A Paradox of the Field's Own Success: Unintended Challenges in Bringing Cutting-Edge Science from the Bench to the Market

Author:

Yingling Nathan1,Sena-Esteves Miguel12,Gray-Edwards Heather L.13

Affiliation:

1. Horae Gene Therapy Center, UMass Chan Medical School, Worcester, Massachusetts, USA.

2. Department of Neurology, UMass Chan Medical School, Worcester, Massachusetts, USA.

3. Department of Radiology, UMass Chan Medical School, Worcester, Massachusetts, USA.

Publisher

Mary Ann Liebert Inc

Reference31 articles.

1. September 14, 1990: The Beginning

2. 3. Barrett D, Wendland A, Rose D, et al. Gene, Cell, + RNA Therapy Landscape Report Q3 2023 Quarterly Data Report. American Society of Gene & Cell Therapy-Waukesha, Wisconsin; 2023.

3. 5. United States Food & Drug Administration. Statement from FDA Commissioner Scott Gottlieb, M.D. and Peter Marks, M.D., Ph.D. Director of the Center for Biologics Evaluation and Research on New Policies to Advance Development of Safe and Effective Cell and Gene Therapies. 2019.

4. Estimating the Financial Impact of Gene Therapy in the U.S.

5. 9. Marsden G, Towse A, Pearson SD, et al. Gene Therapy: Understanding the Science, Assessing the Evidence, and Paying for Value. Institute for Clinical and Economic Review, Boston, Massachusetts; 2017.

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