The Centre for the Technologies of Gene and Cell Therapy (CTGCT) hosted the second edition of CTGCT Days from 15 to 17 September 2025 in Ljubljana, Slovenia. Over three days, the conference brought together more than 130 participants, including scientists, clinicians, regulators, and industry experts, to showcase how gene and cell therapies are reshaping medicine and creating new therapeutic frontiers.
Day 1: Opening with Global and Local Breakthroughs
The opening day set a strong scientific tone, beginning with welcome addresses from Roman Jerala and Mojca Benčina, who framed the event around CTGCT’s mission to connect Slovenia to the frontiers of advanced therapies.
The program began with Felix Wensveen (University of Rijeka), who explored how systemic metabolic changes during sickness can be targeted in non-communicable diseases, offering a broad immunological perspective. Next was Jürgen Kuball (UMC Utrecht), who presented his research on the spatial regulation of γδ T cells in colorectal cancer. By mapping receptors that guide γδ T cells into tumors, his team revealed a path toward receptor-guided immunotherapies, sparking discussion on strategies for solid tumors.
Nicola Maciocia (UCL Cancer Institute) addressed the challenges of developing CAR-T cells for T cell malignancies, focusing on strategies to prevent fratricide and relapse. The Slovenian spotlight shone with Robert Zorec (University of Ljubljana & Celica Biomedicals), who presented pioneering work on immunohybridomas—hybrids of dendritic and tumor cells created through lysosomal electrofusion. Zorec emphasized that this represents the first innovative biological medicine for advanced cancer therapy developed in Slovenia, with potential applications far beyond prostate cancer.
The day concluded with a presentation from Tiago Santos (Beacon Discovery, Bruker Cellular Analysis), who demonstrated single-cell functional assays to accelerate CAR-T and TCR-T development.
Day 2: Rare Diseases, Translational Pathways, and Clinical Lessons
Day two shifted attention to rare diseases and translational pathways. Natalia Marek-Trzonkowska (Medical University of Gdańsk) spoke on the complexity of cancer heterogeneity and the importance of selecting appropriate preclinical models. Tina Fink (NIC, Slovenia) introduced a novel CAR signaling configuration that enhances T-cell persistence and efficacy.
Ines Papak (University of Gdańsk) presented work on CD45⁺ stem-like cells in non-small cell lung cancer (NSCLC). These cells, typically linked to immune lineages, unexpectedly showed tumor-forming capacity and plasticity, acting as hidden drivers of initiation and metastasis. Further talks included Jiri Eitler (Masaryk University) on dual-CAR NK cells targeting PD-L1 and HER2/ErbB2, and Kavitha Lakshmi and Anke Fuchs on GMP-compatible CAR Treg production. Ramesh Ganesh (UMC Utrecht) presented results from a European viral vector survey, while Vesna Spasovski (IMGGE Belgrade) outlined stem cell–nanocarrier combinations for osteoarthritis therapy.
Regina Demlová (Masaryk University, CREATIC Centre of Excellence) gave a lecture on bridging research and patient care in pediatric and rare diseases. She showcased CREATIC’s model of linking academic research to accessible therapies for conditions neglected by industry, reinforcing the central role of academic centers in advancing ATMPs.
The Slovenian session featured Tanja Jesenko (Institute of Oncology Ljubljana) revisiting Slovenia’s first gene therapy trial with IL-12 electrotransfer, and Damjan Osredkar (UMC Ljubljana) highlighting the future of rare disease care. Matjaž Sever (UKC Ljubljana) spoke on academic CAR-T manufacturing, and Viktor Glaser (BeCAT, Charité Berlin) introduced multiplex-edited allogeneic CAR-T cells. Closing the scientific part, Polona Šafarič Tepeš and Seth Plancher (The Feinstein Institutes for Medical Research, Department of OBGYN at NYU Grossman Long Island School of Medicine, USA) reflected on U.S. models of hospital-based translational innovation.
The day ended with a round table on Regulatory and Clinical Pathways for Gene and Cell Therapy. Panelists included Lenka Součková (Masaryk University), Leila Amini (Charité Berlin), Jürgen Kuball (UMC Utrecht), Tanja Jesenko (Institute of Oncology Ljubljana), and Suzana Vidic (JAZMP & EMA CAT).
Discussions highlighted the knowledge gap in regulation, both at applicant and agency level, the challenges of fragmented national systems such as Germany’s federal structure, and the importance of planning regulatory strategy early. EMA’s evolving openness, hospital exemption variations, and the economic feasibility of ultra-rare therapies were all debated. The panel emphasized that translation requires scientific excellence, regulatory foresight, and stronger cooperation between clinicians, regulators, and research institutions like CTGCT.
Day 3: Gene Editing for Human Health and the Road to Commercialization
The final day centered on gene editing technologies and their therapeutic applications.
Ajda Lenardič (ETH Zurich) delivered a presentation on iPSC-derived functional muscle stem cells for Duchenne muscular dstrophy. By generating donor-derived muscle stem cells in animal hosts, her work pointed toward a new regenerative strategy for this devastating pediatric condition.
The session featured Robert Torrance on STAT1 gene editing for immunodeficiency, Tina Lebar (NIC, Slovenia) on novel tyrosine recombinases, and Frank Buchholz (TU Dresden) on designer recombinases for genome enginering.
Emma Morris (UCL, London) provided her keynote highlight with advances in gene editing for inborn errors of immunity, sharing early clinical data on rare monogenic diseases such as CTLA4 insufficiency and STAT1 GOF. Her talk underscored the shift from proof-of-concept to therapies with tangible clinical benefit.
Later talks included Ana Dolinar Češarek (NIC) on biosafety concerns with rAAV co-delivery, Oscar Wilkins (UCL) on precision therapeutics in ALS and FTD, James Sleigh (UCL) on axonal transport in neuropathies, and Luis Pereira de Almeida (University of Coimbra) on allele-specific silencing in Spinocerebellar Ataxia Type 3.
The day concluded with a round table on Bridging Innovation and Commercialization in Gene and Cell Therapy. Panelists included Zora Čechová (Masaryk University), Luis Pereira de Almeida (University of Coimbra), Boštjan Čeh (Labena Group), Mojca Pečar (Slovenian Intellectual Property Office), and Miroslav Gregorič (Slovenian Parliament).
Panelists reflected on how gene and cell therapies represent both unmet medical opportunities and major commercial challenges. Discussion spanned early planning around market, IP, and regulation, the role of hospital exemptions, the importance of technology transfer offices, and the need for policy support for R&D investment. A recurring theme was the necessity of stronger cooperation between researchers, clinicians, and business leaders to ensure discoveries reach patients. The panel ended with a call to action for young researchers, entrepreneurs, and policymakers: to prepare early, communicate openly, and keep patient benefit at the center.
Takeaways
CTGCT Days 2025 demonstrated that gene and cell therapy is no longer a distant promise but an emerging clinical reality. Over three days, the conference showcased world-class science, Slovenian innovation, and European collaboration, positioning Ljubljana as a growing hub for translational medicine.
We thank all speakers, participants, partners, and sponsors for contributing to three days of inspiring exchange and collaboration.