Regenerative medicine is the field of medicine that develops ways to repair or replace damaged, diseased or failing cells, tissues and organs, through tissue engineering, cell transplantation and artificial or bioartificial organs and tissues.[1] Its tools fall into four broad groups: cell therapy, which uses living cells (including stem cells such as somatic (adult) stem cells); tissue engineering with biomaterials; gene therapy; and medicines that stimulate the body’s own repair. A small number of products in each group are approved by regulators for specific conditions; most uses are still experimental. How stem cells themselves work, and how cells differentiate into specialised types, is explained on our page about stem cell therapy.
What Regenerative Medicine Covers
Regulators use their own definitions. In US law, a regenerative medicine therapy is a cell therapy, a therapeutic tissue engineering product, a human cell and tissue product, or a combination product using them; the US Food and Drug Administration (FDA) also counts certain gene therapies and xenogeneic (animal) cell products.[2] The European Union groups the same products as advanced therapy medicinal products, of three main types: gene therapy medicines, somatic cell therapy medicines and tissue-engineered medicines, some of which also contain a medical device, such as cells on a biodegradable scaffold.[3] Gene therapy means modifying cells, using genetic sequences or other means, to treat or reverse disease.[4]
Approved Examples by Approach
The table gives examples of approved products, with the regulator and the approved use. Each approval covers only the stated condition.
| Approach | Example | Regulator and approved use |
|---|---|---|
| Cell therapy | Cord blood products of blood-forming stem cells (nine on the US list) | FDA; disorders that affect blood production[5][6] |
| Cell therapy | Cultured cartilage cells from the patient on a collagen membrane (MACI) | FDA; full-thickness cartilage defects of the knee in adults[7] |
| Cell therapy | Dopamine-producing nerve precursor cells made from donor induced pluripotent stem cells (AMCHEPRY) | Japan, conditional and time-limited approval, March 2026; motor symptoms of Parkinson’s disease not controlled by medicines[8] |
| Tissue engineering | Donor skin cells in a collagen layer (STRATAGRAFT) | FDA; adult thermal burns that still contain intact dermis[9] |
| Tissue engineering | Acellular tissue-engineered blood vessel (SYMVESS) | FDA; urgent repair of arterial injury in a limb when the patient’s own vein cannot be used[10] |
| Tissue engineering | Heart muscle cell sheets made from donor induced pluripotent stem cells (RiHEART) | Japan, conditional and time-limited approval, March 2026; severe heart failure from ischemic cardiomyopathy[8] |
| Gene therapy | Voretigene neparvovec (LUXTURNA) | FDA; retinal dystrophy caused by mutations in both copies of the RPE65 gene[11] |
| Gene therapy | Patient’s own blood stem cells edited or modified (CASGEVY, LYFGENIA) | FDA; sickle cell disease (CASGEVY also transfusion-dependent beta thalassemia)[12] |
| Stimulating repair | Becaplermin gel, a recombinant platelet-derived growth factor (REGRANEX) | FDA; diabetic neuropathic ulcers of the leg or foot that reach the tissue under the skin or deeper and have an adequate blood supply, added to standard ulcer care; cancers distant from the treated site have been reported in users[13] |
Human embryonic stem cell lines, able to form tissues of all three germ layers, were first derived in 1998,[14] and induced pluripotent stem cells made from donor cells (for AMCHEPRY, from blood cells) are the starting material for the two Japanese products in the table.[8]
Where Mesenchymal Stromal Cell Therapy Sits
Approvals of mesenchymal stromal cells (MSCs) are few and narrow. As of November 2026, the only MSC product on the FDA list is remestemcel-L (RYONCIL), for children aged 2 months and older with acute graft-versus-host disease that has not responded to steroids.[5][15] Japan has approved more MSC products, each for one narrow use: donor bone marrow MSCs for the same complication (TEMCELL HS, 2015); donor fat MSCs for complex perianal fistulas in Crohn’s disease (Alofisel, 2021); donor fat MSC sheets for skin erosions in epidermolysis bullosa (Allostem Sheet, 2026); a patient’s own knee synovium MSCs for meniscal injury repaired by suturing (Savyscus, 2026); and, with conditional, time-limited approval, a patient’s own bone marrow MSCs for traumatic spinal cord injury (STEMIRAC, 2018) and modified donor bone marrow MSCs for chronic weakness after traumatic brain injury (Akuugo, 2024).[8] In the European Union, Alofisel was withdrawn on 13 December 2024 at the company’s request,[16] after a confirmatory trial of 568 patients found no benefit over placebo,[17] and the company said it was discussing the outcome with authorities in the other countries where it was approved.[18] Outside approved products and their stated uses, MSC treatment is investigational.
How Regulators Classify Cell Products
- United States. A human cell or tissue product escapes premarket approval only if it meets all of these conditions: it is minimally manipulated; it is intended for homologous use, meaning it repairs, reconstructs, replaces or supplements the recipient’s cells or tissues and performs the same basic function there as in the donor;[19] it is not combined with another article (apart from water, crystalloids or a sterilising, preserving or storage agent); and either it has no systemic effect and does not depend on living cells’ metabolic activity, or it is for the patient’s own use, use in a first- or second-degree blood relative, or reproductive use. Such products must still be registered and listed with the FDA.[20] Other regenerative medicine products need FDA approval before they can be marketed, and before approval they can be given only within an FDA-regulated clinical trial.[6]
- European Union. Gene therapy, somatic cell therapy and tissue-engineered medicines are regulated as advanced therapy medicinal products.[3]
- Japan. Regenerative medical products can receive a conditional, time-limited approval, as the induced pluripotent stem cell products and STEMIRAC above did.[8]
How to Judge a Regenerative Medicine Claim
The International Society for Stem Cell Research advises patients that very few stem cell treatments have been proven safe and effective. The best-established is blood-forming stem cell transplantation for certain blood and immune disorders and to rebuild the blood system after some cancer treatments; some bone, skin and corneal injuries can be treated with tissue grafts. All other uses should be considered highly experimental. Because tissue-specific stem cells make only the cell types of their own tissue, the same treatment is unlikely to work for many unrelated diseases.[21]
The FDA warns that products marketed as regenerative medicine without approval, including stem cells, fat-derived stromal vascular fraction, cord blood, amniotic fluid, Wharton’s jelly and exosomes, have caused blindness, tumors, serious infections and other harms. It states that no regenerative medicine therapy is approved for conditions such as ALS, Alzheimer’s or Parkinson’s disease, stroke, heart disease, emphysema or COPD, osteoarthritis, tendonitis, or back, knee or shoulder pain, and that being charged for these products outside a clinical trial usually means the product is being offered illegally. A listing on clinicaltrials.gov, or a company’s registration with the FDA, does not mean a product is legally marketed.[6] Useful questions before any treatment are whether the product is approved for that condition by a named regulator, or whether it is given in a registered trial under regulatory oversight, and what the published controlled trials found.[21][6]
Published Clinical Citations
[1] ^ National Library of Medicine. Regenerative Medicine (MeSH descriptor D044968). Medical Subject Headings. https://meshb.nlm.nih.gov/record/ui?ui=D044968
[2] ^ US Food and Drug Administration. Regenerative Medicine Advanced Therapy Designation. Content current as of 4 November 2025. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation
[3] ^ European Medicines Agency. Advanced therapy medicinal products: overview. https://www.ema.europa.eu/en/human-regulatory-overview/advanced-therapy-medicinal-products-overview
[4] ^ National Library of Medicine. Genetic Therapy (MeSH descriptor D015316). Medical Subject Headings. https://meshb.nlm.nih.gov/record/ui?ui=D015316
[5] ^ US Food and Drug Administration. Approved Cellular and Gene Therapy Products. Content current as of 30 October 2026. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products
[6] ^ US Food and Drug Administration. Important Patient and Consumer Information About Regenerative Medicine Therapies. Content current as of 8 April 2024. https://www.fda.gov/vaccines-blood-biologics/consumers-biologics/important-patient-and-consumer-information-about-regenerative-medicine-therapies
[7] ^ US Food and Drug Administration. MACI (autologous cultured chondrocytes on porcine collagen membrane). BL 125603. Content current as of 26 August 2024. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/maci-autologous-cultured-chondrocytes-porcine-collagen-membrane
[8] ^ Pharmaceuticals and Medical Devices Agency (Japan). List of Approved Regenerative Medical Products, April 2015 to July 2026. https://www.pmda.go.jp/files/000283257.pdf
[9] ^ US Food and Drug Administration. STRATAGRAFT (allogeneic cultured keratinocytes and dermal fibroblasts in murine collagen-dsat). STN 125730. Content current as of 31 January 2024. https://www.fda.gov/vaccines-blood-biologics/stratagraft
[10] ^ US Food and Drug Administration. SYMVESS (acellular tissue engineered vessel-tyod). STN 125812. Content current as of 25 February 2025. https://www.fda.gov/vaccines-blood-biologics/symvess
[11] ^ US Food and Drug Administration. LUXTURNA (voretigene neparvovec-rzyl). STN 125610. Content current as of 9 June 2022. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/luxturna
[12] ^ US Food and Drug Administration. FDA approves first gene therapies to treat patients with sickle cell disease. Press announcement, 8 December 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapies-treat-patients-sickle-cell-disease
[13] ^ LRM Therapeutics. REGRANEX (becaplermin) gel 0.01%. US prescribing information. DailyMed set ID 377b3021-13d7-96d5-e063-6394a90a8ca3, published 11 May 2026. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=377b3021-13d7-96d5-e063-6394a90a8ca3
[14] ^ Thomson JA, Itskovitz-Eldor J, Shapiro SS, et al. Embryonic stem cell lines derived from human blastocysts. Science. 1998;282(5391):1145-1147. https://doi.org/10.1126/science.282.5391.1145
[15] ^ US Food and Drug Administration. RYONCIL (remestemcel-L-rknd). STN 125706. Content current as of 17 January 2025. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/ryoncil
[16] ^ European Medicines Agency. Alofisel (darvadstrocel): withdrawal of the marketing authorisation in the European Union. https://www.ema.europa.eu/en/medicines/human/EPAR/alofisel
[17] ^ Colombel JF, Garcia Olmo D, Chen ST, et al. Darvadstrocel in patients with Crohn’s disease with complex perianal fistulas: the ADMIRE CD II phase 3 randomized trial. Gastroenterology. 2026;170(7):1562-1570. https://doi.org/10.1053/j.gastro.2025.12.033
[18] ^ Takeda Pharmaceutical Company. Takeda provides an update on Alofisel (darvadstrocel). Statement, 13 December 2024. https://www.takeda.com/newsroom/statements/2024/takeda-alofisel-update-2024/
[19] ^ US Code of Federal Regulations. Title 21, section 1271.3(c): definition of homologous use. https://www.ecfr.gov/current/title-21/chapter-I/subchapter-L/part-1271/subpart-A/section-1271.3
[20] ^ US Code of Federal Regulations. Title 21, section 1271.10: Are my HCT/Ps regulated solely under section 361 of the PHS Act and the regulations in this part, and if so what must I do? https://www.ecfr.gov/current/title-21/chapter-I/subchapter-L/part-1271/subpart-A/section-1271.10
[21] ^ International Society for Stem Cell Research. Nine Things to Know About Stem Cell Treatments. A Closer Look at Stem Cells. https://www.closerlookatstemcells.org/stem-cells-medicine/nine-things-to-know-about-stem-cell-treatments/