U.S. Food and Drug Administration (FDA) | Institution Profile | iPS Cells & Regenerative Medicine

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INSTITUTION PROFILE / iPS CELLS & REGENERATIVE MEDICINE

FDA
The regulator that guards the entry point to the U.S. regenerative medicine market

The FDA is an agency of the U.S. federal government. For regenerative medicine, cell therapy and gene therapy, including products made from iPS cells, it reviews and decides whether a clinical trial may begin and whether a product may be sold. It does not hand out research funding. This checkpoint applies to every company that wants to sell in the United States, including those based abroad. This profile looks at one question only: how much influence the FDA actually has over iPS cells and regenerative medicine.

Sources: FDA overview documents, the CBER and OTP pages, the list of approved products, the RMAT page, FDA guidance documents and the Code of Federal Regulations (eCFR), all primary sources. Last updated October 2026.

What this article covers

The FDA in 30 seconds
What kind of body it is: the four types of influence
What it does for iPS cells and regenerative medicine
How far its influence reaches (powers and geography)
Companies and organisations it deals with
Outlook
Glossary, references and claim-to-source audit

1. The FDA in 30 seconds

FULL NAMEU.S. Food and Drug AdministrationUsually abbreviated FDA

COUNTRYUnited StatesHeadquarters at the White Oak Campus in Silver Spring, Maryland

TYPE OF BODYFederal regulatory agencyAn agency within the Department of Health and Human Services (HHS)

ESTABLISHEDRegulatory work began in 1906It took its present name in 1930

STAFF20,593 (FTE)The whole FDA, in fiscal 2025. The references give no separate figure for CBER

BUDGETAbout US$7 billionFiscal 2025. 51% federal budget, 49% industry user fees

ROLE IN iPS CELLS AND REGENERATIVE MEDICINEReviewing trials and approvalsHandled by OTP (Office of Therapeutic Products) within CBER (Center for Biologics Evaluation and Research)

REACHEvery product sold in the U.S.Includes companies based abroad. It does not fund research

The one thing to grasp first

The FDA is the body that controls the entry point for getting regenerative medicine, cell therapy and gene therapy, including iPS cell-based products, to patients in the United States. It is not a funder of research. It is the party that reviews and decides whether a clinical trial may start and whether a product may be sold. Because this checkpoint applies to every company that wants to sell in the United States, Japanese and European companies cannot avoid it Sourced. At the same time, the FDA approves products one application at a time, and it cannot decide approvals in other countries.

2. What kind of body it is: the four types of influence

Unlike the companies in this series, the institutions that appear in coverage of iPS cells and regenerative medicine do not make cells themselves. What separates them is the route by which they influence the industry, and that gives four types.

Fig. 1 Four types of institution involved in iPS cells and regenerative medicine, and where the FDA sits. The classification is our own, and the examples are institutions covered in this series Our calculation. The FDA is the representative regulator, and its reach is the U.S. market (including foreign companies).

The FDA's authority rests on statute. CBER's own introduction says that CBER regulates biological products under the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act Sourced.

3. What it does for iPS cells and regenerative medicine

The FDA's role in regenerative medicine can be broken down as follows Sourced. Its system covers cell therapy, gene therapy and tissue-engineered products generally, not iPS cells alone.

Role	What it involves	System or document behind it
Who reviews	OTP, within CBER, reviews cell and gene therapy products. OTP has six offices, 14 divisions and 33 branches, and was formed by reorganising the former OTAT	The page announcing OTP's establishment. The list of approved products is also published under OTP's name
Starting a trial	To begin a clinical trial in humans, the sponsor files an IND (investigational new drug application) and presents safety and quality information	Genome editing guidance (which refers to 21 CFR 312.23)
Expedited programme (RMAT)	Where a regenerative medicine product targets a serious condition and early clinical data suggest it can meet an unmet medical need, the sponsor can request designation when filing the IND or afterwards. The FDA notifies its decision within 60 days	The page on RMAT designation under section 3033 of the 21st Century Cures Act
Guidance	Published guidance includes expedited programmes for regenerative medicine therapies (February 2019, final) and human gene therapy products incorporating human genome editing (January 2024, final)	The page for each guidance document
Where human cell and tissue products draw the line	A product that meets all four conditions, such as minimal manipulation and use for the same function it performed in the body, falls into a category (the "section 361" category) that needs only registration and controls, not marketing approval. A product that does not meet them needs approval	21 CFR 1271.10 and the July 2020 guidance
Warnings on unapproved products	The FDA tells consumers that stem cells, adipose-derived cells, cord blood, exosomes and similar products are widely sold without approval, and it takes reports of adverse events	The FDA's consumer information (June 2021)

A materials engineer's view: iPS cell-derived products are likely to fall on the "approval required" side

Of the conditions in 1271.10, "minimal manipulation" is the first hurdle Sourced. iPS cells go through heavy processing: they are generated, cultured and differentiated into the target cells. They therefore do not meet this condition, and are likely to be subject to a Biologics License Application (BLA) (our interpretation). A company selling an iPS cell-derived product in the United States should therefore expect to assemble its cell manufacturing process and quality control documentation and go through OTP review.

The list of approved cellular and gene therapy products (updated 17 September 2026) includes CAR-T cell therapies for blood cancers and a genome-editing treatment for sickle cell disease Sourced. In the product names and manufacturers we checked against the list, we found no product identifiable as iPS cell-derived Our calculation.

4. How far its influence reaches (powers and geography)

Aspect	What the FDA has	What it does not have
Powers	Reviewing the start of trials, approving products for sale, granting designations under expedited programmes such as RMAT, applying the categories for human cell and tissue products, and warning the public about unapproved products	Allocating research funding, or setting reimbursement and prices (the FDA's overview document describes its mission as regulation and lists neither)
Geography	Products sold in the United States. About 96% of biologics sold are imported, and there are 697 registered foreign facilities handling biologics. Japanese and European companies are covered too	Decisions on approvals in other countries. Authority over other countries' research ethics or systems
Scale	20,593 staff (FTE, whole FDA) and a budget of about US$7 billion. OTP has six offices, 14 divisions and 33 branches	Staff numbers or budget for CBER or OTP alone (not given in the references)
International reference point	It publishes its guidance and the documents behind its approvals	Power to make other countries' regulators follow it

A note on reading the numbers

The staff and budget figures are for the FDA as a whole, which also covers food, tobacco, animal drugs and more Sourced. OTP, which handles iPS cells and regenerative medicine, is only one part of it. Also, the absence of iPS cell-derived products from the approved list does not mean the FDA refuses to approve iPS cell-derived therapies. The FDA's system covers cell therapy in general, and the references do not say that iPS cell-derived products are treated differently.

5. Companies and organisations it deals with

The FDA is not a company's partner. It is the party that reviews companies' applications. We show only the relationships that can be confirmed from FDA publications.

Fig. 2 The FDA's relationships with companies and clinics. Sources: the FDA's list of approved cellular and gene therapy products, the Tregzi product page, the FDA news release of 8 December 2023 and the FDA's consumer information. The selection of products is ours Our calculation.

Examples of approvals confirmed in FDA publications

Product (as the FDA writes it)	Manufacturer (as the FDA writes it)	What the FDA documents show
CASGEVY (exagamglogene autotemcel)	Vertex Pharmaceuticals Incorporated	Approved 8 December 2023. The first FDA-approved treatment to use genome editing technology (FDA news release)
TREGZI (allogeneic regulatory T-cell immunotherapy)	Orca Biosystems Inc	Approval letter dated 1 July 2026. Used with hematopoietic stem cell transplantation from a matched donor in adults with blood cancers
ABECMA, BREYANZI	Celgene, Juno (both part of Bristol-Myers Squibb)	Listed among the approved products
KYMRIAH, ITVISMA	Novartis (the FDA names the relevant subsidiaries)	Listed among the approved products

Note: none of these is an iPS cell-derived product. They are examples of the kinds of cell and gene therapy the FDA has actually approved. Manufacturer names follow the FDA's list and the individual product pages Sourced.

6. Outlook

What the evidence suggests	Primary information it rests on	Category
If an iPS cell-derived product is ever approved in the United States for the first time, the OTP office responsible for cell therapy and human tissue manufacturing controls will be at the centre of the manufacturing and quality review	OTP is organised into three offices for manufacturing controls (chemistry, manufacturing and controls, or CMC), covering gene therapy, cell therapy and human tissue, and plasma proteins, plus offices for clinical work, pharmacology and toxicology, and review management	Inference
For regenerative medicine products, using expedited programmes such as RMAT will become a standard part of development planning	RMAT is set out in the 21st Century Cures Act and can be requested at the IND stage, and the FDA notifies its decision within 60 days. The final 2019 guidance explains the programme	Inference
When the first iPS cell-derived product will be approved	The approved products list (updated 17 September 2026) contains no product identifiable as iPS cell-derived	Not yet confirmed

7. Glossary

CBER: The Center for Biologics Evaluation and Research. The part of the FDA that handles vaccines, blood products, cell and gene therapies and similar products.

OTP: CBER's Office of Therapeutic Products. It reviews cell therapies and gene therapies.

IND: An investigational new drug application. The procedure in which a sponsor gives the FDA safety and quality information before starting a clinical trial in humans.

BLA: A Biologics License Application, the application for marketing approval of a biological product. Cell therapies and gene therapies are approved in this form.

RMAT: Regenerative Medicine Advanced Therapy designation. A programme that speeds up development of regenerative medicine products for serious conditions.

HCT/P: Human cells, tissues, and cellular and tissue-based products. A product that meets conditions such as minimal manipulation falls into a category that does not need marketing approval.

8. References

FDA FDA at a Glance (January 2026 edition): fiscal 2025 budget of about US$7 billion and its funding split, 20,593 FTE, about 96% of biologics sold being imported, the number of registered biologics facilities, and locations. https://www.fda.gov/media/154548/download
FDA FDA History: the start of its present regulatory work under the 1906 Pure Food and Drugs Act, and the adoption of its present name in 1930. https://www.fda.gov/about-fda/fda-history
FDA Center for Biologics Evaluation and Research (CBER) introduction page: mission and governing statutes. https://www.fda.gov/about-fda/fda-organization/center-biologics-evaluation-and-research-cber
FDA Establishment of the Office of Therapeutic Products: OTP's launch, the reorganisation of the former OTAT, and the structure of six offices, 14 divisions and 33 branches. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/establishment-office-therapeutic-products
FDA Approved Cellular and Gene Therapy Products: the list of approved products under OTP's remit, updated 17 September 2026. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
FDA Regenerative Medicine Advanced Therapy Designation: RMAT requirements, requests at the IND stage and notification within 60 days. Updated 4 November 2025. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation
FDA Guidance "Expedited Programs for Regenerative Medicine Therapies for Serious Conditions" (February 2019, final). https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-regenerative-medicine-therapies-serious-conditions
FDA Guidance "Human Gene Therapy Products Incorporating Human Genome Editing" (January 2024, final). https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-products-incorporating-human-genome-editing
FDA Guidance "Regulatory Considerations for Human Cells, Tissues, and Cellular and Tissue-Based Products: Minimal Manipulation and Homologous Use" (July 2020, final). https://www.fda.gov/regulatory-information/search-fda-guidance-documents/regulatory-considerations-human-cells-tissues-and-cellular-and-tissue-based-products-minimal
U.S. Code of Federal Regulations (eCFR) 21 CFR 1271.10 Are my HCT/P's regulated solely under section 361 of the PHS Act …: the four conditions for the section 361 category. https://www.ecfr.gov/current/title-21/chapter-I/subchapter-L/part-1271/subpart-B/section-1271.10
FDA Important Patient and Consumer Information About Regenerative Medicine Therapies (3 June 2021): a warning about unapproved regenerative medicine products. https://www.fda.gov/vaccines-blood-biologics/consumers-biologics/important-patient-and-consumer-information-about-regenerative-medicine-therapies
FDA TREGZI product page: BLA 125868, manufacturer Orca Biosystems Inc, approval letter dated 1 July 2026. https://www.fda.gov/vaccines-blood-biologics/tregzi
FDA News release "FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease" (8 December 2023): Casgevy is the first FDA-approved treatment to use genome editing technology. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease

9. Claim-to-source audit

Claim in the article	Category	Source
Full name, headquarters location, status as an agency within HHS, fiscal 2025 budget of about US$7 billion (51% federal, 49% user fees), 20,593 FTE	Sourced	Reference 1 https://www.fda.gov/media/154548/download
Regulatory work beginning in 1906 and the present name adopted in 1930	Sourced	Reference 2 https://www.fda.gov/about-fda/fda-history
CBER regulating biological products under the Public Health Service Act and the Federal Food, Drug, and Cosmetic Act	Sourced	Reference 3 https://www.fda.gov/about-fda/fda-organization/center-biologics-evaluation-and-research-cber
OTP's launch, the reorganisation of the former OTAT, and the structure of six offices, 14 divisions and 33 branches	Sourced	Reference 4 https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/establishment-office-therapeutic-products
The list of approved cellular and gene therapy products (including products from Vertex, Orca Biosystems, the BMS group and Novartis), updated 17 September 2026	Sourced	Reference 5 https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
RMAT requirements, requests at the IND stage and notification within 60 days	Sourced	Reference 6 https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation
The final guidance on expedited programmes for regenerative medicine (February 2019)	Sourced	Reference 7 https://www.fda.gov/regulatory-information/search-fda-guidance-documents/expedited-programs-regenerative-medicine-therapies-serious-conditions
The guidance on gene therapy incorporating human genome editing (January 2024) and the information to be presented in an IND	Sourced	Reference 8 https://www.fda.gov/regulatory-information/search-fda-guidance-documents/human-gene-therapy-products-incorporating-human-genome-editing
The guidance on minimal manipulation and homologous use (July 2020)	Sourced	Reference 9 https://www.fda.gov/regulatory-information/search-fda-guidance-documents/regulatory-considerations-human-cells-tissues-and-cellular-and-tissue-based-products-minimal
The four conditions for the section 361 category (minimal manipulation, homologous use only, no combination with other substances, and no systemic effect)	Sourced	Reference 10 https://www.ecfr.gov/current/title-21/chapter-I/subchapter-L/part-1271/subpart-B/section-1271.10
The warning about the sale of unapproved stem cell and similar products, and the taking of adverse event reports	Sourced	Reference 11 https://www.fda.gov/vaccines-blood-biologics/consumers-biologics/important-patient-and-consumer-information-about-regenerative-medicine-therapies
TREGZI's manufacturer and the approval letter dated 1 July 2026	Sourced	Reference 12 https://www.fda.gov/vaccines-blood-biologics/tregzi
Casgevy's approval date and its status as the first approved treatment to use genome editing technology	Sourced	Reference 13 https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
About 96% of biologics sold being imported, and 697 registered foreign facilities	Sourced	Reference 1 https://www.fda.gov/media/154548/download
The four types of institution, and the selection of approved products	Our calculation	Our own classification and selection (products chosen from references 5, 12 and 13)
No product identifiable as iPS cell-derived on the approved list	Our calculation	The result of this article's check of the product names and manufacturers in reference 5
iPS cell-derived products involve heavy processing and are likely to be subject to a BLA	Inference	This article's reading of the "minimal manipulation" condition in reference 10
The first two points in the outlook (section 6)	Inference	This article's reading of references 4, 6 and 7
When the first iPS cell-derived product will be approved	Not yet confirmed	Not stated in reference 5; this article does not estimate it
Staff numbers or budget for CBER or OTP alone	Not yet confirmed	Not stated in references 1, 3 and 4; this article does not estimate them

Last updated 1 October 2026 / Troy Technical
All figures come from the FDA's official website and guidance and from federal regulations. Items marked "Inference" are this article's reading of the primary information. This article is not medical advice.