Advanced Therapy Medicinal Products (ATMPs) have huge potential to be game changers in the treatment of chronic diseases. Instead of simply treating the symptoms of disease, these medicines offer the promise of a sustainable improvement or even cure for countless patients urgently needing new therapeutic options. This has been impressively demonstrated with recently approved gene therapies for rare diseases and CAR T cell therapies for haematological cancers. Despite the enormous potential of ATMPs as curative medicines for a wide range of chronic diseases, so far, very few products have made it to the market and consequently to patients.
Thus, in addition to scientific breakthroughs, the development and ultimately the availability of ATMPs in healthcare systems requires novel and collaborative thinking also in pre-clinical and clinical testing, safety assays, technical, manufacturing, engineering, regulatory, ethical, economic and commercial fields as well as changes to infrastructure. Large-scale collaborations across multiple disciplines and between research groups worldwide are essential and was the reason behind founding the RESTORE initiative.
The preparatory action for a large-scale research initiative, RESTORE (funded by the European Union’s Horizon 2020 research and innovation programme under grant agreement No 820292, https://www.restore-horizon.eu/) has been running since March 2019 with the aim of promoting the development of ATMPs and their implementation into patient care in Europe and beyond. ATMPs break the mould of classical drug development. Unfortunately, many of the well-established regulations and milestones in classical drug development are no longer useful in the case of ATMPs, for example drug discovery, preclinical testing, conventional drug manufacturing and delivery processes, clinical development plans and pay-per-pill payment models. Thus, bringing ATMPs to the clinic in a sustainable and affordable fashion requires not just a paradigm shift in manufacturing and regulatory processes, but also in the mindset behind traditional translational research, medical reimbursement models and healthcare policies. This will not be the work of individuals, or even single organisations, but rather will require a concerted effort to drive the disruptive innovation necessary for ATMP development and in an ideal world make these available to all those in need of such therapies. Indeed, RESTORE has endeavoured to drive this concerted effort through the establishment of several working groups covering a wide range of topics with the contributions of RESTORE supporters from a variety of disciplines, details of which can be found on the RESTORE website (https://www.restore-horizon.eu/public-deliverables/).
The aim therefore of this topic is to not only give an overview on the challenges in implementation of ATMPs, but to also open up the discussion at all levels of research, including within the value chain to encourage collaboration and consideration of the requirements for successful ATMP development from conception to a commercially viable product.
To streamline ATMP development it is necessary to identify the most pressing issues in bringing ATMPs into the clinic, gathering together original research at the cutting edge of ATMP development for translational applications in all areas from basic science, technical/manufacturing, engineering, regulatory, and economics/commercial issues to infrastructure.
Suggested areas for article submission that cover, but are not limited to, the following topics:
1) Concepts
2) ATMP Pipeline
• New oncologic and non-oncologic targets for specific cell (and gene) therapies
• Off-the shelf allogeneic cell therapies – challenges and solutions
• MSCs from different sources- similarities and differences
• iPSC – how to harness their power as universal allogeneic resources for Advanced Therapies ?
• Gene therapy- challenges in viral and non-viral vector development and gene editing for in vivo/ex vivo gene therapy
3) Manufacturing
• GMP cell sorting
• Manufacturing issues, advanced fabrication procedures (e.g. 3D printing, automated closed systems) and scaling-up
• Product characterization and release assays
• Tissue engineering and composite products
4) Preclinical Testing
• Advanced in vitro models: human (multi)-organ-on-a-chip technologies
• Advanced humanized animal models
5) Clinical Trials
• Design of early and late clinical trials
• Refined translation
• Regulatory science for ATMPs
• Mechanistic side studies and biomarkers for safety, PK/PD, mode-of-action, stratification of patients
6) Out-scaling and implementation into healthcare
• Considerations for choosing the right mode of delivery for “living” cell therapy products
• Expansion of therapeutic options from rare disease and hematologic cancer to larger clinical indications
• Health Technology Assessment, health economics and reimbursement
• Patient view and patient engagement in the development of ATMPs
• Models for the development of ATMPs from preclinical research to market authorization
• CAR T cell therapies: Status in Europe and lessons learned
• Ethical considerations
In this research topic we welcome the submission of: Original Research, Systematic Review, Methods, Review, Mini Review, Hypothesis and Theory, Perspective
Advanced Therapy Medicinal Products (ATMPs) have huge potential to be game changers in the treatment of chronic diseases. Instead of simply treating the symptoms of disease, these medicines offer the promise of a sustainable improvement or even cure for countless patients urgently needing new therapeutic options. This has been impressively demonstrated with recently approved gene therapies for rare diseases and CAR T cell therapies for haematological cancers. Despite the enormous potential of ATMPs as curative medicines for a wide range of chronic diseases, so far, very few products have made it to the market and consequently to patients.
Thus, in addition to scientific breakthroughs, the development and ultimately the availability of ATMPs in healthcare systems requires novel and collaborative thinking also in pre-clinical and clinical testing, safety assays, technical, manufacturing, engineering, regulatory, ethical, economic and commercial fields as well as changes to infrastructure. Large-scale collaborations across multiple disciplines and between research groups worldwide are essential and was the reason behind founding the RESTORE initiative.
The preparatory action for a large-scale research initiative, RESTORE (funded by the European Union’s Horizon 2020 research and innovation programme under grant agreement No 820292, https://www.restore-horizon.eu/) has been running since March 2019 with the aim of promoting the development of ATMPs and their implementation into patient care in Europe and beyond. ATMPs break the mould of classical drug development. Unfortunately, many of the well-established regulations and milestones in classical drug development are no longer useful in the case of ATMPs, for example drug discovery, preclinical testing, conventional drug manufacturing and delivery processes, clinical development plans and pay-per-pill payment models. Thus, bringing ATMPs to the clinic in a sustainable and affordable fashion requires not just a paradigm shift in manufacturing and regulatory processes, but also in the mindset behind traditional translational research, medical reimbursement models and healthcare policies. This will not be the work of individuals, or even single organisations, but rather will require a concerted effort to drive the disruptive innovation necessary for ATMP development and in an ideal world make these available to all those in need of such therapies. Indeed, RESTORE has endeavoured to drive this concerted effort through the establishment of several working groups covering a wide range of topics with the contributions of RESTORE supporters from a variety of disciplines, details of which can be found on the RESTORE website (https://www.restore-horizon.eu/public-deliverables/).
The aim therefore of this topic is to not only give an overview on the challenges in implementation of ATMPs, but to also open up the discussion at all levels of research, including within the value chain to encourage collaboration and consideration of the requirements for successful ATMP development from conception to a commercially viable product.
To streamline ATMP development it is necessary to identify the most pressing issues in bringing ATMPs into the clinic, gathering together original research at the cutting edge of ATMP development for translational applications in all areas from basic science, technical/manufacturing, engineering, regulatory, and economics/commercial issues to infrastructure.
Suggested areas for article submission that cover, but are not limited to, the following topics:
1) Concepts
2) ATMP Pipeline
• New oncologic and non-oncologic targets for specific cell (and gene) therapies
• Off-the shelf allogeneic cell therapies – challenges and solutions
• MSCs from different sources- similarities and differences
• iPSC – how to harness their power as universal allogeneic resources for Advanced Therapies ?
• Gene therapy- challenges in viral and non-viral vector development and gene editing for in vivo/ex vivo gene therapy
3) Manufacturing
• GMP cell sorting
• Manufacturing issues, advanced fabrication procedures (e.g. 3D printing, automated closed systems) and scaling-up
• Product characterization and release assays
• Tissue engineering and composite products
4) Preclinical Testing
• Advanced in vitro models: human (multi)-organ-on-a-chip technologies
• Advanced humanized animal models
5) Clinical Trials
• Design of early and late clinical trials
• Refined translation
• Regulatory science for ATMPs
• Mechanistic side studies and biomarkers for safety, PK/PD, mode-of-action, stratification of patients
6) Out-scaling and implementation into healthcare
• Considerations for choosing the right mode of delivery for “living” cell therapy products
• Expansion of therapeutic options from rare disease and hematologic cancer to larger clinical indications
• Health Technology Assessment, health economics and reimbursement
• Patient view and patient engagement in the development of ATMPs
• Models for the development of ATMPs from preclinical research to market authorization
• CAR T cell therapies: Status in Europe and lessons learned
• Ethical considerations
In this research topic we welcome the submission of: Original Research, Systematic Review, Methods, Review, Mini Review, Hypothesis and Theory, Perspective