Periodic Reporting for period 1 - CTGCT (Centre of Excellence for the Technologies of Gene and Cell Therapy)
Reporting period: 2023-09-01 to 2025-02-28
Summary
The Centre of Excellence Centre for the Technologies of for Gene and Cell Therapy (CTGCT) in Slovenia focuses on advancing biomedical procedures, particularly gene and cell therapies (GCTs) and cancer immunotherapies. Using an interdisciplinary approach that integrates synthetic biology and immunology, CTGCT collaborates with leading institutions like UCL, UMC Utrecht, TU Dresden, and Charité Berlin to develop GCT technologies, foster industry partnerships, and promote personalized medicine.
With a focus on neurological and rare genetic diseases and cancer immunotherapy, CTGCT bridges research and clinical applications by ensuring GMP-quality manufacturing of therapeutic reagents. It actively involves clinicians and patient organizations to align its efforts with healthcare needs.
Key Objectives:
Establish a state-of-the-art facility in Slovenia for translating research into therapies.
Attract top international scientists and foster research collaborations.
Develop a GMP facility for clinical-grade therapy production.
Support first-in-human clinical trials for rare diseases and cancer immunotherapy.
Facilitate technology transfer, patents, and industry collaborations to drive commercialization.
Expected Impact:
CTGCT aims to revolutionize treatments for cancer and rare genetic diseases, improve patient outcomes, and position Slovenia as a leader in advanced therapies. The project will also drive economic growth, create jobs, and strengthen industry partnerships while enhancing public health.
The Centre of Excellence Centre for the Technologies of for Gene and Cell Therapy (CTGCT) in Slovenia focuses on advancing biomedical procedures, particularly gene and cell therapies (GCTs) and cancer immunotherapies. Using an interdisciplinary approach that integrates synthetic biology and immunology, CTGCT collaborates with leading institutions like UCL, UMC Utrecht, TU Dresden, and Charité Berlin to develop GCT technologies, foster industry partnerships, and promote personalized medicine.
With a focus on neurological and rare genetic diseases and cancer immunotherapy, CTGCT bridges research and clinical applications by ensuring GMP-quality manufacturing of therapeutic reagents. It actively involves clinicians and patient organizations to align its efforts with healthcare needs.
Key Objectives:
Establish a state-of-the-art facility in Slovenia for translating research into therapies.
Attract top international scientists and foster research collaborations.
Develop a GMP facility for clinical-grade therapy production.
Support first-in-human clinical trials for rare diseases and cancer immunotherapy.
Facilitate technology transfer, patents, and industry collaborations to drive commercialization.
Expected Impact:
CTGCT aims to revolutionize treatments for cancer and rare genetic diseases, improve patient outcomes, and position Slovenia as a leader in advanced therapies. The project will also drive economic growth, create jobs, and strengthen industry partnerships while enhancing public health.
Establishment of CTGCT
Significant progress has been made in establishing the Centre of Excellence Centre for the Technologies of Gene and Cell Therapy (CTGCT) to advance cancer immunotherapy and gene editing for rare diseases. The legal entity D22 – Centre for the Technologies of Gene and Cell Therapy was formally approved in September 2024, with Dr. Mojca Benčina appointed as Head. CTGCT operates as an infrastructure center within the National Institute of Chemistry (NIC).
Recruitment of International Scientists
CTGCT has recruited key personnel, including a financial expert, project manager, CTGCT manager, translation officer, and cleanroom specialist. The next phase focuses on hiring researchers, veterinarians for preclinical research, and quality control experts. During RP1, 12 personnel (3 full-time, 9 part-time) were employed with an average FTE of 0.4.
Research Partnerships
CTGCT has established collaborations with top institutions, submitting three EU project proposals (UNLOCKinG, RESPOND, AMMNIC) and partnering with UCL researchers. It is also involved in the EU Horizon Excellence Hub GeneH project with the University of Coimbra, focusing on gene therapy advancements. Additionally, Prof. Jerala secured an ERC Advanced Grant (PROFI) to support CTGCT’s technological research.
GMP Facility Development
A GMP facility is planned to be operational by year 5, with building plans completed for cleanrooms, preclinical labs, and quality control. This facility will support gene and cell therapy development.
Research Translation & Regulatory Discussions
CTGCT is advancing first-in-human clinical trial preparations and developing customized therapies for rare diseases and cancer immunotherapy. In May 2024, it organized a roundtable on ATMP regulation to discuss gene and cell therapy applications, regulatory challenges, and clinical trial processes in Slovenia, the Netherlands, and Germany.
Technology Transfer & Commercialization
CTGCT is actively working on technology transfer for commercialization, aiming for eight patents, two licenses, four industrial collaborations, and two spin-offs by year 6. So far, three patent applications have been filed, with three more in preparation. A Translation Office and Committee have been established to support these efforts.
Significant progress has been made in establishing the Centre of Excellence Centre for the Technologies of Gene and Cell Therapy (CTGCT) to advance cancer immunotherapy and gene editing for rare diseases. The legal entity D22 – Centre for the Technologies of Gene and Cell Therapy was formally approved in September 2024, with Dr. Mojca Benčina appointed as Head. CTGCT operates as an infrastructure center within the National Institute of Chemistry (NIC).
Recruitment of International Scientists
CTGCT has recruited key personnel, including a financial expert, project manager, CTGCT manager, translation officer, and cleanroom specialist. The next phase focuses on hiring researchers, veterinarians for preclinical research, and quality control experts. During RP1, 12 personnel (3 full-time, 9 part-time) were employed with an average FTE of 0.4.
Research Partnerships
CTGCT has established collaborations with top institutions, submitting three EU project proposals (UNLOCKinG, RESPOND, AMMNIC) and partnering with UCL researchers. It is also involved in the EU Horizon Excellence Hub GeneH project with the University of Coimbra, focusing on gene therapy advancements. Additionally, Prof. Jerala secured an ERC Advanced Grant (PROFI) to support CTGCT’s technological research.
GMP Facility Development
A GMP facility is planned to be operational by year 5, with building plans completed for cleanrooms, preclinical labs, and quality control. This facility will support gene and cell therapy development.
Research Translation & Regulatory Discussions
CTGCT is advancing first-in-human clinical trial preparations and developing customized therapies for rare diseases and cancer immunotherapy. In May 2024, it organized a roundtable on ATMP regulation to discuss gene and cell therapy applications, regulatory challenges, and clinical trial processes in Slovenia, the Netherlands, and Germany.
Technology Transfer & Commercialization
CTGCT is actively working on technology transfer for commercialization, aiming for eight patents, two licenses, four industrial collaborations, and two spin-offs by year 6. So far, three patent applications have been filed, with three more in preparation. A Translation Office and Committee have been established to support these efforts.
a. Healthcare
CTGCT has contributed to patient care by participating in new regulations, including the 2024 proposal for the Regulation on Non-routine Preparation of Advanced Therapy Medicinal Products in Slovenia, now in inter-ministerial coordination. It has supported the development of Urbagen, a drug for CTNNB1 syndrome, which is currently in manufacturing. A roundtable on ATMP regulation examined the impact of existing regulations, the use of gene and cell therapies in Slovenia, the Netherlands, and Germany, and areas for regulatory improvement. CTGCT’s initiatives help accelerate advanced therapies, improve regulations, and enhance public and professional acceptance of gene and cell therapies.
b. Economic Growth
CTGCT has strengthened academia-industry collaboration, aiming to expand industrial partnerships and career opportunities. It has helped establish regional connections, leading to the AMMNIC project, a proposed postdoctoral training program on molecular disease research and therapy development, involving Slovene, Croatian, and Serbian partners. CTGCT is also part of EU Horizon Excellence Hub GeneH, working with the University of Coimbra to advance gene therapy research. These initiatives support innovation, workforce development, and economic growth.
c. Scientific Progress
CTGCT has advanced research in RNA technologies, non-viral delivery, and CAR-T cell therapy regulation, with publications in 10 scientific articles in journals such as Nature Communications, ACS Nano, and Cell Discovery. It organized the CTGCT Conference 2024, two roundtables, and multiple panel discussions, promoting collaboration among researchers, clinicians, and patient organizations. CTGCT members have presented at several international scientific conferences, contributing to the field of gene and cell therapies.
CTGCT has contributed to patient care by participating in new regulations, including the 2024 proposal for the Regulation on Non-routine Preparation of Advanced Therapy Medicinal Products in Slovenia, now in inter-ministerial coordination. It has supported the development of Urbagen, a drug for CTNNB1 syndrome, which is currently in manufacturing. A roundtable on ATMP regulation examined the impact of existing regulations, the use of gene and cell therapies in Slovenia, the Netherlands, and Germany, and areas for regulatory improvement. CTGCT’s initiatives help accelerate advanced therapies, improve regulations, and enhance public and professional acceptance of gene and cell therapies.
b. Economic Growth
CTGCT has strengthened academia-industry collaboration, aiming to expand industrial partnerships and career opportunities. It has helped establish regional connections, leading to the AMMNIC project, a proposed postdoctoral training program on molecular disease research and therapy development, involving Slovene, Croatian, and Serbian partners. CTGCT is also part of EU Horizon Excellence Hub GeneH, working with the University of Coimbra to advance gene therapy research. These initiatives support innovation, workforce development, and economic growth.
c. Scientific Progress
CTGCT has advanced research in RNA technologies, non-viral delivery, and CAR-T cell therapy regulation, with publications in 10 scientific articles in journals such as Nature Communications, ACS Nano, and Cell Discovery. It organized the CTGCT Conference 2024, two roundtables, and multiple panel discussions, promoting collaboration among researchers, clinicians, and patient organizations. CTGCT members have presented at several international scientific conferences, contributing to the field of gene and cell therapies.