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I am a member of the National Academies Forum on Regenerative Medicine, which was established in 2016. The forum convenes people from academia, industry, government, patient and provider organizations, regulators, foundations, and others to address the challenges facing the applications and opportunities for regenerative medicine. The charge to forum members is to identify barriers to scientific advances and to examine the impact of current policies on advancing discovery, development, and translation of effective therapies.

We meet four times a year, and agenda topics span a range of issues using public workshops, discussions, and commissioned papers to inform the field. I’d like to share the highlights from the four previous forum workshops.

  1. Exploring the State of the Science in the Field of Regenerative Medicine: Challenges of and Opportunities for Cellular Therapies (October 2016)

Several themes emerged from the workshop that highlighted challenges and areas of opportunity, as well as future innovation including understanding and characterizing cells, improving model systems, clinical translation, navigating regulatory pathways, and more.

  1. Navigating the Manufacturing Process and Enduring the Quality of Regenerative Medicine Therapies (June 2017)

This workshop focused on the challenges, opportunities, and best practices for defining and measuring the quality of cell and tissue products, as well as the raw materials in research and manufacturing of regenerative medicine therapies.

  1. Exploring Sources of Variability Related to the Clinical Translation of Regenerative Medicine Products (October 2018)

The forum used the Regenerative Engineering Society’s definition of regenerative engineering as the convergence of advanced materials sciences, stem cell science, physics, developmental biology, and clinical translation for the regeneration of complex tissues and organ systems.

Forum speakers highlighted the many factors that can affect the variability of regenerative engineering products, including the immune system, disease state, and other characteristics of the patient and the donor, the manufacturing process, and preservation techniques.

  1. Exploring Novel Clinical Trial Designs for Gene-Based Therapies (November 2019)

The workshop focused on gaining an understanding of the design complexities and ethical issues associated with clinical trials for gene-based therapies.

This workshop drew attention to a few points. First, that clinically meaningful, reliable, and rigorous endpoints are important for gene therapy trials, where trials may be smaller, and treatments are irreversible. Additionally, long-term follow-up is critical to identifying and mitigating delayed risks to patients who receive investigational gene therapies.

The forum continues its work and has established a working group on systems thinking, with a goal to examine systems-based analytical methods that could advance the mechanistic understanding of regenerative medicine products to overcome challenges with manufacturing and patient outcomes.

Additional workshop perspectives will be forthcoming. It has been an amazing and rewarding opportunity for me to participate in the Academies Forum on Regenerative Medicine.

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