Cell- and tissue-based therapies and gene therapies hold great promise in the field of both immunotherapy and regenerative medicine. A high-quality Cell Therapy Facility (CTF) infrastructure at the Utrecht Science Park will accelerate innovative cellular therapy, gene therapy and vaccine research and supports highest standard of care for patients in The Netherlands at reasonable costs. Many of these innovative medicinal products (Advanced Therapy Medicinal Products and Stem cell products) are manufactured through cell and tissue engineering and therefore development raises a unique set of biological, technological, logistical and societal issues that make them significantly different from conventional medicinal products. At the Utrecht CTF, key expertise from all domains will be centralized and utilize state-of-the-art equipment to form the bridge between concept and clinical development.
A national hub for ATMP development and Cellular therapy.
The emerging paradigm of personalized medicine has been a driving force for the development of Advanced Medicinal Therapeutics Products (AMTPs) at an accelerating pace in the past five years. Whereas the recent focus was to understand their fundamentals and potential, the field is now ready to deliver these products to the patient.
The Utrecht CTF infrastructure will drive the transition of personalized and universal immune-oncology and regenerative medicine solutions into clinical care as outlined in the Dutch National Research Agenda. We envision achieving this by: 1) establishing a central hub for stem cell and ATMP development where researchers, clinicians and pharma can collectively develop and evaluate new medicinal concepts; 2) increasing the capacity of Good Manufacturing Practice (GMP)-grade facilities that meet the state-of-the art requirements of stem cell processing and ATMP production.
Highly specialized stem cell processing and ATMP development and production centers are sought after worldwide, driven by a need to efficiently make promising cell-based therapies available to patients. Academic centers that upgrade their infrastructure can not only develop and evaluate new cellular therapies in collaboration with pharmaceutical industries but can also provide these products to patients at reasonable costs. Utrecht CTF will aim to be one of the key infrastructures in the Netherlands and Europe and will be connected to European and world leading ATMP development centers. The Utrecht CTF hub will significantly contribute to strengthen the network of comparable national and international infrastructures.
The aim of the CTF is a smooth transition from research to efficient, safe and affordable certified production. The CTF serves as hub for innovation for novel and cost-effective products and processes. This will be achieved by:
• bringing together all stakeholders that are involved in cellular drug development and daily clinical practice and form a translational drug development team. This team would include pre-clinical developers; clinicians; experts on intellectual property; pharmaceutical experts of quality and GMP, product developers; regulatory and health technology experts, health economy experts and payers.
• enabling efficient development of innovative ATMP concepts into a GMP-grade product ready for testing in phase I/II clinical studies by bringing together the stakeholders. It is crucial to bring the drug development team early in the development process together with a team of experts that are experienced in product development awareness regarding simplicity, GMP and end-user requirements. As such the change for later successful medicinal product but also partnering with industry and final market approval and accessibility for the patient will be dramatically increased.
• increasing critical GMP cleanroom capacity and state-of-the art equipment for the production for clinical studies in NL and EU.
• increasing the involvement of the pharmaceutical industry by offering professional expertise in ATMP development, high quality infrastructures including cleanroom capacity and access to clinicians and patient populations.
• improving interactions with regulators, policy makers including EMA by offering a professional ATMP expertise team including regulatory experts in order to improve product research and development.
Finally, CTF will function as center for training next generation ATMP developers and can be incorporated in PhD, PharmD, MD study programmes at the Utrecht Science Park. CTF aims for continued knowledge exchange and transparency. CTF will make production and clinical outcome data publicly available through close collaboration with European registries (i.e. EBMT), also when no clinical effects are observed, clinical endpoints not reached or during hospital exemption programs.