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Takeaways from the In Vivo Gene Therapy Symposium
Connecting Leaders Shaping Advanced Therapy Medicinal Product (ATMP) Development in the Nordics
On October 1 we were delighted to bring together subject matter experts from across the Nordic region to explore the latest developments in in vivo gene therapies. This event was co-hosted with the Karolinska ATMP Center, established by Karolinska Institutet and Karolinska University Hospital to further advance cell, gene, and tissue therapies.
Knut Steffensen, Director of the ATMP Center, opened the agenda, which covered a wide range of topics, from the latest therapeutic advances to investor and market insights.
Presentations
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Advances on Preclinical Models for In Vivo Gene Therapies
Johanna Uhari-Väänänen, Group Leader, Neuropharmacology, Charles River
A look into how the Charles River site in Kuopio, Finland supports the development of gene therapies through in vivo models and readouts with insights into trending disease models, novel routes of administration, and a range of success stories within clinical gene therapy.
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Charles River Incubator and Accelerator Program
Andrew Frazer, Associate Director, Scientific Solutions, Gene Therapy, Charles River
Launched in 2024 to provide services beyond traditional manufacturing capabilities, CIP and CAP provide collaborative support for innovative early-stage ATMP developers to streamline their journey from discovery to clinical application.
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CircVec: A Powerful Circular RNA Expression Platform to Enhance Viral and Non-viral Gene and Cell Therapies
Eoghan O'Leary, VP of Research, Circio
A circular RNA expression platform that has the potential to address common viral gene therapy challenges, such as the requirement for high dose levels and risk of associated adverse events; the platform is currently focused on preclinical development and has the potential to deliver durable, versatile platform solutions for a range of disease indications.
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Engineered Extracellular Vesicles and Synthetic LNPs for Targeted Delivery of Gene Editors and RNA Therapeutics
Samir El Andaloussi, Professor of Biomolecular Medicine and Advanced Therapies, Karolinska Institute
A detailed review of current research focus and data demonstrating the high potential of non-viral delivery systems with gene therapy applications.
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Market Access
Freddie Henriksson, Market Access & Government Affairs Director, Johnson & Johnson Innovative Medicine
Insights into patient access for gene therapies including challenges within current systems, national and regional initiatives within the Nordics, and suggested new pathways to address value proposition, risk, and funding.
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Perspectives and Challenges in Toxicological Studies for In Vivo Gene Therapies
Roberto Nitsch, Director Gene Therapy, AstraZeneca
A deep dive into toxicology and safety assessment for in vivo gene therapies, including novel alternative technologies and method development for rapid and cost-effective screening.
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The Journey of In Vivo Gene Therapy: From Scientific Discovery to Patient Impact
Anu Jääskeläinen, Nordic Medical Lead, Novartis
An overview of the development, approval, and commercialization for Onasemnogene abeparvovec (Zolgensma); this gene therapy blockbuster has now been used to treat over 5,000 patients worldwide for spinal muscular atrophy (SMA) with access extended to the Nordics since 2021.
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The development of gene therapy products demands a holistic, science-driven approach that goes well beyond traditional manufacturing capabilities. Charles River has established two novel initiatives, the Charles River Accelerator Program (CAP) and the Charles River Incubator Program (CIP), to leverage our full range of expertise and experience to support developers' transition from discovery through early clinical stages and beyond. This unique, integrated approach is designed to identify and address translational bottlenecks early, increasing the probability of clinical success for novel therapies and enabling technologies.”
Andrew Frazer, Associate Director, Scientific Solutions, Gene Therapy
In Vivo Gene Therapy Patient Spotlight
Attendees also uncovered all-important patient perspectives from the Karolinska Institute team.
Anders Kvanta, Professor and Senior Physician specializing in Ophthalmology at the Karolinska Institute and St. Erik's Eye Hospital, shared a fascinating journey through the basis, design, and management of gene therapy clinical trials for Bothnia dystrophy, a rare genetic disorder of the retina caused by mutations in the retinaldehyde-binding protein 1 (RLBP1) gene of the visual cycle. He also provided a future outlook for agnostic therapies that have the potential to address a wide range of Inherited Retinal Dystrophies (IRD).
Providing a first-hand account demonstrating the benefits of the AAV-based gene therapy Zolgensma for young patients with Spinal Muscular Atrophy (SMA), Thomas Sejersen, Professor and Senior Physician specializing in Neuromuscular Disorders in Childhood at the Karolinska Institutet, also gave an overview of the gene therapy pipeline focusing on treatment of Duchenne Muscular Dystrophy (DMD).
The State of Gene Therapies Panel
Wrapping up the agenda with a panel discussion on the state of gene therapies in the Nordics, Ewa Ellis, Karolinska ATMP Center, was joined by Knut Steffensen and Dr. Ramin Baghirzade, Charles River, plus expert panelists Eoghan O'Leary, Anders Kvanta, Sebastian Bauer, and Jan-Olov Sandberg.
Consolidating perspectives from academia, emerging and established biotech, large pharma, and first-hand medical care, highlights included an engaging discussion on key trends and challenges within the gene therapy sector, ranging from barriers to translational science and clinical application to a more competitive and selective funding ecosystem in recent years.
Discussion also focused on challenges specific to the Nordic region, such as the significant gap that has emerged between fast-paced, high-impact research and the lagging infrastructure required for clinical manufacture and commercialization. While some innovative initiatives are underway to address healthcare funding and patient access, panelists agreed that it may not be a simple one-size-fits-all solution and that the benefits of standardization across the region may be difficult to realize.
Despite shared challenges, there was a strong sense of optimism, and a consensus was that gene therapy is here to stay. Organizations like the Karolinska Institute are leading the way with state-of-the-art research and real-world clinical applications, and the potential of advanced therapies is undeniable.
Significant advancement in the sector in the coming years is predicted, with the emergence of novel and targeted delivery systems set to transform the way in which we can apply gene therapy and open the door to developing products for new and previously untreatable diseases. Similarly, improvements in manufacturing technology and regulatory guidance are expected, and these factors are set to have an overall positive impact on both cost of goods and speed to market. When coupled with improved systems for healthcare adoption and patient access, the future is looking bright for the Nordic region and worldwide.
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