ArgusEye releases AguaOne, a breakthrough in downstream bioprocess development. < Back ArgusEye introduces AugaOne to accelerate bioprocess development ArgusEye releases AguaOne, a breakthrough in downstream bioprocess development. ArgusEye , a provider of innovative sensor solutions for real-time monitoring of biological systems, today announced the introduction of the AugaOne™ sensor system. AugaOne is the first product in the Company’s sensor system platform, Auga™, and is tailored to accelerate downstream monoclonal antibody (mAb) process development by providing specific real-time and automated in-line data with high sensitivity, without requiring sample pre-treatment. Utilising a nanoplasmonic sensing technology platform, AugaOne offers specific real-time monitoring of critical quality attributes and process parameters to determine the optimal process. Adapted for downstream processing, AugaOne facilitates applications such as in-line detection of product breakthrough, as well as the automated control of a multi-column chromatography setup. Its highly flexible, modular design enables the system to be easily and quickly integrated into existing equipment, increasing process efficiency and intensifying process development. Primarily developed to support biopharmaceutical scientists in the detection and quantification of mAbs, AugaOne can handle complex samples such as cell culture and plasma, delivering robust, accurate data independent of matrix effects, cells, and temperatures, with the plan to support process development of other biomolecules in the future. Additionally, its in-line monitoring capabilities greatly reduce the need for time-consuming, manually performed off-line analysis, significantly reducing production lead times. Erik Martinsson, CEO and Co-Founder, ArgusEye, said: Biopharmaceutical development relies heavily on time-consuming and labor-intensive off-line analysis for quality control, with limited products available for specific in-line or in-line monitoring. Our Auga™ platform aims to transform the bioprocessing workflow, enabling rapid, accurate data generation in real-time. Through the introduction of our new AugaOne sensor, we are accelerating mAb process development workflows, taking us a step further in our aim to support the rapid, continuous and cost-effective manufacturing of biologics to bring lifesaving treatments to the market.” ArgusEye overview ArgusEye develops and delivers innovative sensor systems for real-time monitoring and analysis of biological systems and processes. The patented technology, born from extensive academic research, utilizes nanoplasmonic sensing combined with fiber optics. By enabling specific real-time in-line analysis, previously confined to off-line analytics, ArgusEye aims to transform how biologics are developed and produced. Author BioFocus Newsroom Previous Next

< Back Hookworm Therapy: Advancing Type 2 Diabetes treatment? New research looks at the potential of hookworms to offer improvements in insulin resistance among volunteers at risk of type 2 diabetes. Nature Communications , in its summer edition of 2023, brought forth ground-breaking research that could potentially revolutionise the treatment and prevention of metabolic diseases , particularly type 2 diabetes. Researchers utilised live hookworm infections which resulted in significant improvements in insulin resistance among volunteers at risk of type 2 diabetes, igniting enthusiasm for a larger-scale international trial involving hookworm therapy. When hookworms come to mind, the typical association is tiny parasitic worms that can cause illness. Nevertheless, since the 70s scientists have been investigating helminth (worm) infections as a possible strategy to address a range of ailments encompassing allergies, autoimmune diseases, and now, metabolic conditions. The theory behind this approach is rooted in the hygiene hypothesis, which suggests that the decline in parasitic worm infections, due to improved sanitation, may have contributed to rising rates of metabolic and autoimmune diseases. Metabolic Diseases and Type 2 Diabetes: A Growing Concern Metabolic diseases encompass a range of health conditions often characterised by inflammatory immune responses. An example of a metabolic disease is type 2 diabetes—a chronic ailment characterised by insulin resistance and elevated blood sugar levels. The prevalence of type 2 diabetes is increasing globally, with over half a billion adults already diagnosed. In the UK, more than 4.9 million individuals are presently diagnosed with diabetes, and the vast majority of them (around 90%) have type 2 diabetes. This growing health concern calls for innovative preventative approaches to reduce the burden on individuals and healthcare systems. The Hygiene Hypothesis: Why hookworms? In regions where parasitic worms are endemic, the prevalence of type 2 diabetes and other metabolic diseases tends to be lower. However, the introduction of deworming programs in these areas has been followed by an increase in metabolic disease rates. Interestingly, when people are ‘dewormed’, their insulin resistance tends to surge . Insulin resistance is a critical factor in regulating blood sugar levels and plays a role in the development of metabolic issues like type 2 diabetes and cardiovascular disease. However, it is crucial to acknowledge that the rise in diabetes after interventions in these areas may result from lifestyle changes, such as a reduction in exercise or increased fast food consumption. Figure 1: Effect of deworming on frequency of metabolic disease Hookworms and Immune Response: The connection Low-grade, systemic inflammation is a well-established contributor to insulin resistance. A biased type-1 immune response can worsen insulin resistance by targeting components in the insulin-signalling pathway. Therefore, to combat insulin resistance in metabolic diseases, maintaining a balanced inflammatory response by favouring a type 2 or regulatory immune response, is essential to halt the detrimental cascade of inflammation. Helminth infections are associated with type 2 immune responses and alterations to the gut microbiome . Studies involving worm infections in animal models have demonstrated improvements in metabolic parameters, type 2 immune responses, and changes in the gut microbiome, but no human trials have been conducted. Until now. The study: A Pioneering Human Trial The world-first human trial conducted at James Cook University involved experimental hookworm infection in 40 participants, all at risk for type 2 diabetes and cardiovascular disease. In a two-year, randomised, double-blinded clinical trial, 27 participants were infected with either 20 or 40 Necator americanus larvae, while 13 received placebos. In order to induce a placebo effect, or in this instance, a nocebo effect, researchers employed chilli pepper sauce to simulate the sensation of hookworm larvae penetrating the arm. This approach is commonly referred to as a sham intervention, as it led the control group participants to believe that they had also been exposed to hookworms. The primary aim of this phase 1 study was to confirm the safety of this innovative therapy. The study showed that adverse events were more frequent in the hookworm-treated group, mainly manifesting as gastrointestinal symptoms, but completion rates were comparable to the placebo group. Remarkably, the study found that low hookworm infection was associated with improvements in insulin resistance and fasting blood glucose, with all but one participant opting to keep their hookworms after the trial. Notably, the consistent monitoring of dietary and physical activity habits did not reveal any discernible changes between the infected and control groups. Figure 2: Summary of study findings Promising Results: The Path Ahead The research offers a promising insight into the potential of live hookworms for improving metabolic health. While these results are encouraging, it is essential to recognize that this is just the beginning. Larger clinical trials are needed to determine the optimal dosage of hookworms and to clarify the mechanisms behind their positive effects on metabolic health. Researchers hope to uncover the specific molecules released by hookworms that suppress inflammation and improve metabolism. In the quest to harness the power of hookworms, scientists are exploring protein-based treatments that mimic the effects of live worms, potentially providing a more palatable and controlled alternative for patients. However, many questions remain unanswered, including how hookworms precisely impact human metabolism, whether through immune system manipulation and/or weight loss. Closing Thoughts: Are we hooked? Before rushing to embrace hookworms as a solution for metabolic and autoimmune diseases, it's vital to remember that these results are preliminary and come with some caveats. Individual responses may vary depending on the dosage and follow-up protocols. This research paves the way for future investigations, offering new hope in the fight against type 2 diabetes and metabolic diseases. As the scientific community delves deeper into the potential of hookworms, we may unlock new possibilities for improved metabolic health and overall well-being. Author Petra Gudelj , freelance contributor Previous Next

First advanced solid tumour patient dosed in a phase I clinical trial assessing preliminary efficacy of bispecific antibody, NWY001. < Back Chipscreen NewWay Biosciences Initiates Phase I Clinical Trial First advanced solid tumour patient dosed in a phase I clinical trial assessing preliminary efficacy of bispecific antibody, NWY001. Chengdu Chipscreen NewWay Biosciences Co., Ltd. (NewWay) has marked a significant milestone with the initiation of the phase I clinical trial for NWY001, the world's first PD-1/CD40 bispecific antibody to enter clinical trials. The dosing of the first patient occurred on January 5, 2024, at Sun Yat-Sen University Cancer Center in China. NWY001 aims to transform "cold" tumors into "hot" tumors, potentially enhancing cancer patients' sensitivity to PD-(L)1 immune checkpoint inhibitors, particularly those resistant to PD-(L)1 antibody monotherapy. NWY001's unique mechanism involves the synergistic targeting of PD-1 and CD40 pathways, activating the CD40 pathway in a PD-1 dependent manner. This approach is designed to mitigate the common toxicities associated with CD40 agonistic monoclonal antibodies. The phase I clinical trial is a multi-center, non-randomized, open-label study focusing on evaluating safety, tolerability, preliminary efficacy, pharmacokinetic properties, and potential biomarkers associated with NWY001 treatment in patients with advanced solid tumors. Dr. Bin Liu, Scientific Director of Chipscreen NewWay, expressed gratitude to Sun Yat-Sen University Cancer Center, Chipscreen's clinical and related teams, and the enrolled patients and their families. The initiation of the trial is expected to address the ineffectiveness or toxicity challenges posed by PD-(L)1 immune checkpoint inhibitor monotherapy and combination therapies involving CD40 agonists. In a related development, on February 27th, 2023, Biocytogen Pharmaceuticals' subsidiary, Eucure (Beijing) Biopharma Co., Ltd., entered an exclusive licensing agreement with Chipscreen NewWay Biosciences for the clinical development and commercialization of bispecific antibody YH008 (NWY001) in Greater China, encompassing Mainland China, Hong Kong, Macau, and Taiwan. Chipscreen NewWay, a biotech company affiliated with Chipscreen, is dedicated to developing large molecules and novel therapeutic modalities, with a focus on oncology and autoimmunity. The company has built an extensive molecular R&D center in Chengdu Hi-Tech Zone, employing an experienced team for antibody and ADC R&D projects. Chipscreen, established in 2001, is a biopharmaceutical company specializing in original drug development across various disease areas, including oncology, metabolic diseases, autoimmune diseases, central nervous system disorders, and antiviral therapies. As the world eagerly awaits the results of the NWY001 phase I clinical trial, this groundbreaking PD-1/CD40 bispecific antibody represents a promising advancement in cancer treatment, offering hope for enhanced therapeutic outcomes in patients with advanced solid tumors. Author BioFocus Newsroom Previous Next

< Back 16th – 18th September, 2025 Philadelphia, PA 15th Advancing Women’s Leadership in Pharma & Healthcare Join a dynamic community dedicated to breaking barriers and advancing women’s leadership in pharma and healthcare. The 15th Advancing Women’s Leadership in Pharma & Healthcare Conference brings together senior leaders, rising executives, and change-makers committed to advancing women in leadership. This premier event explores strategies to build inclusive cultures, accelerate career progression, and strengthen leadership impact across the pharmaceutical and healthcare sectors. Through thought-provoking keynotes, real-world case studies, and peer-driven discussions, attendees will gain practical insights to lead with confidence, influence organizational change, and shape the future of healthcare. Previous Register now Next

< Back 11th – 12th November, 2024 Philadelphia, PA 5th Next-Gen MSL Excellence The key challenges facing Medical Science Liaisons are not new – but they are also not solved. Now with the industry more thoroughly focused around remote work, it can be harder than ever to maintain relationships with KOLs and find the time for the networking required for proper relationship maintenance. Understanding that new technologies may not necessarily solve old problems as much as they merely change them, the 5th Next-Gen MSL Excellence Summit returns to Philadelphia this November for the most in-depth detailed analysis available on this topic. Previous Register now Next

New capabilities aim to shift laboratories from reactive troubleshooting to proactive problem-solving, but the real test will be whether AI agents can earn scientists' trust. < Back Tecan Brings Agentic AI to the Lab Bench with NVIDIA-powered Introspect Upgrade New capabilities aim to shift laboratories from reactive troubleshooting to proactive problem-solving, but the real test will be whether AI agents can earn scientists' trust. Tecan has added agentic AI capabilities to its Introspect lab analytics platform, which leverages NVIDIA's BioNeMo Agent Toolkit . The Swiss laboratory automation company announced the update on June 24, positioning it as a step toward what both firms call the "Data-Driven Laboratory", a vision they first outlined back in March. The pitch is straightforward enough: instead of finding out something's gone wrong after the fact, AI agents embedded in Introspect will continuously sift through lab data, workflows, and system performance to flag issues before they affect results. Early access to the upgraded platform is now open, with Tecan targeting pharmaceutical, biotechnology, and clinical lab environments first. Agentic AI vs. traditional lab monitoring: what's actually different Lab software has gotten reasonably good at telling you something broke. What it's been worse at is telling you something's about to break, or quietly costing you throughput without anyone noticing. This is the gap Tecan is trying to close. By layering NVIDIA's agent toolkit onto Introspect, the company says agents can spot hidden patterns limiting scalability or efficiency, then turn that analysis into concrete recommended actions rather than just another dashboard alert. Mukta Acharya, who heads Tecan's Life Sciences Business division, framed it as a structural shift rather than an incremental feature. Combining Tecan's laboratory expertise with NVIDIA's toolkit, she said, is meant to enable a new generation of intelligent lab solutions that proactively support scientists, improve productivity, and help speed up scientific outcomes. The real obstacle to agentic AI in regulated labs is trust, not technology What's notable in Tecan's announcement is how much attention goes to the safety architecture rather than just the AI's capabilities. The companies say they're building specific guardrails for responsible and reliable deployment of agentic AI in lab settings, controls intended to support transparency and keep automation within defined limits. That emphasis isn't surprising, and it's arguably the more interesting part of the story. Letting an algorithm flag an anomaly is one thing. Letting it take autonomous action inside a pharma or clinical workflow, where a wrong call could mean a failed batch, a compliance breach, or a scrapped clinical sample, is a different proposition entirely. Regulated lab environments don't move fast on automation that can't show its reasoning, and Tecan seems aware that adoption will hinge less on what the agents can technically do and more on whether lab managers and QA teams trust the audit trail behind their decisions. Tecan and NVIDIA's longer game is physical AI and next-gen lab instrumentation This release builds on the broader collaboration Tecan and NVIDIA announced in March, which set out a roadmap for AI-powered lab platforms. The companies say they intend to keep developing this technology, including exploring "Physical AI" for next-generation lab instrumentation, suggesting agentic software is one piece of a longer-term push to bring robotics and AI more tightly into lab hardware itself. For now, the rollout is limited to early access, and it remains to be seen how agentic AI performs once it's handling real laboratory data under real regulatory scrutiny rather than in a controlled pilot. The concept is compelling. Whether labs see meaningful productivity gains or just another layer of software to manage will become clearer as adoption widens beyond this initial phase. Author BioFocus Newsroom Previous Next

< Back 16th – 17th November, 2026 London, UK AI, Forecasting, Insights and Analytics for Smarter Decisions in Pharma Advance forecasting, AI and analytics to drive strategic commercial decision-making across functions in Pharma. The Marcus Evans AI, Forecasting, Insights and Analytics for Smarter Decisions in Pharma conference will explore how pharma leaders are embedding Gen AI for long-term forecasting, Agentic AI for Real-World-Evidence generation and advanced analytics into cross-functional workflows to improve forecasting confidence, commercial responsiveness and long-term strategic planning while maintaining strong data and AI governance. Learn from case studies, expert-led panels, roundtable discussions and interactive workshops on Gen AI-powered forecasting, AI-enabled commercial workflows, rare disease forecasting, lifecycle forecasting and strategic decision-making across evolving therapy markets. Whether you are looking to strengthen forecasting assumptions, accelerate AI adoption, improve launch planning or drive more informed commercial decisions, this event will deliver practical insights and actionable strategies from senior forecasting, analytics and AI leaders across pharma. Topics Covered: Drive strategic decision-making through forecasting, scenario planning, and market intelligence Shift from Forecasting to Foresight with Agentic AI and Real-World Evidence to Prepare for Multiple Futures Advance rare disease forecasting through integrated data and patient-centric insights Build forecasting agility in volatile and high-growth therapy markets Maximize forecasting confidence through healthcare data governance and analytical integrity Structure enterprise AI, forecasting and analytics functions through shared governance and operating models Best Practices and Case Studies from: Philippe Barillon , Executive Director, Strategic Intelligence & Business Impact, Global Corporate Affairs, Novartis Alireza Moayyeri , Senior Director, RWE Global, UCB Paul Boutry , Head of Strategic Planning & Analytics, BioMarin Gerardo Martinez, Senior Director , Global Market Access – Pipeline (Immunology & Rare Diseases), CSL Christopher Meyer , Head of Strategy & Operational Excellence, Ferring Pharmaceuticals Peter Elsig Raun , Director, Commercial Leadership Office and Projects, Lundbeck Special discounts available to BioFocus UK readers. For more information please contact: Stefanos Ioannou, Digital Media and PR Executive at stefanosi@marcusevanscy.com or visit: https://tinyurl.com/5hjv6yu9 Previous Register now Next

Researchers develop a 3D-printed small-scale, single-use bioreactor that features real-time monitoring of cell growth. < Back 3D Printing of Bioreactors: a New Horizon for Bioprocess Development Researchers develop a 3D-printed small-scale, single-use bioreactor that features real-time monitoring of cell growth. The continuous development of upstream bioprocesses requires cost-effective and customizable bioreactors for optimizing production processes. Leveraging the recent advances in additive manufacturing, this research introduces a small-scale 3D printed bioreactor designed for both mammalian and microbial cultivations. The bioreactor boasts a 90 mL working volume and incorporates inline pH and dissolved oxygen probes, along with a levitating magnetic stirrer. A unique feature is the integration of aeration channels and a sampling port directly into the vessel walls. Additionally, a 3D printed customizable optical biomass sensor enhances the bioreactor's functionality. The study evaluated the bioreactor's performance through technical characterization and proof-of-concept cultivations. Results indicated that the mixing time and oxygen mass transfer were adequate for cultivating both mammalian and microbial cells at high densities. For instance, an Escherichia coli fed-batch cultivation achieved an impressive maximum OD600 of 204. In another demonstration, a fed-batch cultivation of a Chinese hamster ovary cell line producing IgG antibodies achieved a peak viable cell density of 10.2 × 106 cells mL−1 and a maximum product titer of 2.75 g L−1. A notable aspect of the bioreactor is its 3D printed customizable optical biomass sensor, allowing real-time monitoring of cell growth. By employing a three-parameter fit, the inline biomass signal was successfully correlated to corresponding offline values with satisfactory accuracy. This innovation holds promise for advancing the efficiency of upstream bioprocessing by providing a cost-effective and flexible tool for investigating and optimizing production processes. Author BioFocus Newsroom Previous Next

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 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

A new CDMO partnership aims to bring clinical-grade AAV gene therapies to patients with LGMD types 2C and 2D, with an IND filing targeted for the second half of 2026 < Back Andelyn Biosciences and Evolyra Therapeutics Join Forces to Advance Gene Therapies for Limb-Girdle Muscular Dystrophy A new CDMO partnership aims to bring clinical-grade AAV gene therapies to patients with LGMD types 2C and 2D, with an IND filing targeted for the second half of 2026 Andelyn Biosciences, a Columbus, Ohio-based cell and gene therapy contract development and manufacturing organisation (CDMO), has announced a manufacturing partnership with Evolyra Therapeutics to produce clinical-grade adeno-associated virus (AAV) gene therapies for two forms of limb-girdle muscular dystrophy (LGMD): types 2C and 2D, also designated LGMDR5 and LGMDR3. The deal brings together Evolyra's proprietary next-generation viral vector with Andelyn's established manufacturing infrastructure, and represents another step forward in a field that is steadily gaining momentum. Limb-girdle muscular dystrophies are a group of around 32 distinct genetic disorders characterised by progressive weakness and wasting of the muscles surrounding the hips and shoulders. There are no approved disease-modifying treatments for any LGMD subtype, and the conditions can significantly reduce quality of life, mobility, and life expectancy. With more than 580,000 patients affected worldwide and a global market estimated to exceed $100 billion, the commercial and clinical case for effective therapies is considerable. Evolyra's programmes target the LGMDR3 and LGMDR5 subtypes specifically, a segment representing roughly 20,000 patients and an $11 billion market opportunity. At the core of Evolyra's approach is AAVMYO2, a proprietary muscle-targeting AAV vector that the company says substantially improves skeletal muscle expression while reducing liver toxicity relative to first-generation vectors. Liver toxicity has been one of the more persistent concerns in systemic AAV gene therapy, and addressing it at the vector level rather than through post-treatment management is a meaningful engineering priority. Evolyra reports complete protein expression and muscle restoration in preclinical animal models, though as with all such results, the translation to human trials will be the real test. Evolyra's approach is designed to deliver a complete, functional gene rather than a partial replacement, which the company considers essential for effective treatment Evolyra Therapeutics of conditions like LGMDR3 and LGMDR5. The company is building what it describes as a scalable, modular gene therapy platform, drawing on a consortium of over 500 patients to provide biomarker data, disease progression tools, and trial readiness support Evolyra Therapeutics. Under the partnership, Andelyn will apply its AAV Curator® Platform to support IND-enabling studies and Good Manufacturing Practice (GMP) production for Evolyra's clinical trials. The platform is designed to support the full arc of rare disease therapy development, from preclinical stages through to clinical manufacturing, without requiring companies to rebuild their supply chain at each phase transition. "Evolyra exemplifies the kind of transformative science we are eager to support," said Matt Niloff, Chief Commercial Officer at Andelyn Biosciences. "Their innovative approach to treating LGMDs with an AAV gene therapy aligns with our mission to accelerate access to life-changing treatments for patients with rare and ultra-rare diseases. We are very honored to help bring this breakthrough therapy to the patients who need it most." Nicholas Johnson, MD, CEO of Evolyra Therapeutics, pointed to the manufacturing continuity as a particular advantage: "We appreciate the partnership and commitment from Andelyn Biosciences. Their platform will allow a seamless transition between the different phases of our therapeutic development, ultimately accelerating our ability to bring these therapies to individuals living with LGMD as quickly as possible." Evolyra is targeting an IND submission in the second half of 2026, with Phase I/II clinical trials to follow. The company recently closed a $5 million seed round and is currently in the process of raising a $20 million Series A to fund programme advancement. That funding trajectory is not unusual for a company at this stage, though completing the Series A in the current biotech financing environment will matter considerably for whether the 2026 timeline holds. The partnership arrives at a time of genuine, if cautious, progress in LGMD gene therapy more broadly. In March 2026, Atamyo Therapeutics shared the first clinical results for its ATA-200 gene therapy in LGMD-2C/R5, from a Phase Ib/II trial at the University of Florida's Powell Gene Therapy Center. Ddw-online Meanwhile, Sarepta Therapeutics has multiple LGMD programmes in clinical development, with its SRP-9003 therapy meeting the primary endpoint of its Phase 3 EMERGENE trial, demonstrating robust beta-sarcoglycan protein expression in treated patients. CGTLive® These parallel programmes in overlapping LGMD subtypes underscore both the scale of unmet need and the competitive dynamics emerging in this space. Evolyra will need to differentiate on both the clinical profile of AAVMYO2 and its speed to trials. The Andelyn partnership is designed to help on the latter count, and the combination of an experienced CDMO with a next-generation vector and a patient-connected clinical network gives Evolyra a credible, if still early-stage, development platform. For patients with LGMDR3 and LGMDR5, who currently have no therapeutic options beyond symptom management, the prospect of multiple well-resourced gene therapy programmes entering clinical testing is the most tangible reason for optimism. Whether any of them ultimately translate into approved treatments remains to be seen. Author BioFocus Newsroom Previous Next

< Back 5th – 7th February, 2025 Santa Clara, CA PMWC 2025 PMWC, the “Precision Medicine World Conference” is the largest & original annual conference dedicated to precision medicine. PMWC’s mission is to bring together recognized leaders, top global researchers and medical professionals, and innovators across healthcare and biotechnology sectors to showcase practical content that helps close the knowledge gap between different sectors, thereby catalyzing cross-functional fertilization & collaboration in an effort to accelerate the development and spread of precision medicine. Previous Register now Next