Updated 2025 List of FDA-Approved Cell and Gene Therapies

What are the current FDA-approved cell and gene therapies? How many of those involve true stem cells?

Many of us have wished for a more specific list of FDA-approved stem cell therapies. Patients and fellow scientists often asked me about such lists. They even wish for annotated lists.

For that reason, I’ve made and kept updated a comprehensive list of FDA Approved Cell and Gene Therapies, which is the focus of today’s post. These include FDA-approved stem cell therapies. I’ve updated the list now in 2025 to include Casgevy and Lyfgenia, approved cell and gene therapies for sickle cell.

FDA and cell therapies | List of diseases treated by stem cells | Key Context | Cell & Gene Therapies | List of FDA approved therapies 2025 | FDA approved stem cell clinics? | Reasons for optimism | References

FDA cleared cell therapies

It’s exciting that the FDA has approved 35 gene and cell therapies.

Note that you can watch me discuss this post in a new YouTube Video below.

List of diseases treated by stem cells that are FDA-approved

What about today in 2025? Before we get to the approved products, I made a short list of the diseases treated by FDA-approved stem cells.

Note that only very specific products matched with specific forms of these diseases have the FDA OK. For instance just because damaged cartilage is on the list doesn’t mean anything goes. There’s no approval to treat it with fat stem cells, bone marrow, umbilical cord cells or exosomes. The approved sickle-cell treatments are combined cell and gene therapies.

• AADC deficiency
• Melanoma
• Mantle Cell Lymphoma
• Large B-Cell Lymphoma
• B-Cell ALL
• Prostate Cancer
• Some other kinds of cancer (immune system reconstitution)
• Facial wrinkles (specific type)
• Receding gums
• Damaged cartilage
• Leber congenital amaurosis (a retinal disorder)
• Sickle Cell Disease

The list of diseases has been growing. What about the products?

Context for FDA-approved stem cell therapies

We need to turn to the FDA itself for the clearest answer on where things stand now.

What does the agency have to say about what they have approved?

In mid-2020, they issued an advisory, “Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes.” In it they wrote:

“Stem cell products are regulated by FDA, and, generally, all stem cell products require FDA approval. Currently, the only stem cell products that are FDA-approved for use in the United States consist of blood-forming stem cells (also known as hematopoietic progenitor cells) that are derived from umbilical cord blood. These products are approved for use in patients with disorders that affect the production of blood (i.e., the “hematopoietic” system) but they are not approved for other uses.”

They now have approved a few more stem cell drugs including Mesoblast’s MSC product for some GvHD cases.

No unproven clinics have FDA approval.

Still, too often stem cell clinics claim that what they offer is “FDA approved”. In actuality at best what some of them sell is not FDA approved, but rather technically compliant with the rules for 361 products. What this means in English is that the products are not regulated as drugs.

FDA-approved cell and gene therapies

An interesting side note is that the agency for several years now has been combining together cell and gene therapies into one category. This is also reflected in their Regenerative Medicine Advanced Therapy (RMAT) designation program, which contains both types. RMATs still have to go through the trial approval process but get special consideration. You can see a help infographic below that my intern Mina made of the clinical trial approval process.

The formal FDA list can be found here: “Approved Cellular and Gene Therapy Products.”

List of FDA approved cell and gene therapies

Here’s the list. Note that many approved products are almost the same in the type of cells (umbilical cord) and applications.

• ABECMA (idecabtagene vicleucel) for refractory myeloma, Celgene Corporation.
• ADSTILADRIN for unresponsive bladder cancer, Ferring Pharmaceuticals
• ALLOCORD (Cord Blood), SSM Cardinal Glennon Children’s. “For use in unrelated donor hematopoietic progenitor cell transplantation.”
• BREYANZI CAR-T-cell therapy for adults with relapsed or refractory large B-cell lymphoma, Juno Therapeutics.
• CARVYKTI for refractory myeloma, Janssen
• CASGEVY, Sickle cell disease, Vertex.
• CLEVECORD, Cleveland Cord Blood Center. Similar to ALLOCORD.
• Ducord, Cord Blood, Duke Med School, Similar to ALLOCORD.
• ELEVIDYS for DMD, Sarepta Therapeutics
• GINTUIT for receding gums, Organogenesis.
• HEMACORD (cord blood), New York Blood Center. This and the four that follow are similar to ALLOCORD.
• Cord Blood, Clinimmune Labs.
• Cord Blood – MD Anderson.
• Cord Blood – LifeSouth.
• Cord Blood – Bloodworks
• IMLYGIC An oncolytic gene therapy product for treatment of melanoma, BioVex.
• Kebilidi, AADC deficiency, PTC Therapeutics.
• KYMRIAH CAR-T cell therapy for B-cell acute lymphoblastic leukemia  (B-Cell ALL), Novartis.
• LANTIDRA for Type 1 diabetes, CellTrans Inc.
• LAVIV Treatment of deep wrinkles on the sides of our noses, Fibrocell Technologies.
• LUXTURNA for Leber congenital amaurosis, Spark Therapeutics. 
• LYFGENIA, bluebird bio.
• MACI for damaged cartilage, Vericel.
• OMISIRGE for blood cancers, Gamida Cell.
• PROVENGE (sipuleucel-T) for prostate cancer, Dendreon.
• Regenecyte, immune system reconstitution such as after chemo for certain cancers, StemCyte, Inc.
• RETHYMIC for athymia, Enzyvant Therapeutics.
• ROCTAVIAN for severe hemophilia A, BioMarin Pharmaceutical.
• Ryoncil, some cases of pediatric GvHD, Mesoblast.
• SKYSONA for CALD, bluebird bio.
• STRATAGRAFT for burns, Stratatech.
• TECARTUS, CAR-T therapy for treatment of adults with relapsed/refractory mantle cell lymphoma, Kite Pharma.
• YESCARTA CAR-T for large B-cell lymphoma that hasn’t responded to other therapy, Kite Pharma.
• ZOLGENSMA (Nusinersen), AveXis.
• ZYNTEGLO for ß-thalassemia, bluebird bio.

List of FDA-approved stem cell clinics?

What about a list of FDA-approved stem cell clinics?

Such a list doesn’t exist.

Why?

The reason is because no clinics have FDA approval. While some clinics sell stem cells that don’t need formal FDA drug approval, most need that approval and yet don’t have it. Why don’t they have the approval?  In part, it’s because the clinics don’t even try to work with the FDA. At a more fundamental level, the clinics mostly also do not do real research. They don’t collect robust data on safety and efficacy.

No exosome therapy has gotten the FDA’s okay either. Despite that, it is widely sold at clinics across the U.S.

Adipose cell therapy or SVF is also not legal to market at this point. Recent court decisions have left things unclear as to what SVF clinics will do next.

Reasons for optimism looking to the future

Setting aside the unproven clinics for the moment, there is good reason to be upbeat about our space.

I’m going to update this The Niche post as the agency adds to its list.

Yes, I think the list will continue to grow over the next few years, which is exciting. There’s at least a 50% chance that one or more additional therapies in this space will garner approval this year. I believe that some of these could be driven by politics but I’m just generally optimistic about our arena.

References and notes on FDA-approved cell and gene therapies

1. Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes, July 22, 2022, FDA advisory
2. FDA, list of “Approved Cellular and Gene Therapy Products.” Current as of early 2025.
3. I didn’t list Spinraza for  SMA or spinal muscular atrophy as it isn’t a cell or gene therapy. It is an antisense oligonucleotide therapy.
4. Note that the FDA has not approved SOT or supportive oligonucleotide therapy. Even so, I haven’t seen any FDA action on those clinics.
5. The Alliance for Regenerative Medicine has what we might call an international list of cleared cell and gene therapies here.I’m not sure it is as up to date as my list here though.