Pick of the posters: ESGCT 2025
At this year’s 32nd Annual Congress of the European Society of Gene and Cell Therapy (ESGCT; 7−10 October; Seville, Spain), we took a trip around the poster hall and picked out some posters that stood out to us. Check out our top 4 below.
Enhancing LAMA2 expression in murine mesenchymal stromal cells through gene writing: a preclinical approach for MDC1A
Òscar Garriga-Monterde, Vall d’Hebron Institute of Research, Universitat Autònoma de Barcelona (both Barcelona, Spain)
What is your research on and what are its key takeaways?
My research is about developing an ex vivo therapy with edited mesenchymal stromal cells to try to cure MDC1A, which is a rare congenital neuromuscular disorder. In this condition, we observed that a protein called laminin 211 is affected. This protein consists of three subunits, and in this condition, the LAMA2 gene – which encodes the alpha-2 subunit – is mutated. Laminin 211 protein is essential for binding the extracellular matrix to the muscular fibers so its absence leads to inflammation, muscle fibrosis and patient death at a premature age.
My research goal is trying to use mesenchymal stromal cells and edit them with novel tools like hyPB (hyperactive piggyBac), PASTE or FiCAT (Find and cut-and-transfer, developed by my supervisor, Maria Pallarès-Masmijtjà, which is a hyperactive piggyBac transposase fused with CRISPR). This will allow us to insert copies of the gene and boost the LAMA2 expression ex vivo and infuse the cells – in this case, we’re working with a murine model and trying to cure them.
What would you hope the impact of this research will be on how we use mesenchymal stromal cells and the way we use gene editing for muscular dystrophy?
Well, mesenchymal stromal cells are used nowadays in research for some diseases, for example, Duchenne muscular dystrophy. In this approach, we’re trying to use a platform approach to check if these types of cells can be used for other neuromuscular diseases and also combine them with novel gene editing tools. Now, we’re focused on using hyperactive piggyBac transposase because we are working with a gene that’s really long – it’s about 9.8 kilobases. So conventional gene editing and gene delivery technologies are not feasible. We can’t use, for example, AAV for this, so we went with another approach. That’s mainly the goal – to see if this approach can be used in other neuromuscular diseases and other genetic pathologies that have similar issues with long genes.
If you had unlimited resources, what work would you do next?
That’s a great question. The main problem is that we don’t have a lot of funds. First, when talking about my research, I would accelerate things like buying plasmids, constructs, or other materials, because in our case, we have to produce everything. We cannot go to a company and ask if they can generate the constructs or other materials for us. So, I think having funding would accelerate my research.
And, when not talking about my research – if I had unlimited resources, I think I would try this approach or other similar approaches with other rare diseases. I’m currently working with rare diseases and it’s really, really difficult – not just in Spain, I think in other countries too – to finance projects that are trying to develop preclinical cures for rare diseases. So, I think having more resources would be a great benefit for this research area.
EGFRvIII-targeted virus-like particles as a versatile platform for selective cell killing in glioblastoma
Canan Bayraktar-Odabas, Koç University Research Center for Translational Medicine (Istanbul, Turkey)
What is your research on and what are its key takeaways?
My research focuses on engineering virus-like particles (VLPs) that selectively recognize and kill glioblastoma cells harboring the EGFRvIII mutation. By displaying an anti-EGFRvIII single-chain antibody on the VLP surface, we achieved targeted delivery of toxic or genome-editing cargo proteins specifically to cancer cells. This modular system can be easily reprogrammed for different targets and cargos, offering a flexible therapeutic platform with high selectivity and minimal off-target effects.
What impact do you hope this research will have on the way glioblastoma is treated?
Glioblastoma remains one of the most lethal human cancers, marked by relentless recurrence and resistance to nearly all available therapies. Current treatments – surgery, radiotherapy and chemotherapy – offer only limited survival benefits and often come at the cost of severe neurological and systemic side effects. The additional challenge lies in the blood–brain barrier, which prevents most therapeutic agents from reaching the tumor effectively.
Our goal is to develop a precise and adaptable delivery system that can overcome these biological barriers and act only where it’s needed. By engineering EGFRvIII-targeted VLPs that selectively recognize and destroy tumor cells, we aim to provide efficacy without collateral toxicity. If successfully translated, this strategy could complement or even replace non-specific viral vectors and chemotherapy, leading to safer, more targeted and ultimately more humane treatments for patients.
In the long term, it has the potential to transform glioblastoma therapy from an inevitable recurrence into a controllable, personalized condition.
How do virus-like particles compare to other delivery methods for targeting cancer cells?
Each delivery system brings its own strengths – and limitations – to the table. Lipid nanoparticles have revolutionized nucleic acid delivery, but their targeting precision and endosomal escape efficiency remain limited, especially in solid tumors like glioblastoma. AAVs, while effective in gene therapy, carry packaging size constraints, pre-existing immunity issues and challenges in retargeting to specific tumor antigens.
VLPs occupy a unique space between these two worlds. Like AAVs, they harness nature’s highly evolved architecture for efficient cell entry, yet they remain non-replicating and genome-free, eliminating genotoxic risk. Unlike lipid nanoparticles, their protein-based shells can be genetically engineered – allowing precise control over tropism, surface display and cargo encapsulation. This makes them ideal for antibody-guided targeting, where a single-chain variable fragment directs the VLP specifically to cancer cells expressing EGFRvIII, leaving healthy tissue untouched.
In essence, VLPs combine the safety and scalability of synthetic systems with the biological intelligence of viral design. They’re not merely carriers – they are programmable biological machines, capable of evolving alongside our therapeutic ambitions.
If you had unlimited resources, what work would you do next?
With unlimited resources, I would reimagine the VLP from the ground up. Beyond expanding the targeting repertoire, my vision is to redesign the VLP’s architecture itself – using directed evolution and AI-guided protein engineering to create a diverse library of capsid variants optimized for low immunogenicity, enhanced stability and efficient cargo encapsulation. The goal would be to discover the most refined and adaptable VLP form – one capable of navigating biological barriers and delivering therapeutic cargos with near-surgical precision.
At the same time, I would broaden the therapeutic spectrum by packaging a wide range of functional cargos – from apoptotic and genome-editing proteins to immune-modulating factors — to enable synergistic, multi-layered treatments. These engineered “smart particles” would then be tested in patient-derived organoids and real-time in vivo imaging models, to understand their behavior within the complex brain microenvironment.
Engineering a non-viral glucose-responsive gene system for regulated insulin expression and functional diabetes remission
An Nisaa Nurzak, University of Nottingham, Biodiscovery Institute (UK)
What is your research on and what are its key takeaways?
My research focuses on developing a non-viral, glucose-responsive gene system that enables regulated insulin expression for diabetes treatment. It explores the use of plasmid-based gene constructs delivered through a nanoparticle system to achieve safe and controlled gene expression without relying on viral vectors. The goal is to create a platform that can respond to glucose fluctuations and contribute to functional diabetes remission.
What impact do you hope this research will have on gene therapy approaches for addressing Type 2 diabetes?
This research aims to reduce dependence on insulin injections and improve patient quality of life by developing a non-viral, glucose-responsive gene system for regulated insulin expression. By using a plasmid–nanoparticle delivery platform, it minimizes the risks associated with viral delivery systems while enabling long-term, cell-based glucose regulation without permanent genetic modification. Ultimately, this work contributes to the development of next-generation gene therapies that are adaptive, reversible and clinically translatable, offering a promising approach for managing and potentially achieving functional remission of Type 2 diabetes.
What was a significant challenge you encountered during this research and how did you overcome it?
A significant challenge in this research was achieving stable and efficient gene expression using a non-viral plasmid–nanoparticle delivery system, as early constructs exhibited limited cellular uptake and inconsistent expression. To address this, multiple plasmid designs were developed and evaluated to identify the most effective configuration for glucose-responsive and sustained insulin expression. In parallel, nanoparticle formulation parameters such as charge ratio, particle size and transfection efficiency were optimized to enhance delivery performance. Through iterative plasmid design and delivery refinement, a reliable system was established that achieved consistent, glucose-dependent gene regulation suitable for functional diabetes management.
If you had unlimited resources, what work would you do next?
If I had unlimited resources, I would focus on translating this non-viral glucose-responsive gene system into preclinical and clinical applications. This would include developing targeted nanoparticle delivery systems capable of efficiently and selectively reaching insulin-producing or hepatic cells in vivo. I would also aim to conduct long-term studies in diabetic animal models to evaluate safety, stability and therapeutic efficacy over time. Furthermore, I would invest in advanced bioengineering approaches, such as integrating synthetic biology and AI-guided circuit design, to enhance the precision and adaptability of the glucose-responsive system. Ultimately, the goal would be to develop a clinically viable, tunable and reversible gene therapy platform capable of achieving sustained functional remission of Type 2 diabetes.
Establishment of a scalable neuronal reporter model of Machado-Joseph disease based on gene-edited patient cells as a platform for high-throughput drug repurposing
Frederico Pena, Centre for Neuroscience and Cell Biology, Gene Therapy Center of Excellence (Coimbra, Portugal)
What is your research on and what are its key takeaways?
My research focuses on Machado-Joseph disease, which is a neurodegenerative motor disease and is currently without a cure. Our current models for the disease suffer from low clinical translatability, so I am currently trying to create a new disease model that improves that translatability and that can help with the repurposing of drugs that are already commercially available, and that may have hidden potential to help treat Machado-Joseph disease.
What impact do you hope this research will have on treatment development for Machado-Joseph disease?
A disease model with better clinical relevance can help in several ways. Firstly, the closer our model is to the disease context in patients, the better the insights and conclusions we can gather about how the disease behaves. Secondly, de novo drug development takes a long amount of time that the patients cannot wait for, and very large amounts of money. We hope that our model can help here by repurposing commercially available drugs to Machado-Joseph disease. A repurposed drug would naturally arrive to the patients much quicker, as well as reducing development costs.
What was a significant challenge you encountered during this research and how did you overcome it?
One of the more significant challenges may have been the optimization of the cell line. From the gene editing, to the differentiation protocol, it required quite a degree of trial and error, but we are happy with how the model turned out.
If you had unlimited resources, what work would you do next?
With unlimited resources, I believe we would expand our drug screening to more drugs and/or compounds and check what pathways may be altered in the disease, as well as further characterize and validate our model. Finally, we would validate any potential interesting drugs in several models, both in vitro and in vivo to ensure efficacy in helping treat Machado-Joseph disease.
The opinions expressed in this interview are those of the interviewee and do not necessarily reflect the views of RegMedNet or Taylor & Francis Group.