ESGCT Conference Takeaways: A specialty laboratory perspective

Experts from Cerba Research, a leading specialty laboratory solutions provider, recently attended the 31^st European Society of Gene and Cell Therapy (ESGCT) Congress to share some of its most recent cell and gene therapy developments with an audience of industry experts, academia, sponsors and patient organizations.

Each year, the ESGCT conference attracts key opinion leaders  in gene and cell therapy from Europe and around the world, spotlighting recent breakthroughs and advancements in the field and highlighting novel  techniques that are  moving toward safe and curative treatments for both major and rare diseases. Events such as the ESGCT play a key role in helping to advance, improve and launch these novel treatments into clinical practice, as well as ensuring that cell and gene therapies are safe and effective. 

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Key Takeaways ESGCT Congress

Advances in Rare Disease Treatments

With cell and gene therapy R&D heading towards genome-orientated therapies, this year’s conference featured a wealth of presentations on genome editing techniques targeting both rare and common diseases. It was noted that for some diseases, gene therapies may be the only effective treatment option.

Notably, a presentation by an expert from a cardiovascular gene therapy developer introduced a novel target using genome-editing technique aimed at addressing unmet needs in cholesterol management. This technique targets genetic and refractory hypercholesterolemia syndromes, which put individuals at risk for atherosclerotic cardiovascular disease (ASCVD).

In this presentation, the speaker emphasized that gene editing could provide lifelong reductions in low-density lipoprotein cholesterol (LDL-C), overcoming the limitations of chronic care. The study introduced a novel, in vivo base-editing technique that inactivates the PSCK9 gene. This technique is genetically and pharmacologically validated to lower LDL-C and reduce cardiovascular risk, a leading global cause of mortality.¹ Although initially focused on a rare subset of patients, this approach also holds promise for broader applications, potentially benefiting millions, with cardiovascular diseases emerging as a significant area of focus for gene editing.

Additionally, advancements in gene therapy were also discussed, particularly in relation to children with rare genetic disorders that cause deafness. After receiving AAV-based gene therapy injections directly into the cochlea, some children born deaf have regained hearing and begun to develop speech, demonstrating the powerful curative potential of gene therapies.

This rapid acceleration of gene therapy development, with some advances even reaching clinical trials in a matter of months, is fuelled by the integration of cutting-edge technologies, novel therapies and artificial intelligence, bringing genomic therapies closer to real-world applications.

Progress in CAR T-Cell Therapy for Autoimmune Diseases

Originally intended for cancer treatment, CAR T-cell therapies are now being repurposed to treat a variety of autoimmune disorders. These therapies are designed to combat antibody-producing cells, B-cells, that mistakenly trigger immune responses against the body, much like they target cancerous B-cells.

Building upon these immune therapies, next-generation vectors—such as lenti-viral and non-viral delivery mechanisms—are accelerating advancements in cell engineering. These therapies offer the potential for both personalized* and universal* applications. In personalized therapies, a patient’s own cells are modified through gene and cell therapy and then reintroduced into their body. In contrast, universal therapies utilize allogeneic stem cells, helping to address the common challenge of graft-versus-host disease.

Innovations in Treatment Monitoring Methods

Cerba Research also presented some key developments for monitoring cell and gene therapies. One poster showcased Cerba Research’s newly developed qPCR assays, which sensitively monitors Ad5 vector shedding and biodistribution to ensure the safe clinical administration of Ad5 gene therapy in cancer patients.² Another highlighted Cerba Research’s unbiased sequencing approach accurately identifies vector integration sites using CRISPR-Cas9 and long read sequencing. This technique monitors if the insertion of therapeutic genes into the genome are safe, thereby elucidating the risk of therapy-induced oncogenesis.³

Feedback during the conference strongly suggested that these techniques will become a mainstay approach for detecting and managing the safety of different gene/cell therapies, with Cerba Research ready to offer these techniques.

As the landscape of gene and cell therapy continues to progress from rare diseases to unmet clinical needs of larger populations, the combination of technological advancements, such as genome editing, delivery systems and regulatory support will likely pave the way for more effective therapies"

Karthikeyan Devaraju, PhD Sr Scientist Cell & Gene Therapy Development, Cerba Research

Sequencing techniques are now gaining increased attention in clinical research, and coupled with digital PCR, molecular diagnostics is advancing the whole field. Cerba Research is supporting this movement, offering a portfolio of services, starting from product development, pre-clinical and clinical services to post-marketing surveillance for cell, gene and genome-editing therapies.

 Offering a diverse toolset, from optical genome mapping to sequencing and digital PCR, is essential to verify that potential treatments are accurately targeting intended genomic alterations without off-target effects. These can be implemented in a pre-clinical and clinical setting to ensure that the genomic alterations do what they are supposed to do and there are no off-target effects in clinical research. We also offer a range of biomarker analysis tools to monitor the effect of treatments. All these techniques are developed in-house as Cerba Research continues to support the safe and effective implementation of cell, gene and genome therapies.

Conclusion

The field of gene and cell therapy is rapidly advancing, with key stakeholders consolidating resources to tackle both mainstream and rare diseases. Current clinical trials are expected to reveal key challenges in effective treatment, while innovations in genetic engineering are enhancing therapeutic vectors that address concerns related to dosage toxicity and immunogenicity. Significant progress is made in genome editing using new classes of nucleases and base editors as well as targeting mutations that affect the majority of the population.
In addition, regulatory bodies are keenly watching this space of biomedical R&D, working alongside therapy developers, CROs and laboratory solutions providers to ensure that regulations are implemented in line with current developments and requirements.

In conclusion, as the landscape of gene and cell therapy continues to progress, the combination of technological advancements and regulatory support will likely pave the way for more effective, therapies and treatments, ultimately benefiting a larger patient population.

To learn more about Cerba Research’s state-of-the-art capabilities, contact a member of the team today [link to https://cerbaresearch.com/contact-us/] and find out how we can assist you with your clinical trial challenges. Alternatively you can visit our website here [link to: www.cerbaresearch.com] for further information.

Karthikeyan Devaraju, PhD Sr Scientist Cell & Gene Therapy Development

Karthikeyan Devaraju has over 15 years’ experience in the regenerative medicine and advanced therapies (diverse cell and gene therapies). His expertise spans stem cells, cell fate engineering, gene delivery systems, product development and both process and analytical development / optimization using diverse technology platforms. He received his PhD in Stem Cells and Neurology from Lund University. He has held various scientific positions in academic research institutions, bio-pharma and CDMOs.

References

1. Khera, A.V. Developing single-course gene editing medicines to treat cardiovascular disease. Verve Therapeutics.
2. Shu, Y.L., et.al., AAV1-hOTOF Gene Therapy for Autosomal Recessive Deafness 9. Fudan University.
3. Buyle, V.G., Nuijten, P., Ingelse, S., Jimenez, V.C., and Devaraju, K. 2024. Monitoring adenoviral gene therapy. Research and Development, Cerba Research- Netherlands. https://cerbaresearch.com/app/uploads/2024/10/Cerba-Research_ESGCT_Monitoring-adenoviral-gene-therapy_Digital.pdf
4. Hout, A.J., Buyle, VG., Pagano, JFB., Devaraju, K. 2024. Precision mapping viral integrationi landscapes with CRISPR-Cas- and long read sequencing. Research and Development, Cerba Research- Netherlands. https://cerbaresearch.com/app/uploads/2024/10/Cerba-Research_ESGCT_Precision-Mapping-Viral-Integration-Landscapes_Digital.pdf

About Cerba Research

Cerba Research is a leading specialty laboratory services provider with the capacity and breadth of a global central laboratory network. Our highly qualified scientists provide insight on the latest biomarkers, assays and testing approaches and develop innovative solutions for unique challenges across all research phases, to pharmaceutical, biotechnology, medical device, government, public health, and CRO organizations. Cerba Research’s extensive capability in laboratory testing and global logistics including Cell & Gene Therapy, Bioanalysis, Flow Cytometry, HistoCytopathology, and Next Generation Sequencing, enables us to drive operational agility at scale in a wide range of therapeutic areas, with recognized expertise in Virology, Immunology, Oncology and Cell & Gene Therapy. Cerba Research is part of the Cerba HealthCare Group with 15,000 employees on five continents, driven to advance diagnosis and health.