Skip to main content
Proceedings

VIEW LARGER COVER

Recognizing the potential design complexities and ethical issues associated with clinical trials for gene therapies, the Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine held a 1-day workshop in Washington, DC, on November 13, 2019. Speakers at the workshop discussed patient recruitment and selection for gene-based clinical trials, explored how the safety of new therapies is assessed, reviewed the challenges involving dose escalation, and spoke about ethical issues such as informed consent and the role of clinicians in recommending trials as options to their patients. The workshop also included discussions of topics related to gene therapies in the context of other available and potentially curative treatments, such as bone marrow transplantation for hemoglobinopathies. This publication summarizes the presentation and discussion of the workshop.

Suggested Citation

National Academies of Sciences, Engineering, and Medicine. 2020. Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/25712.

Import this citation to:

Publication Info

126 pages |  6 x 9 | 

ISBNs: 
  • Paperback:  978-0-309-67298-6
  • Ebook:  978-0-309-67301-3
DOI: https://doi.org/10.17226/25712

Copyright Information

The National Academies Press (NAP) has partnered with Copyright Clearance Center's Marketplace service to offer you a variety of options for reusing NAP content. Through Marketplace, you may request permission to reprint NAP content in another publication, course pack, secure website, or other media. Marketplace allows you to instantly obtain permission, pay related fees, and print a license directly from the NAP website. The complete terms and conditions of your reuse license can be found in the license agreement that will be made available to you during the online order process. To request permission through Marketplace you are required to create an account by filling out a simple online form. The following list describes license reuses offered by the NAP through Marketplace:

  • Republish text, tables, figures, or images in print
  • Post on a secure Intranet/Extranet website
  • Use in a PowerPoint Presentation
  • Distribute via CD-ROM
  • Photocopy

Click here to obtain permission for the above reuses. If you have questions or comments concerning the Marketplace service, please contact:

Marketplace Support
International +1.978.646.2600
US Toll Free +1.855.239.3415
E-mail: support@copyright.com
marketplace.copyright.com

To request permission to distribute a PDF, please contact our Customer Service Department at customer_service@nap.edu.

loading iconLoading stats for Exploring Novel Clinical Trial Designs for Gene-Based Therapies: Proceedings of a Workshop...