Notice2026-11054
Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing; Draft Guidance for Industry; Availability
Primary source
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Published
June 3, 2026
Issuing agencies
Health and Human Services DepartmentFood and Drug Administration
Abstract
The Food and Drug Administration (FDA, Agency, or we) is announcing the availability of a draft document entitled "Leveraging Prior Knowledge in the Development of Human Gene Therapy Products Incorporating Genome Editing." The draft guidance document provides sponsors engaged in the development of human gene therapy (GT) products incorporating ex-vivo and in vivo genome editing (GE) of human somatic cells (GE products) with FDA's recommendations on the type of prior knowledge that may be scientifically appropriate to leverage to advance product development.
Full Text
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<title>Federal Register, Volume 91 Issue 106 (Wednesday, June 3, 2026)</title>
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[Federal Register Volume 91, Number 106 (Wednesday, June 3, 2026)]
[Notices]
[Pages 33178-33179]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-11054]
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DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration
[Docket No. FDA-2026-D-1257]
Leveraging Prior Knowledge in the Development of Human Gene
Therapy Products Incorporating Genome Editing; Draft Guidance for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, Agency, or we) is
announcing the availability of a draft document entitled ``Leveraging
Prior Knowledge in the Development of Human Gene Therapy Products
Incorporating Genome Editing.'' The draft guidance document provides
sponsors engaged in the development of human gene therapy (GT) products
incorporating ex-vivo and in vivo genome editing (GE) of human somatic
cells (GE products) with FDA's recommendations on the type of prior
knowledge that may be scientifically appropriate to leverage to advance
product development.
DATES: Submit either electronic or written comments on the draft
guidance by September 1, 2026 to ensure that the Agency considers your
comment on this draft guidance before it begins work on the final
version of the guidance.
ADDRESSES: You may submit comments on any guidance at any time as
follows:
Electronic Submissions
Submit electronic comments in the following way:
<bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Follow the instructions for submitting comments. Comments submitted
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a>
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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
<bullet> 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:
<bullet> 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.
<bullet> 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-2026-D-1257 for ``Leveraging Prior Knowledge in the Development of
Human Gene Therapy Products Incorporating Genome Editing; Draft
Guidance for Industry.'' Received comments will be placed in the docket
and, except for those submitted as ``Confidential Submissions,''
publicly viewable at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets
Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-
402-7500.
<bullet> 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 <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
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
[[Page 33179]]
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: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
Docket: For access to the docket to read background documents or
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 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 draft guidance to
the Office of Communication, Outreach and Development, Center for
Biologics Evaluation and Research (CBER), Food and Drug Administration,
by emailing <a href="/cdn-cgi/l/email-protection#1a73747e6f696e68633478737576757d7379695a7c7e7b34727269347d756c"><span class="__cf_email__" data-cfemail="a1c8cfc5d4d2d5d3d88fc3c8cecdcec6c8c2d2e1c7c5c08fc9c9d28fc6ced7">[email protected]</span></a>. The draft guidance may also
be obtained by mail by calling CBER at 1-800-835-4709 or 240-402-8010.
See the SUPPLEMENTARY INFORMATION section for electronic access to the
draft guidance document.
FOR FURTHER INFORMATION CONTACT: Rhea Chakraborty, Center for Biologics
Evaluation and Research, Food and Drug Administration, 240-402-7911.
SUPPLEMENTARY INFORMATION:
I. Background
FDA is announcing the availability of a draft document entitled
``Leveraging Prior Knowledge in the Development of Human Gene Therapy
Products Incorporating Genome Editing; Draft Guidance for Industry.''
This draft guidance, when finalized, will reflect FDA's current
thinking on the type of prior knowledge that may be scientifically
appropriate to leverage to advance product development. This draft
guidance also provides recommendations on how sponsors may consider
leveraging prior knowledge to increase review efficiency and accelerate
product development across multiple programs. These recommendations
include how to leverage chemistry, manufacturing, and controls;
nonclinical; and clinical prior knowledge. The ability to leverage
prior knowledge to expedite product development may be particularly
helpful in the context of GE products intended to treat rare diseases.
While this draft guidance specifically focuses on GE products, some of
the recommendations, when finalized, may also be applicable to other
cell and GT products, such as adeno-associated viral vectors,
nanoparticle-based GT products, and ex vivo-modified cell-based GTs
that do not incorporate GE. However, additional considerations may also
apply to these related product types, based on the specific product and
manufacturing process, that are beyond those recommended in this draft
guidance.
FDA is issuing this draft guidance in accordance with a commitment
outlined in the reauthorization of the Prescription Drug User Fee Act
(PDUFA VII) under the 2022 FDA User Fee Reauthorization Act.
This draft guidance is being issued consistent with FDA's good
guidance practices regulation (21 CFR 10.115). The draft guidance, when
finalized, will represent the current thinking of FDA on ``Leveraging
Prior Knowledge in the Development of Human Gene Therapy Products
Incorporating 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.
As we develop final guidance on this topic, FDA will consider
comments on costs or cost savings the guidance may generate, relevant
for Executive Order 14192.
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 (PRA) (44 U.S.C. 3501-3521). The collections of information
in 21 CFR parts 210 and 211 relating to current good manufacturing
practice have been approved under OMB control number 0910-0139. The
collections of information in 21 CFR part 312 for the submissions of
investigational new drug applications, including clinical trials, have
been approved under OMB control number 0910-0014. The collections of
information in 21 CFR part 601 relating to the submissions of biologic
license applications have been approved under OMB control number 0910-
0338. The collections of information in 21 CFR part 1271 relating to
human gene therapy products have been approved under OMB control number
0910-0543.
III. Electronic Access
Persons with access to the internet may obtain the draft guidance
at <a href="https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances">https://www.fda.gov/vaccines-blood-biologics/guidance-compliance-regulatory-information-biologics/biologics-guidances</a>, <a href="https://www.fda.gov/regulatory-information/search-fda-guidance-documents">https://www.fda.gov/regulatory-information/search-fda-guidance-documents</a>, or
<a href="https://www.regulations.gov">https://www.regulations.gov</a>.
Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2026-11054 Filed 6-2-26; 8:45 am]
BILLING CODE 4164-01-P
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