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  4. Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Announcement - 09/02/2021 - 09/03/2021
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Advisory Committee Meeting

Event Title
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Announcement
September 2 - 3, 2021

Scheduled

Date:
September 2 - 3, 2021
Day1:
- ET
Day2:
- ET


Please note that due to the impact of this COVID-19 pandemic, all meeting participants will be joining this advisory committee meeting via an online teleconferencing platform.

The online web conference meeting will be available at the following: 

Day 1: https://youtu.be/58KjL9_p9Tw
Day 2: https://youtu.be/yLggQF0XUUY

Day 1:

Day 2:

Agenda
The meeting presentations will be heard, viewed, captioned, and recorded through an online teleconferencing platform. The CTGTAC committee will meet in open session on both days to discuss the toxicity risks of adeno-associated virus (AAV) vector-based gene therapy products. The discussion topics include oncogenicity risks due to vector genome integration and safety issues identified during preclinical and/or clinical evaluation. On September 2, 2021, in the morning, under session 1, CTGTAC committee will meet to discuss and make recommendations on vector integration and oncogenicity risks. In the afternoon, under session 2, the committee will discuss and make recommendations on hepatotoxicity issues. On September 3, 2021, in the morning, under session 3, the committee will meet to discuss and make recommendations on thrombotic microangiopathy issues. In the afternoon, under session 4, the committee will discuss and make recommendations on non-clinical findings of neurotoxicity, especially related to the dorsal root ganglion toxicity issues.  Also, in the afternoon, under session 5, the committee will discuss and make recommendations on clinical findings of neurotoxicity, based on brain MRI studies.

Meeting Materials
Interested persons may present data, information, or views, orally or in writing, on issues pending before the committee.
FDA is establishing a docket for public comment on this meeting. The docket number is FDA-2021-N-0651.

The docket will close on September 1, 2021. Please note that late, untimely filed comments will not be considered. The https://www.regulations.gov electronic filing system will accept comments until 11:59 p.m. Eastern Time at the end of September 1, 2021. Comments received by mail/hand delivery/courier (for written/paper submissions) will be considered timely if they are postmarked or the delivery service acceptance receipt is on or before that date.

Comments received on or before August 26, 2021 will be provided to the committee. Comments received after August 26, 2021, and by September 1, 2021, will be taken into consideration by FDA. In the event that the meeting is cancelled, FDA will continue to evaluate any relevant applications, submissions, or information, and consider any comments submitted to the docket, as appropriate.

You may submit comments as follows:
Electronic Submissions

  • Federal eRulemaking Portal: https://www.regulations.gov. Follow the instructions for submitting comments. Comments submitted electronically, including attachments, to https://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 https://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

  • Mail/Hand delivery/Courier (for written/paper submissions):  Dockets Management Staff (HFA-305), Food and Drug Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
  • For written/paper comments submitted to the Dockets Management Staff, 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 number FDA-2021-N-0651 for “Cellular, Tissue, and Gene Therapies Advisory Committee; Notice of Meeting; Establishment of a Public Docket; Request for Comments.” Received comments, those filed in a timely manner (see ADDRESSES), will be placed in the docket and, except for those submitted as “Confidential Submissions,” publicly viewable at https://www.regulations.gov or at the Dockets Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-402-7500.

  • 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.” FDA 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 https://www.regulations.gov. Submit both copies to the Dockets Management Staff. If you do not wish your name and contact information 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 the 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: https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf.

Docket: For access to the docket to read background documents or the electronic and written/paper comments received, go to https://www.regulations.gov and insert the docket number, found in brackets in the heading of this document, into the “Search” box and follow the prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, 240-402-7500.

FOR FURTHER INFORMATION CONTACT: Jarrod Collier or Joanne Lipkind, Center for Biologics Evaluation and Research, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 71, Rm. 6306, Silver Spring, MD 20993-0002, via e-mail at CTGTAC@fda.hhs.gov; or FDA Advisory Committee Information Line, 1-800-741-8138 (301-443-0572 in the Washington, DC area). A notice in the Federal Register about last minute modifications that impact a previously announced advisory committee meeting cannot always be published quickly enough to provide timely notice. Therefore, you should always check the Agency's website and scroll down to the appropriate advisory committee meeting link, or call the advisory committee information line to learn about possible modifications before joining the meeting.

On September 2, 2021, oral presentations from the public will be scheduled twice each day between approximately 12:45 p.m. and 1:15 p.m. and 4:05 p.m. and 4:35 p.m. on September 2, and between 11 a.m. and 11:30 a.m. and 1:50 p.m. and 2:20 p.m. on September 3 Eastern Time. 

Those individuals interested in making formal oral presentations should notify CTGTAC@fda.hhs.gov and submit a brief statement of the general nature of the evidence or arguments they wish to present, the names and addresses of proposed participants, and an indication of the approximate time requested to make their presentation on or before August 18, 2021.

Time allotted for each presentation may be limited. If the number of registrants requesting to speak is greater than can be reasonably accommodated during the scheduled open public hearing session, FDA may conduct a lottery to determine the speakers for the scheduled open public hearing session. The contact person will notify interested persons regarding their request to speak by August 19, 2021.

Webcast Information
CBER plans to provide a free of charge, live webcast of the Cellular, Tissue, and Gene Therapies Advisory Committee meeting. If there are instances where the webcast transmission is not successful; staff will work to re-establish the transmission as soon as possible.

The online web conference meeting will be available at the following links:
Day 1: https://youtu.be/58KjL9_p9Tw
Day 2: https://youtu.be/yLggQF0XUUY

Contact Information

  • Jarrod Collier or Joanne Lipkind:CTGTAC@fda.hhs.gov
  • FDA Advisory Committee Information Line: 1-800-741-8138 (301-443-0572 in the Washington, DC area) Please call the Information Line for up-to-date information on this meeting.
  • For press inquiries, please contact the Office of Media Affairs at fdaoma@fda.hhs.gov or 301-796-4540.

A notice in the Federal Register about last minute modifications that impact a previously announced advisory committee meeting cannot always be published quickly enough to provide timely notice. Therefore, you should always check the meetings main page or call the appropriate advisory committee hot line/phone line to learn about possible modifications before coming to the meeting.

FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our website for procedures on public conduct during advisory committee meetings.

Notice of this meeting is given under the Federal Advisory Committee Act (5 U.S.C. app. 2).

Official FR Notice


A notice in the Federal Register about last minute modifications that impact a previously announced advisory committee meeting cannot always be published quickly enough to provide timely notice. Therefore, you should always check the agency’s website or call the committee’s Designated Federal Officer (see Contact Information) to learn about possible modifications before coming to the meeting.

Persons attending FDA’s advisory committee meetings are advised that the agency is not responsible for providing access to electrical outlets. FDA welcomes the attendance of the public at its advisory committee meetings and will make every effort to accommodate persons with disabilities. If you require accommodations due to a disability, please contact the committee’s Designated Federal Officer (see Contact Information) at least 7 days in advance of the meeting.

Answers to commonly asked questions including information regarding special accommodations due to a disability may be accessed at: Common Questions and Answers about FDA Advisory Committee Meetings.

FDA is committed to the orderly conduct of its advisory committee meetings. Please visit our Web site at Public Conduct During FDA Advisory Committee Meetings for procedures on public conduct during advisory committee meetings.

Notice of this meeting is given under the Federal Advisory Committee Act (5 U.S.C. app.2).


Event Materials

Title File Type/Size Source Organization
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Briefing Document- FDA pdf (1.19 MB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Acknowledgement of Financial Interests- Charles Vite pdf (30.65 KB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Acknowledgement of Financial Interests- Kenneth Berns pdf (52.80 KB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Acknowledgement of Financial Interests- Roland Herzog pdf (54.19 KB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Waiver to Allow Participation- Charles Vite pdf (59.14 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Waiver to Allow Participation- Kenneth Berns pdf (75.38 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Waiver to Allow Participation- Roland Herzog pdf (85.05 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Briefing Document- FDA Errata pdf (47.42 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Draft Agenda pdf (66.76 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Discussion Questions pdf (75.30 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Draft Roster pdf (153.58 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Waiver to Allow Participation- Barry Byrne pdf (38.90 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2-3, 2021 Meeting Acknowledgement of Financial Interests- Barry Byrne pdf (85.42 KB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2, 2021 Meeting Presentation - Adeno-Associated Virus-Related Toxicities in Nonhuman Primates pdf (2.11 MB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2, 2021 Meeting Presentation - rAAV Integration: In Vitro & Mice pdf (782.98 KB) Non-FDA
Cellular, Tissue. and Gene Therapies Advisory Committee September 2, 2021 Meeting Presentation - AAV Integration Studies in Large Animal Models: Non-Human Primates and Dogs pdf (899.88 KB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2, 2021 Meeting Presentation- Toxicity Risks of Adeno-associated Virus (AAV) Vectors for Gene Therapy (GT) pdf (758.82 KB) FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 2, 2021 Meeting Presentation - Systemic AAV: Clinical Findings of Hepatotoxicity pdf (1.15 MB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 3, 2021 Meeting Presentation- Clinical and Nonclinical Consequences of Direct CNS Parenchymal Administration of AAV Vectors pdf (1.19 MB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 3, 2021 Meeting Presentation - Clinical Findings of Thrombotic Microangiopathy (TMA) pdf (600.06 KB) Non-FDA
Cellular, Tissue, and Gene Therapies Advisory Committee September 3, 2021 Meeting Presentation - Nonclinical Findings of Dorsal Root Ganglion, Spinal Cord, and Peripheral Nerve Toxicities pdf (3.63 MB) Non-FDA

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