[Federal Register Volume 89, Number 20 (Tuesday, January 30, 2024)]
[Notices]
[Pages 5913-5914]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2024-01788]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2021-D-0398]


Human Gene Therapy Products Incorporating Human Genome Editing; 
Guidance for Industry; Availability

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of availability.

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SUMMARY: The Food and Drug Administration (FDA or Agency) is announcing 
the availability of a final guidance entitled ``Human Gene Therapy 
Products Incorporating Human Genome Editing; Guidance for Industry.'' 
The guidance document provides recommendations to sponsors developing 
human gene therapy products incorporating genome editing (GE) of human 
somatic cells. Specifically, the guidance provides recommendations 
regarding information that should be provided in an investigational new 
drug (IND) application to assess the safety and quality of the 
investigational GE product, including information on product design, 
product manufacturing and testing, nonclinical safety assessment, and 
clinical trial design. The guidance announced in this notice finalizes 
the draft guidance of the same title dated March 2022.

DATES: The announcement of the guidance is published in the Federal 
Register on January 30, 2024.

ADDRESSES: You may submit either electronic or written comments on 
Agency guidances at any time as follows:

Electronic Submissions

    Submit electronic comments in the following way:
     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

    Submit written/paper submissions as follows:
     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 No. 
FDA-2021-D-0398 for ``Human Gene Therapy Products Incorporating Human 
Genome Editing.'' Received comments 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.'' 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 https://www.regulations.gov. 
Submit both copies to the Dockets Management Staff. 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: 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.
    You may submit comments on any guidance at any time (see 21 CFR 
10.115(g)(5)).
    Submit written requests for single copies of the guidance to the 
Office of Communication, Outreach and Development, Center for Biologics 
Evaluation and Research (CBER), Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 3128, Silver Spring, MD 20993-0002. Send 
one self-addressed adhesive label to assist the office in processing 
your requests. The guidance may also be obtained by mail by calling 
CBER at 1-800-835-4709 or 240-402-8010. See

[[Page 5914]]

the SUPPLEMENTARY INFORMATION section for electronic access to the 
guidance document.

FOR FURTHER INFORMATION CONTACT: Tami Belouin, Center for Biologics 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 71, Rm. 7301, Silver Spring, MD 20993-0002, 240-
402-7911.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the availability of a document entitled ``Human 
Gene Therapy Products Incorporating Human Genome Editing; Guidance for 
Industry.'' The guidance document provides recommendations to sponsors 
developing human gene therapy products incorporating GE of human 
somatic cells. Specifically, the guidance provides recommendations 
regarding information that should be provided in an IND application to 
assess the safety and quality of the investigational GE product, 
including information on product design, product manufacturing and 
testing, nonclinical safety assessment, and clinical trial design.
    In the Federal Register of March 16, 2022 (87 FR 14897), FDA 
announced the availability of the draft guidance of the same title 
dated March 2022. FDA received numerous comments on the draft guidance 
and those comments were considered as the guidance was finalized. 
Changes to the guidance include clarifying the recommendations for GE 
components used only once (for example, in the manufacture of a master 
cell bank), expectations for potency assays, considerations for 
nonclinical studies with respect to potential for off-target toxicity, 
and applicability of accelerated approval to GE products. In addition, 
editorial changes were made to improve clarity. The guidance announced 
in this notice finalizes the draft guidance dated March 2022.
    Elsewhere in this issue of the Federal Register, FDA is announcing 
the availability of another human gene therapy final guidance document 
entitled ``Considerations for the Development of Chimeric Antigen 
Receptor (CAR) T Cell Products; Guidance for Industry.''
    This guidance is being issued consistent with FDA's good guidance 
practices regulation (21 CFR 10.115). The guidance represents the 
current thinking of FDA on ``Human Gene Therapy Products Incorporating 
Human Genome Editing.'' It does not establish any rights for any person 
and is not binding on FDA or the public. You can use an alternative 
approach if it satisfies the requirements of the applicable statutes 
and regulations.

II. Paperwork Reduction Act of 1995

    While this guidance contains no collection of information, it does 
refer to previously approved FDA collections of information. The 
previously approved collections of information are subject to review by 
the Office of Management and Budget (OMB) under the Paperwork Reduction 
Act of 1995 (44 U.S.C. 3501-3521). The collections of information in 21 
CFR part 312 have been approved under OMB control number 0910-0014; the 
collections of information in 21 CFR parts 210 and 211 have been 
approved under OMB control number 0910-0073; the collections of 
information in 21 CFR part 1271 have been approved under OMB control 
number 0910-0543.

III. Electronic Access

    Persons with access to the internet may obtain the guidance at 
https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances, https://www.fda.gov/regulatory-information/search-fda-guidance-documents, or 
https://www.regulations.gov.

    Dated: January 24, 2024.
Lauren K. Roth,
Associate Commissioner for Policy.
[FR Doc. 2024-01788 Filed 1-29-24; 8:45 am]
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