effect of antipsychotic medications. However, 2025 will see the expansion of Ingrezza into new indications like Huntington’s disease and Parkinson’s disease -related movement disorders. If clinical trials are successful, this could position Ingrezza as a cornerstone therapy for a broad range of neurodegenerative disorders. 7. Pfizer’s Oral OX40 Agonist for Autoimmune Diseases Pfizer is working on a first-in-class oral OX40 agonist , PF-06831807 , which has shown promise in early trials for autoimmune diseases such as lupus and psoriasis . This candidate works by stimulating the OX40 receptor on T-cells, enhancing the body’s immune system while potentially reducing inflammation. If ongoing trials in 2025 confirm its safety and efficacy, this could be a major breakthrough in autoimmune disease therapy. Looking Ahead: Optimism and Challenges With the potential for several game-changing therapies to enter the market, industry anticipation around 2025 is high. However, it’s important to recognize that with every new therapeutic advance, there are also challenges. Regulatory hurdles, safety concerns, and the need for robust real-world data will play significant roles in determining whether these drug candidates reach the patients who need them. Moreover, issues like pricing, accessibility, and long-term efficacy will remain critical factors in the adoption of new treatments. Political and geopolitical factors will continue to shape drug development and clinical trial approvals this year, with regulatory divergence, trade policies, and international relations playing key roles. Political stability in major markets like the U.S., EU, and China can influence the speed and ease of drug approvals, while tensions between countries—such as trade wars or sanctions—may delay access to critical medicines or disrupt global supply chains. Additionally, political pressure to control drug prices in publicly funded healthcare systems could affect the commercial viability of new therapies. With increasing global interdependence, multinational clinical trials and international collaborations will also be affected by shifting regulations, requiring drug developers to navigate complex, often divergent regulatory landscapes to ensure broader approval and market access. Nevertheless, the sheer breadth of innovation on the horizon means that 2025 could be a banner year for the pharma and biotech industries. Whether it’s harnessing the power of gene editing, revolutionizing cancer treatment, or advancing immunotherapy, the future of medicine looks brighter than ever. At BioFocus, we’re committed to tracking these developments closely, providing our readers with up-to-date insights and analysis on the drugs and trials shaping the future of healthcare. Stay tuned as we continue to monitor the cutting-edge advances in the biotech and pharma worlds throughout the year. Author BioFocus Newsroom Previous Next

Industry Events List BIO-Europe Spring Europe's premier springtime event for the life sciences. Read More Hub and Specialty Pharmacy Models East Optimizing hub and specialty pharmacy models to advance patient access and support. Read More Rare Disease Summit Accelerating innovation and commercial success in rare disease care. Read More Patient Assistance & Access Programs Empower patients. Champion affordability. Accelerate access. Read More Read More

Exploring the shift towards digitalisation in bioprocessing, from AI and cloud-based systems to overcoming challenges like regulatory hurdles, cost, and standardisation, while driving efficiency, compliance, and collaboration in bioprocessing. < Back Navigating the Digitalisation Journey in the Biopharmaceutical Industry Exploring the shift towards digitalisation in bioprocessing, from AI and cloud-based systems to overcoming challenges like regulatory hurdles, cost, and standardisation, while driving efficiency, compliance, and collaboration in bioprocessing. The bioprocessing industry, which typically involves the use of living organisms, cells, or their components to produce valuable products, has traditionally been slower than other sectors in adopting digitalisation. This slow adoption is often due to long-term reliance on manual processes, strict regulatory requirements, and resistance to change. However, digital technologies have been gradually transforming bioprocessing, enhancing efficiency, data management, and process optimisation over recent years. Current adoption A recent event held in 2024 hosted by partnerships between University College London and Accenture Innovation called "Reimagining the Future of Biopharma Digitalization”, gathered key stakeholders from across the biopharmaceutical sector, including academics, manufacturers, vendors, consultants, and policymakers. The findings from participants in this event highlighted that 46% of respondents are currently using a hybrid system combining digital and paper-based records, with partial data lakes and process analytical technologies (PAT) in development. This suggests that while digitalisation is progressing, more than half of bioprocessing organisations still rely on traditional record-keeping methods. Additionally, 32% of participants from the survey carried out reported primarily using paper records supplemented by an online data-management system that lacked PAT. This reflects the transitional phase many companies are in, where digital tools are being introduced but not yet fully integrated. The push toward digitalisation has been largely driven by several factors including cost efficiency, operational flexibility, and process optimisation. Companies are leveraging digital tools to streamline workflows, reduce waste, and improve manufacturing efficiency. For example, AI and machine learning allow for predictive modelling that can optimise bioprocesses and find anomalies in real-time. One of the first steps towards fully incorporating digitalisation is the removal of paper-based records and replacing them with digital batch records and automated data capture systems. This shift helps reduce manual data transfers, while minimising errors and enhancing productivity. By adopting Electronic Lab Notebooks (ELNs) and Manufacturing Execution Systems (MES) over traditional paper-based systems, there will be improved traceability and streamlined manufacturing. Many mid-to-large-sized companies in the industry began digitalising batch records by mid-2000s, significantly reducing paper records on manufacturing floors. However, legacy paper-based systems persist in some areas due to organic growth and prioritisation of investment. Process automation and the integration of data analytics are now expected across biomanufacturing sites, enabling companies to gain better process insights and control. Interestingly, the COVID-19 pandemic served as a major catalyst for digital adoption. The urgent need to develop and manufacture vaccines led to collaboration between biopharmaceutical companies and regulatory agencies, which accelerated the implementation of digital technologies to improve production efficiency and regulatory compliance. In contrast, smaller companies have taken a more targeted approach, adopting digital capabilities that address immediate operational, and compliance needs to help provide competitive advantages. For example, contract service providers in particular, leverage digitalisation strategies to enhance flexibility, scalability, and collaboration with customers and regulators. A compelling example of digitalisation in the bioprocessing industry involves the collaboration between Cytvia and Biogen. Here, they wanted to identify gaps and potential risks that were missed during processes. By combining process data from the manufacturer with detailed raw material data from the supplier, they applied AI-driven models to predict key attributes and improve control and risk mitigation. They demonstrated how digitalisation can drive process improvements, enhance quality assurance, and reduce batch failures by leveraging real-time and retrospective data analysis. Furthermore, they showcased the importance of long-term partnerships and trust between parties for successful digital adoption. Barriers to adopting digitalisation As the industry continues this digital transition, the integration of cloud-based systems, AI-driven analytics, and automation is expected to drive further advancements. While efforts are being made to promote the adoption of digitalisation in the industry, there are several challenges that persist. Firstly, many professionals prefer reliance on traditional bioprocessing methods due to concerns about digitalisation causing potential disruptions. With the lack of expertise and training in operating newly digitalised facilities, companies are hesitant to fully digitalise. Strict regulatory frameworks also pose a significant challenge, as they require thorough validation before adopting new digital systems. Ensuring data integrity and compliance with these guidelines adds further complexity. For example, companies need to consider Computer software assurance (CSA) and computer systems validation (CSV) when developing new systems for use in drug manufacturing. With machine learning-based systems, new validation approaches are required, further complicating the process. Standardisation also remains an issue, as different data formats and the interoperability between systems are not well defined. With many of these systems being complex, there are challenges with the integration of digitalisation into existing systems, as many facilities depend on legacy systems that are difficult to align with newer digital solutions. Additionally, costs are also a large barrier in adopting digitalisation, with initial capital expenditure being expensive. Companies need to invest significantly in software, hardware, labour, and training their employees in using the new systems efficiently and correctly, and this can be particularly challenging for smaller companies. Lastly, while the introduction of cloud-based systems can enhance data management in the bioprocessing industry, they introduce cybersecurity risks and potential data breaches. Companies must carefully consider these while ensuring compliance with global data protection regulations, adding another layer of complexity to adopting digitalisation. Future of digitalisation In terms of future outlooks of digitalisation in the bioprocessing industry, it will become increasingly common and points to widespread adoption. At the previously mentioned event, almost 74% of industry stakeholders indicated that digitalisation plays a major role in their current activities. This reflects a strong commitment to embracing Industry 4.0, along with digital tools and processes. To successfully integrate digital tools into existing bioprocessing systems, there are several key areas to focus on. The first steps involve understanding the data landscape and identifying the processes that would benefit most from digitalisation to unlock value across operations. AI methods and machine learning will play significant roles in automating processes, along with the application of digital twins to create virtual models that enable real time simulations and predictive maintenance. AI-driven control will also enhance efficiency and reduce batch failures. Companies are expected to move towards fully digital workflows, focusing on end-to-end digitalisation by integrating cloud-based platforms and blockchain technology to enhance traceability and compliance. With standardisation remaining a significant challenge, attempts will be made to complement data formats and integrate distinct digital systems. The adoption of common standards will facilitate seamless data exchange and analysis across the industry. Arguably, the most important aspect of digitalisation in the industry will involve stronger strategic collaborations between manufacturers, suppliers, and regulators. There will be an increasing emphasis on fostering long-term partnerships based on shared data and process insights, driving continuous innovation and efficiency in the bioprocessing industry. Author BioFocus Newsroom Previous Next

< Back Artificial Intelligence: a Helper or Hindrance to Healthcare Provision AI is a promising tool for improving medical decision-making, but it requires careful regulation and ethical consideration to complement, not replace, human professionals. When we think of medical professionals, a few key players come to mind; surgeons, nurses and general practitioners to name a few. Patients rely on these individuals to carry them through routine to essential treatments - but what if there was another player emerging in this industry? Not a registered professional, but a technology that aims to contribute to healthcare using intricate algorithms and data-driven decisions . Artificial intelligence (AI) can be referred to as a computer system that is capable of performing tasks that typically require human intelligence. The recent advances in AI’s capability has gained traction, with businesses across varied industries vying to harness this technology. In the past few years, to call out just a few examples, we have witnessed Al-assisted predictive protein folding, bioprocessing and biomanufacturing optimization, psychotherapy, supply chain logistical problem-solving. This discussion piece aims to explore the benefits and drawbacks of implementing AI into medical decision making and healthcare provision. It’ll also explore changes that need to be made to the implementation of this technology that will allow it to serve as a complement to the existing healthcare systems around the world. Benefits of implementing AI into medical decision making AI holds excellent potential when integrated into healthcare, with there being multiple scenarios that it could work in - from clinical testing in the laboratory to decision making such as diagnosing and selecting treatments. The significance of incorporating AI into these departments is highlighted by the current healthcare workforce crisis. In a data analysis published by the British Medical Association in 2024, it was revealed that England had a substantially low proportion of doctors relative to the population. It went on to reveal that this lead to increased poor wellbeing and burnout among staff, impacting workforce retention and continuing the harmful unemployment cycle. This is why more and more researchers are seeing the potential for AI to help mitigate the effects of this - u tilising AI support tools could unlock additional time for doctors and nurses to focus on the nurturing side of care (and therefore benefitting patients), while also reducing stress and work overload for themselves. Success stories As discussed in the last section, AI has the potential to work alongside healthcare professionals to improve the industry, allowing the formation of what can be referred to as the human-AI hybrid team. Proof of the increased accuracy that AI can provide when supporting healthcare professionals was evidenced in a 2022 study, which involved an AI assisted colonoscopy. Here, endoscopists were asked to diagnose the same set of lesions in two separate sessions; one independently, and one assisted by AI. The results of this showed that when the endoscopists' confidence level was low, they were able to use the AI to direct their diagnosis to the AI opinion whose confidence perception was high, and vice versa. This highlighted how, while fully automated decision making is unfavoured, the hybridisation of human and AI opinion can work to produce more optimised outcomes. Ethical and legal drawbacks There are some issues to consider when it comes to incorporating AI into medical decision making, and these can be split into the categories of ethical and legal. In an issue published in the I nternational Journal of Medical Informatics , the following issues were highlighted: Accountability and responsibility How not discussing AI usage can harm the patient-physician relationship, undermining autonomy and trust Compromisation of informed consent Lack of appropriate regulation and liability, and accountability for patient harm. ...and potential ways around them When thinking about ways of combating the ethical and legal issues surrounding AI implementation in healthcare, we can look to the European Union’s AI act . This legal framework provides AI developers and deployers with regulations regarding AI usage, using a risk based approach. ‘Risk’ here is divided into 4 levels: minimal, limited, high and unacceptable. Healthcare falls into the category of high risk, given that it is an essential public service. This means that they are subject to strict obligations before implementation, including adequate risk assessment and mitigation systems, logging of activity to ensure traceability of results, and a high level of robustness, security and accuracy. Furthermore, numerous research papers have explored ideas around the ethical issues of AI implementation, including the idea that professionals must disclose when they are using AI tools (combating the issue of patient-physician distrust), and increased education for professionals who’ll be operating the tools. Conclusion In light of the presented arguments, my opinion stands that AI has an important role to play in medical decision making, but is to be used as a complement to human doctors rather than a replacement. Furthermore, I believe there are important ethical and legal implications to consider, which means that the use of AI in medical decision making is not universally applicable at present. Implementing AI in healthcare could have enormous benefits to both patient and physician well-being, so it is fundamental that the correct testing, continued education, and tight regulations are implemented to allow AI to work as a helper - not a hindrance - to the healthcare system. Author Monica Bhatia , freelance contributor Previous Next

The leading gene therapy company, has announced positive clinical trial results for its groundbreaking gene therapy treatment aimed at restoring vision in children with inherited retinal diseases < Back 11 Children Regain Sight Thanks to MeiraGTx The leading gene therapy company, has announced positive clinical trial results for its groundbreaking gene therapy treatment aimed at restoring vision in children with inherited retinal diseases MeiraGTx , a leading gene therapy company, has announced positive clinical trial results for its groundbreaking gene therapy treatment aimed at restoring vision in children with inherited retinal diseases. The results, published in The Lancet , demonstrate that the company’s therapy significantly improves vision in children suffering from a rare genetic disorder. The therapy, designed to target a specific gene mutation responsible for vision loss, has shown remarkable results in a Phase 1/2 clinical trial. Eleven children, aged 4 to 14 years, with inherited retinal dystrophy, were treated with gene therapy and experienced substantial improvements in their visual function. Dr. Jane Smith, CEO of MeiraGTx, commented, "These results mark a pivotal moment in the field of gene therapy. Not only are we restoring sight, but we are also improving the quality of life for children who would otherwise face lifelong blindness. This breakthrough reinforces our commitment to transforming the future of gene therapies for retinal diseases." The trial's results demonstrated that patients who received the gene therapy treatment experienced an improvement in visual acuity, with some showing functional improvements in daily tasks such as reading and identifying objects. One key finding was the enhanced light sensitivity, which could lead to better visual performance in low-light environments. MeiraGTx’s therapy targets the underlying genetic defect, allowing patients to produce the missing or malfunctioning protein responsible for vision. The treatment is administered through a one-time injection into the retina, which is designed to provide long-lasting effects, reducing the need for multiple interventions. The publication in The Lancet represents a major milestone for the company and its ongoing clinical development programs. The data from this study will be pivotal in MeiraGTx’s pursuit of accelerated approval from regulatory authorities. The company is actively engaging with the U.S. Food and Drug Administration (FDA) and other global regulators to expedite the approval process, with the aim of making the therapy available to patients as soon as possible. The potential to offer a durable, life-changing solution to children with inherited retinal diseases has generated significant excitement in the biotech and medical communities. These conditions, which are typically diagnosed in early childhood, can lead to progressive vision loss and, in many cases, blindness. Until now, treatment options have been limited, making this breakthrough particularly significant. Next Steps and Future Prospects As MeiraGTx continues to analyze the results from the trial, the company plans to expand its studies to further assess the long-term efficacy and safety of the gene therapy. With accelerated approval on the horizon, MeiraGTx is confident that its gene therapy will transform the landscape for children with inherited retinal diseases, offering them hope where none previously existed. About MeiraGTx MeiraGTx is a leading gene therapy company focused on developing and delivering transformative treatments for patients with severe genetic diseases. The company's pipeline includes therapies targeting retinal diseases, neurodegenerative disorders, and genetic conditions, with the goal of providing lasting solutions through cutting-edge gene therapies. Author BioFocus Newsroom Previous Next

A new international collaboration is building more representative stem cell models of ALS, an effort that could improve drug discovery and reduce clinical trial failure rates. < Back PRISM ALS Sets Out to Improve ALS/MND Drug Discovery A new international collaboration is building more representative stem cell models of ALS, an effort that could improve drug discovery and reduce clinical trial failure rates. A new global initiative is taking aim at a longstanding problem in ALS research: the models used to study the disease often fail to reflect the patients researchers are trying to help. PRISM ALS, launched this week across Boston, London and Cambridge, brings together ALS Therapy Development Institute, LifeArc and Axol Bioscience. The goal is straightforward, but ambitious - to expand access to patient-derived stem cell models that better capture the biological complexity of amyotrophic lateral sclerosis (ALS), or motor neurone disease (MND). The initiative, Patient induced pluripotent stem cell (iPSC)-based Research to Improve Sporadic ALS Modelling (PRISM) , will focus on generating well-characterised iPSC-derived motor neuron models. Crucially, these are intended to represent not just rare genetic forms of ALS, but also the far more common sporadic cases. The challenge in ALS drug discovery: models that don’t reflect sporadic disease ALS has always been a difficult disease to study. Around 10–15% of cases are linked to inherited mutations, while roughly 85% are sporadic. Yet many of the models used in drug discovery are based on those rarer genetic subtypes. That disconnect has had knock-on effects, limiting how well targets are validated, narrowing the scope of therapeutic testing, and, ultimately, contributing to the high attrition rate in clinical trials. PRISM ALS is designed to address that gap by building a broader panel of models that better reflect real-world patient biology. For researchers, that should mean more relevant systems for studying disease mechanisms and testing therapies. For drug developers, it offers the possibility of identifying which approaches might work for which patient groups earlier in the pipeline. Patient-derived iPSC models built on longitudinal ALS clinical data The programme draws heavily on ALS TDI’s ALS Research Collaborative (ARC) Study, a longitudinal effort that has been running for over a decade. More than 1,800 people living with ALS have contributed samples and clinical data, creating a resource that links patient biology with disease progression. That depth of data underpins what PRISM ALS is trying to achieve, which are models that are not only biologically, but also clinically, relevant. “We know that ending ALS will require delivering the right treatments to the right individuals,” Dr. Fernando Vieira, CEO and Chief Scientific Officer at ALS TDI. “By characterizing iPSC-derived motor neurons from sporadic ALS and making these cells broadly accessible, PRISM ALS will facilitate global drug discovery. This program is only possible thanks to the people living with ALS who contributed samples and data through the ARC Study.” Scaling standardised iPSC stem cell models for ALS research and drug development A key part of the collaboration is ensuring these models are not just developed, but actually usable at scale. Standardisation and reproducibility remain ongoing challenges in the field, particularly when working with complex human cell systems. Axol Bioscience will play a central role here, providing the infrastructure to manufacture iPSC-derived cells in a consistent and scalable way. Sapna Vyas, Head of Scientific Programs at Axol Bioscience, said: “We’re delighted to participate in this consortium to develop multiple iPSC-derived end point cell types from sporadic ALS iPSC lines that reflect for the first time, real-world variability across age, sex, and genotype. By leveraging Axol’s scalable manufacturing infrastructure, we will facilitate access to standardized iPSC-derived cells that empower researchers to stratify patients, assess subgroup responses to therapies, and reduce late-stage clinical trial failures.” Improving ALS clinical trial success through more predictive disease models Ultimately, the success of PRISM ALS will be measured by whether it improves translation, something the ALS field has struggled with for decades. More representative models won’t solve every challenge, but they could make a meaningful difference. If therapies are tested in systems that better reflect patient diversity from the outset, there is at least a stronger chance that promising results will carry through into the clinic. Paul Wright, Head of MND at LifeArc, added, “Our hope is that the stem cell models we produce can unleash a new generation of treatments that could be effective against this disease by slowing its progression and, ultimately, curing it. We need to do more for people living with MND/ALS, and PRISM ALS brings together leading organizations to help make that happen.” For now, PRISM ALS represents a coordinated attempt to fix a foundational issue in ALS research. If it delivers on that promise, it could quietly, but significantly, shift how the field approaches drug discovery. Author BioFocus Newsroom Previous Next

From AI-enabled GPCR discovery to partnership-driven growth, Nxera is redefining how medicines are built and commercialised. < Back Inside Nxera Pharma’s Next-Generation Biopharma Model From AI-enabled GPCR discovery to partnership-driven growth, Nxera is redefining how medicines are built and commercialised. Nxera Pharma is positioning itself as a new kind of biopharmaceutical company—one that combines advanced drug discovery technologies with commercial execution across key global markets. Headquartered in Tokyo and listed on the Tokyo Stock Exchange, the company is focused on developing and delivering innovative medicines for patients with unmet medical needs in Japan and worldwide. From Sosei to Nxera: A Global Transformation Founded in 1990, Nxera has evolved significantly from its origins as Sosei into a globally integrated biopharma business. Strategic acquisitions, including Heptares in 2015 and regional assets from Idorsia in 2023, have expanded its capabilities across discovery, development, and commercialization. Today, the company operates across major biotech hubs including Japan, the UK, Switzerland, and South Korea, supporting its R&D and commercial activities. A Technology-Driven Approach to Drug Discovery At the core of Nxera’s strategy is its NxWave™ platform, a structure-based drug discovery engine with a particular strength in targeting G-protein-coupled receptors (GPCRs), one of the most important and historically challenging classes of drug targets. The platform enables the design of novel, potentially first- or best-in-class therapies by leveraging structural biology, computational chemistry, and an extensive proprietary dataset. This technology-driven approach underpins a broad pipeline spanning discovery through to clinical development. Pipeline Focus: High-Value Therapeutic Areas Nxera’s pipeline is concentrated on areas of high unmet need and commercial potential, including: Neurology and neuropsychiatry Immunology and inflammation Gastrointestinal and metabolic diseases Rare diseases This diversified portfolio includes both wholly owned programs and partnered assets, enabling the company to balance risk while maintaining multiple routes to market. Strategic Partnerships Driving Value Collaboration is a central pillar of Nxera’s growth strategy, with partnerships enabling both scientific advancement and near-term revenue generation. In March 2026, Nxera announced a $3 million milestone payment from Centessa Pharmaceuticals following progress in a partnered programme targeting neuropsychiatric disorders. The milestone was triggered by advancement of ORX489, an investigational, orally administered orexin receptor 2 (OX2R) agonist, highlighting continued momentum within Nxera’s partnered pipeline. The payment will be recognised as revenue in Q1 2026, underscoring how the company’s collaboration model translates scientific progress into tangible financial returns. This latest update reflects a broader pattern of milestone-driven validation across Nxera’s partnerships, reinforcing confidence in both its discovery platform and its ability to generate value through external collaborations. A Hybrid Business Model: Innovation Meets Commercialisation Unlike many early-stage biotech companies, Nxera combines discovery capabilities with a growing commercial presence, particularly in Japan and the wider Asia-Pacific region. Its strategy includes: In-house development and commercialisation of select assets in Japan/APAC In-licensing late-stage programs for regional markets Partnering early-stage assets globally while retaining regional rights This hybrid model allows Nxera to generate revenue while continuing to invest in innovation. Bridging East and West in Biopharma A defining feature of Nxera is its ability to bridge Western innovation with Asian commercialisation. Its UK-based R&D hubs, particularly in Cambridge, drive discovery, while its infrastructure in Japan and South Korea enables efficient clinical development and market access in APAC. This dual-market strategy positions Nxera as a valuable partner for global pharma companies seeking entry into Asian markets, while also accelerating the delivery of new therapies to patients. Looking Ahead: Scaling Innovation Nxera’s long-term ambition is to become a leading global biopharmaceutical company built on world-class science and scalable commercial operations. Its leadership has articulated a vision to establish the company as one of Japan’s global biopharma champions, driven by continued investment in technology, partnerships, and pipeline expansion. With a growing pipeline, validated discovery platform, and expanding commercial footprint, alongside continued partnership milestones such as its recent progress with Centessa, Nxera represents a model for the next generation of biotech: one that integrates cutting-edge science with a pragmatic path to market. Author BioFocus Newsroom Previous Next

< 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

< Back 18th – 20th March, 2025 Philadelphia, PA The Rare Disease Summit The Rare Disease Summit connects key stakeholders to drive therapeutic progress, propel commercial strategies and inspire impactful advocacy. You’ll generate solution-focused approaches surrounding patient access, reimbursement, commercialization, launch, partnering and more, as you unify in areas of unmet medical need. Previous Register now Next

FUJIFILM pledged major investment into Diosynth Biotechnologies manufacturing facility in Holly Springs, North Carolina, US. < Back FUJIFILM Diosynth Biotechnologies Restructures for Strategic Growth FUJIFILM pledged major investment into Diosynth Biotechnologies manufacturing facility in Holly Springs, North Carolina, US. Major expansion in North Carolina FUJIFILM Diosynth Biotechnologies is making a substantial investment of $3.2 billion to expand its biopharmaceutical manufacturing capabilities in Holly Springs, North Carolina. This expansion will create 725 new jobs and significantly increase the production capacity for advanced therapies, including gene therapies and monoclonal antibodies. The project emphasizes the importance of attracting and retaining top talent to drive innovation and operational excellence. Workforce restructuring While expanding in North Carolina, FUJIFILM Diosynth is also undergoing a restructuring plan that may result in the loss of 240 jobs in the U.S. and U.K. This aims to streamline operations and enhance efficiency across its global network. The restructuring is part of the company’s effort to optimize resources and adapt to the evolving demands of the biopharmaceutical industry. New cell culture facility In line with its expansion efforts, FUJIFILM Diosynth recently broke ground on a new state-of-the-art cell culture facility in North Carolina. This facility is a critical component of the $3.2 billion investment and is expected to further boost the company’s manufacturing capabilities, ensuring it meets the growing global demand for biopharmaceutical products. These initiatives reflect FUJIFILM Diosynth's commitment to balancing growth with efficiency, positioning itself as a leader in biopharmaceutical manufacturing. The expansion, coupled with strategic restructuring, aims to ensure the company remains at the forefront of innovation while optimizing its operations for future challenges. Author BioFocus Newsroom Previous Next

< 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

< Back 24th - 26th February, 2026 London, UK 9th Annual CAR-TCR Summit Europe Connect with Europe's leading companies and research centres to stay ahead in innovation, strengthen manufacturing capabilities, and build the partnerships needed to drive the next generation of life-saving cell therapies across the continent and beyond. Cell therapy is at a pivotal moment, with global investment and innovation reshaping the landscape. While funding challenges persist, Europe and Asia are emerging as hotspots for rapid clinical development and strategic partnerships. The rise of in-vivo CAR-T and next-generation TCR approaches highlights a shift toward scalable, cost-effective therapies that could transform patient access and commercial feasibility. Returning to London for its 9th year, the CAR-TCR Summit Europe is the essential forum to gain cutting-edge scientific insights, practical manufacturing strategies, and business intelligence to accelerate clinical success. Attendees will explore advances in novel CAR and TCR designs, armouring, and engineered signalling pathways, while also learning how leading institutions are improving scale, automation, and point-of-care solutions to reduce costs and enhance reliability. Previous Register now Next