Turning Regenerative Medicine Breakthrough Ideas and Innovations into Commercial Products

Abstract

The TERMIS-Europe (EU) Industry committee intended to address the two main critical issues in the clinical/commercial translation of Advanced Therapeutic Medicine Products (ATMP): (1) entrepreneurial exploitation of breakthrough ideas and innovations, and (2) regulatory market approval. Since January 2012, more than 12,000 publications related to regenerative medicine and tissue engineering have been accepted for publications, reflecting the intense academic research activity in this field. The TERMIS-EU 2014 Industry Symposium provided a reflection on the management of innovation and technological breakthroughs in biotechnology first proposed to contextualize the key development milestones and constraints of allocation of financial resources, in the development life-cycle of radical innovation projects. This was illustrated with the biofuels story, sharing similarities with regenerative medicine. The transition was then ensured by an overview of the key identified challenges facing the commercialization of cell therapy products as ATMP examples. Real cases and testimonies were then provided by a palette of medical technologies and regenerative medicine companies from their commercial development of cell and gene therapy products. Although the commercial development of ATMP is still at the proof-of-concept stage due to technology risks, changing policies, changing markets, and management changes, the sector is highly dynamic with a number of explored therapeutic approaches, developed by using a large diversity of business models, both proposed by the experience, pitfalls, and successes of regenerative medicine pioneers, and adapted to the constraint resource allocation and environment in radical innovation projects.

Clinical development of gene- and cell-based therapies: overview of the European landscape

“…6 However, despite this impressive scientific output, only few of the products have received marketing authorization approval, with the result that there is only limited benefit for patients. 6–8 Inherent product-technical aspects may have hampered clinical development, such as the availability of human starting material, or small-scale or even fully personalized manufacturing strategies.…”

The Progression of Regenerative Medicine and its Impact on Therapy Translation

“…13,23,33 Numerous reviews have been published on these challenges but with an emphasis on those relating to the clinical phase. 11,13,22,51 Although these will be summarized below, the present study highlights the identification and analysis of the preclinical challenges. Please see Figure 5 for a summary of the preclinical and clinical obstacles discussed herein.…”

“…7 Institutions, such as Canada's Center for Commercialization of Regenerative Medicine, have been launched to help researchers mitigate the risks of cell therapy development by offering technical as well as business services. 12,51 Experts are also stepping up; for example, Drs. Arnold Caplan and Michael West proposed a new regulatory pathway that incorporates large postmarket studies into clinical trials.…”

Clinical Translation of Tissue Engineered Medicinal Products

“…Another challenge for clinical transformation and commercialization of innovative therapeutics in India is that the country does not have higher entrepreneurial exploitation to create innovation, biomedical ecosystem in CTT sector, as it makes risk of burning money, time and manpower with no immediate returns. However, the innovative healthcare products and innovation is the prime generator of economic growth that forms prominent contribution for national GDP growth rate and serves the unmet medical needs [56] . Another challenge is dearth of commitment from the clinical community resulting in commercial failure of cell therapy products.…”