Workshop | Virtual

Event Title

Cell Therapies and Tissue-Based Products: A Public Workshop on Generating Scientific Evidence to Facilitate Development

February 25, 2025

Date:: February 25, 2025
Time:: 8:30 a.m. - 5:00 p.m. ET

The FDA’s Center for Biologics Evaluation and Research (CBER), Office of Therapeutic Products (OTP) hosted a virtual scientific public workshop on February 25, 2025, titled “Cell Therapies and Tissue-based Products: A Public Workshop on Generating Scientific Evidence to Facilitate Development.” The purpose of the workshop was to identify and discuss the current state of the science, development, and regulation for cellular therapies and tissue-based products.

View the recording from the workshop.

FDA convened this public workshop with relevant stakeholders to discuss best practices on generating scientific data necessary to further facilitate the development of stem cell and other cellular therapies. Within six months after the meeting, FDA will issue a transcript of the meeting. This workshop fulfilled the requirement under the Food and Drug Omnibus Reform Act of 2022, section 3205. FDA also published a notice in the Federal Register and opened a docket for public comments.

Agenda

8:30 a.m. - 8:45 a.m. ET	Welcome and Opening Remarks from FDA Peter Marks, MD, PhD, Director, Center for Biologics Evaluation and Research (CBER), FDA Nicole Verdun, MD, Super Office Director, Office of Therapeutic Products (OTP), CBER, FDA
8:45 a.m. - 9:15 a.m. ET	Keynote Address Sally Temple, PhD, Scientific Director, The Neural Stem Cell Institute
9:15 a.m. - 10:40 a.m. ET	Session 1: Tissue- and Cell-Derived Therapies Amanda Olson, MD, Associate Professor of Medicine, MD Anderson Cancer Center Katie Mowry, PhD, Vice President of Research and Development, Organogenesis Steven Stice, PhD, Co-Founder and Chief Scientific Officer, Aruna Bio, Inc. Moderator: Elizabeth Lessey-Morillon, PhD, Office of Cellular Therapy and Human Tissue CMC (OCTHT), OTP, CBER, FDA
10:40 a.m. - 10:50 a.m. ET	BREAK
10:50 a.m. - 12:15 p.m. ET	Session 2: Nonclinical Considerations for Cell Therapy Product Development Leslie Thompson, PhD, Professor, University of California, Irvine Kapil Bharti, PhD, Senior Investigator and Scientific Director, National Eye Institute, National Institutes of Health Jeanne Loring, PhD, Co-Founder, Aspen Neuroscience and Professor Emeritus, Scripps Research Moderator: Maria Torruella Suarez, PhD, Office of Pharmacology/Toxicology, OTP, CBER, FDA
12:15 p.m. – 12:45 p.m. ET	BREAK / Lunch
12:45 p.m. - 2:10 p.m. ET	Session 3: CMC Considerations for Cell Therapy Characterization Presentations by: Evan Snyder, MD, PhD, Director of the Stem Cell Research Center and Professor, Sanford Burnham Prebys Medical Discovery Institute Deborah Hursh, PhD, Principal, Hursh Cell Therapy Consulting, LLC Jane Lebkowski, PhD, President, Regenerative Patch Technologies Moderator: Thomas Finn, PhD, OCTHT, OTP, CBER, FDA
2:10 p.m. - 2:15 p.m. ET	BREAK
2:15 p.m. – 3:40 p.m. ET	Session 4: Cell Therapies for Niche Indications: Clinical Insights and Future Directions Presentations by: Blake Boggess, DO, Professor, Duke University School of Medicine; Duke Sports Medicine Team Physician Ellen Koo, MD, Associate Professor of Clinical Ophthalmology, Bascom Palmer Eye Institute, University of Miami Health System Eduardo Marbán, MD, PhD, Executive Director, Smidt Heart Institute, Mark S. Siegel Family Foundation Distinguished Professor Moderator: Vaishali Popat, MD, Office of Clinical Evaluation, OTP, CBER, FDA
3:40 p.m. - 3:45 p.m. ET	BREAK
3:45 p.m. - 4:30 p.m. ET	Session 5: Considerations for a Revised Risk-Based HCT/P Framework Julie Tierney, JD, Deputy Center Director, CBER, FDA Melissa A. Greenwald, MD, FAST, Chief Medical Officer, American Association of Tissue Banks Moderator: Heather Lombardi, PhD, OCTHT, OTP, CBER, FDA
4:30 p.m. - 4:40 p.m. ET	Insights and Remarks from Keynote Speaker Sally Temple, PhD, Scientific Director, The Neural Stem Cell Institute
4:40 p.m. – 4:45 p.m. ET	Closing Remarks from FDA

Submitting Electronic or Written Comments

In addition to holding the virtual public workshop, FDA opened a docket on January 17, 2025 and is accepting electronic or written comments. Electronic or written comments on this workshop must be submitted no later than 11:59 p.m. ET on March 18, 2025.

Electronic Submissions

Submit electronic comments in the following way:

Federal eRulemaking Portal: visit http://www.regulations.gov and type FDA-2025-N-0121 in the search box. For assistance in submitting electronic comments, please see regulations.gov/help. Comments submitted electronically, including attachments, to http://www.regulations.gov will be posted to the docket unchanged. Because your comment will be made public, you are solely responsible for ensuring that your comment does not include any confidential information that you or a third party may not wish to be posted, such as medical information, your or anyone else’s Social Security number, or confidential business information, such as a manufacturing process. Please note that if you include your name, contact information, or other information that identifies you in the body of your comments, that information will be posted on http://www.regulations.gov.
If you want to submit a comment with confidential information that you do not wish to be made available to the public, submit the comment as a written/paper submission and in the manner detailed (see “Written/Paper Submissions” and “Instructions”).

Written/Paper Submissions

Submit written/paper submissions as follows:

Mail/Hand delivery/Courier (for written/paper submissions): Division of Dockets Management (HFA-305), Food and Drug Administration, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852.
For written/paper comments submitted to the Division of Dockets Management, FDA will post your comment, as well as any attachments, except for information submitted, marked and identified, as confidential, if submitted as detailed in “Instructions.”
Instructions: All submissions received must include the Docket No. FDA-2025-N-0121 for “Cell Therapies and Tissue-Based Products: A Public Workshop on Generating Scientific Evidence to Facilitate Development; Public Workshop; Request for Comments.” Received comments will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at http://www.regulations.gov or at the Division of Dockets Management between 9 a.m. and 4 p.m., Monday through Friday.
Confidential Submissions--To submit a comment with confidential information that you do not wish to be made publicly available, submit your comments only as a written/paper submission. You should submit two copies total. One copy will include the information you claim to be confidential with a heading or cover note that states “THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.” The Agency will review this copy, including the claimed confidential information, in its consideration of comments. The second copy, which will have the claimed confidential information redacted/blacked out, will be available for public viewing and posted on http://www.regulations.gov. Submit both copies to the Division of Dockets Management. If you do not wish your name and contact information to be made publicly available, you can provide this information on the cover sheet and not in the body of your comments, and you must identify this information as “confidential.” Any information marked as “confidential” will not be disclosed except in accordance with 21 CFR 10.20 and other applicable disclosure law. For more information about FDA’s posting of comments to public dockets, see 80 FR 56469, September 18, 2015, or access the information at: http://www.fda.gov/regulatoryinformation/dockets/default.htm.

For access to the docket to read background documents or the electronic and written/paper comments received, go to http://www.regulations.gov and insert the docket number, FDA-2025-N-0121, into the “Search” box and follow the prompts and/or go to the Division of Dockets Management, 5630 Fishers Lane, rm. 1061, Rockville, MD 20852.

Contact

For questions regarding the public workshop, please contact Claire Simon at Claire.Simon@fda.hhs.gov.

U.S. Food and Drug Administration