Key Takeaways
Embryonic, adult, induced pluripotent, and perinatal stem cells are being developed as therapeutics for the treatment of countless diseases, disorders, and injuries, including cardiovascular, neurological, endocrine, metabolic, hepatic, renal, and cancer-related conditions.
In addition to their native regenerative capabilities, stem cells can also be modified to serve as delivery vehicles for other drugs
Stem cells have also been used to generate organoids that can be used in drug discovery or implanted to potentially support regeneration of tissues and organs
The global stem cell therapy market is estimated to be expanding at a compound annually growth rate between 12% and 17% over the next five years.
Going forward, wider application of gene editing technologies, further leveraging of biomaterials and bioengineering, and increased use of AI and automation will continue to be key elements of stem cell therapy advances, including in the areas of immune modulation and precision medicine
As promising readouts are realized for many early candidates, developers will need to shift their focus to achieving practical, cost-effective, larger-scale GMP manufacture of their promising products and designing pivotal trials that provide the evidence needed to satisfy regulators regarding both safety and efficacy.
What Makes Stem Cells Attractive Therapeutics?
The stem cell therapy field has been marked by two major discoveries: embryonic stem cells (ESCs) in 1981 and induced pluripotent stem cells (iPSCs) in 2007. Those two developments have fueled the exploration of embryonic, adult (e.g., mesenchymal, hematopoietic, neural), induced pluripotent, and perinatal stem cells as therapeutics for the treatment of countless diseases, disorders, and injuries. Some examples include cardiovascular, neurological, endocrine, metabolic, hepatic, renal, and cancer-related conditions. Hematopoietic stem cells (HSCs) have been used for years to treat immune disorders and blood cancers. Many clinical trials are underway investigating many other types of stem cell therapies in Parkinson’s disease, diabetes, myocardial infarction, and spinal cord injuries, among other indications.
Found in embryonic and fetal tissues, blood, skin, skeletal muscle, fat, and bone marrow, stem cells are undifferentiated cells with the ability to differentiate into specific types of cells and renew themselves. They secrete a wide range of molecules with immunomodulatory, antiapoptotic, and antimicrobial properties that stimulate tissue regeneration and repair. The extracellular vesicles (e.g., extracellular vesicles (EVs), exosomes) released by stem cells contain numerous lipids, proteins, nucleic acids, including RNA and micro-RNA, and signaling molecules, among others, that participate in intracellular communication and promote the growth and specialization of other stem/progenitor cells.
In addition to their native regenerative capabilities, stem cells can also be modified to serve as delivery vehicles for other drugs.4 Stem cells have also been used to generate organoids that can be used in drug discovery or implanted to potentially support regeneration of tissues and organs (see sidebar).5
Many Types of Stem Cells
Stems cells can be classified in a number of ways.1,3,5 One approach considers their potential for differentiation. Unipotent, multipotent (or oligopotent), pluripotent, and totipotent stem cells can differentiate into single, several, many, and very many cell types. Zygotes are the only totipotent human stem cells. Categorization by source is also common: ESCs, tissue-specific stem cells (TSCs), mesenchymal stem cells (MSCs), and iPSCs.
ESCs are pluripotent and can differentiate into most types of human cells and have a high renewal capacity.1,5 They are useful not only for therapeutic development but in disease modeling for investigation of disease pathways and drug testing. Multipotent adult stem cells (ASCs) can differentiate into an array of cell types related to the originating tissue.
While less potent than ESCs, multipotent ASCs, including HSCs and MSCs, have been shown to demonstrate therapeutic effects when extracted and formulated as both autologous (patient-derived) and allogeneic (donor-derived, off-the-shelf) treatments. Their greatest potential lies in tissue-specific regeneration and repair and slowing the progression of diseases that involve cellular degeneration.
MSCs, for instance, are multipotent stromal stem cells that can be differentiated into structural cell types, including tendon, bone (osteocytes), cartilage (chondrocytes), ligament, muscle (myocytes), and fat (adipocytes) cells.3,5,6 They also have the capacity to self-renew and are recognized for their anti-inflammatory properties, and they have been investigated for the treatment of diseases caused by overactive immune systems.
Perinatal stem cells, mostly collected from umbilical cord blood, have properties that are intermediate between ESCs and ASCs and offer a less controversial option to ESCs.1 In particular, perinatal stem cells typically do not elicit unwanted immune responses, making them attractive for development of allogenic cell therapies.
Induced pluripotent stem cells offer another alternative to ESCs because they are adult cells reprogrammed into stem cells.1,6 They can be differentiated into many cells types, such as neurons, cardiomyocytes, insulin-producing pancreatic cells, and others. In addition to applications in both personalized and allogeneic cell therapies, iPSCs have already been shown to be highly useful in drug discovery and development.
The production of iPSCs is achieved by treating adult somatic cells with a mix of transcription factors. The original set are known as the Yamanaka factors, but researchers has identified other cocktails that allow for generation of a variety of functional cells with specific properties.6
Challenges to Stem Cell Therapy
The promise of stem cell therapy can only be realized if several challenges to the development and at-scale production of these novel treatments can be achieved. The fact that the various cell types both share some common issues and individually face unique issues adds to the challenge.
One common problem is the fact that the number of stem cells in a treatment often corresponds with the potential to achieve the desired regenerative/repair results. Consequently, large numbers of cells are needed, which requires ex vivo expansion before administration.1,6 Expansion while retaining the right phenotype and functionality can be difficult for some stem cell types.5 Some stem cell therapies also result can cause immune responses and inflammation, requiring understanding of potential innate and adaptive immune system responses.3
ESCs pose significant ethical concerns, as access to these stem cells requires the destruction of human embryos.1 Surplus, donated embryos generated during in vitro fertilization are typically used, but it is imperative that donors understand how the outcome of those donated embryos.
While iPSCs are attractive alternatives to ESCs, their use has challenges as well.1,5,6 From an ethical perspective, they can be misused for human cloning and germline editing. From a production perspective, achieving consistent and efficient differentiation and long-term genetic stability can be difficult. Reprogramming is also inefficient: in some cases, less than 1% of cells become the desired iPSCs. In therapeutic applications, iPSCs can be difficult to integrate into host tissue and maintain functionality, and undifferentiated or incorrectly differentiated cells present a risk of tumorigenesis.
For allogeneic stem cell therapies, immunological rejection and host-versus-graft disease can be problematic.3 Gene editing is often pursed to eliminate/impeded genetic elements that contribute to this issue.
Growing Importance of Gene Editing
One of the biggest trends in the stem cell therapy field is the use of gene editing tools to modify the properties of the cells to decrease their immunogenicity, improve their functionality, and/or resolve mutations (e.g., remove or replace faulty or add missing genes).7,8 In addition, when used to generate iPSCs, CRISPR (clustered regularly interspaced short palindromic repeats) techniques can simplify and accelerate the generation of iPSCs.5
Few Approvals but Real Therapeutic Potential
Currently, the only common, approved stem cell therapies involve hematopoietic (or blood) stem cell transplantation. Three of these therapies involve gene editing of patient HSCs: Casgevy (exagamglogene autotemcel, Vertex Pharmaceuticals) and Lyfgenia (lovotibeglogene autotemcel, bluebird bio) for treatment of sickle cell disease, both approved in December 2023,9 and Waskyra (etuvetidigene autotemcel, Fondazione Telethon ETS) for the treatment of the rare disease Wiskott-Aldrich syndrome (WAS), approved in September 2025.10
Just one non-HSC cell therapy has received approval from the US Food and Drug Administration.6 Ryoncil® (remestemcel-L-rknd, Mesoblast Ltd.), an allogeneic bone marrow-derived MSC therapy, received approval in December 2024 for treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients.11
Preclinical studies using ex vivo–generated insulin-secreting cells, neural cells, heart cells, and other tissue-specific cells have achieved measurable improvements. Clinical trials involving a wide variety of stem cell therapies based on ESCs, MSCs, iPSCs, and others continue to realize promising results.1,3-7,12
iPSCs have been differentiated into different types of retinal cells and transplanted into patient eyes to treat various diseases including macular degeneration.
Pancreatic progenitor cells generated from iPSCs — when administered to patients with diabetes — resulted in restored insulin production and improved glycemic control.
Umbilical cord MSCs have been shown to improve muscle strength, spasticity, and fine motor function in stroke patients.
Umbilical cord stem cell therapies have also been shown to be effective in treating patients with severe COVID-19 infections.
Neural stem cells have been investigated for the treatment of many neurological diseases, including Alzheimer’s (AD) and Parkinson’s (PD). For instance, in March 2026, Aspen Neuroscience reported initial positive results for patients with AD and PD treated with its autologous stem cell therapy Sasineprocel, including measurable improvements in function, outcomes, and quality of life.13 Separately, Regeneration Biomedical was granted Fast Track designation for its autologous adipose-derived MSCs for treatment of AD, which has completed a phase I trial and is advancing to phase II.14
A stem cell therapy being investigated for the treatment of heart failure resulted in less scar tissue, increased angiogenesis, and improved heart function; cardiomyocyte therapies are also being studied in other cardiovascular diseases.
Patients with osteoarthritis experienced decreased pain, improved joint function, and some cartilage regeneration following treatment with a bone marrow–derived MSC therapy.
Numerous chimeric antigen receptor T (CAR-T) cell and natural-killer (NK) cell–based immunotherapies derived from stem cells are under development.
The list of investigative stem cell therapies is too long to include them all. Treatments are also in development for dermatological conditions, lung, kidney, liver, and dental diseases, gastrointestinal, endocrine, and autoimmune disorders, chronic wounds and other traumatic injuries, muscle repair, hair regeneration, and many types of cancer.
According to one study, as of December 2024, there were 115 global clinical trials involving 83 different pluripotent stem cell therapies intended to treat 34 different conditions.12 Up to that point, the researchers reported that over 1,200 patients had received doses comprising more than 10¹¹ cells with no significant safety issues.
In February 2025, Fertilio ovarian support cells from Gameto that enable egg maturation outside of the body, reducing the time for in vitro fertilization treatments from weeks to days, became the first iPSC therapy to receive U.S. Food and Drug Administration (FDA) approval for a phase III trial.15 Fate Therapeutics received FDA approval for a phase I trial of its allogeneic iPSC CAR-T cell therapy for treatment of active moderate to severe systemic lupus erythematosus (SLE) in April 2025.16 This approval was followed in June 2025 by three more for allogeneic neural iPSC-based therapies developed by XellSmart targeting central nervous system conditions (PD, spinal cord injury, and amyotrophic lateral sclerosis (ALS)).17
Two examples of stem cell therapy trial activity in 2026 include an anticipated end-of-the-year BLA submission by Beam Therapeutics for risto-cel, its stem cell-based gene-edited therapy for treatment of sickle cell disease18 and the launch in June of a large (2000-patient target) Chinese trial investigating the ability of a cord-blood based MSC therapy to support strength and resilience as people age.19
Recent Advances in Stem Cell Understanding and Technology
Technological developments having the greatest impact in the development of stem cell therapies largely related to the generation of iPSCs. Greater understanding of differentiation mechanisms and the signaling pathways, transcription factors, and epigenetic modifications involved have led to improvements in methods for iPSC production.1
One group has bypassed iPSC generation, using a new mix of transcription factors and other compounds to convert skin fibroblasts into nonskin endodermal tissue, which can develop into different internal organs.6 This approach could potentially save time and cost for large-scale manufacturing and possible increase the safety of PSC treatments. Another has generated induced neural stem cells (iNSCs) from dermal fibroblasts or blood cells. Although “artificially constructed,” these cells don’t different from neural stems cells produced from pluripotent stem cells or primary tissue and could potentially be manufactured more quickly and cost-effectively. The iNSCs were shown to reduce neuroinflammation and restore motor control in mice with multiple sclerosis (MS) symptoms and spinal cord injury.
In addition to the approaches to reducing iPSC generation time, advances in manufacturing technologies are enabling more efficient and cost-effective production of stem cell therapies under GMP environments and at larger scales. Examples include introduction of GMP-grade, fully defined xeno-free culture media for derivation and propagation of pluripotent stem cells and ESC banks generated under GMP conditions and bioreactor technology for scalable adherent cell culture.12 Cellistic, meanwhile, has developed a scalable GMP suspension-based process for the differentiation of human iPSCs into cardiomyocytes.20 Achievement of greater than 70% differentiation efficiency avoids the need for downstream purification steps, simplifying the workflow. In addition, compared with 2D processes, the Cellistic platform has a much smaller footprint and provides greater scalability, process consistency, and operational efficiency and significantly improved process economics.
For genetically modified autologous stem cell therapies, decentralized manufacturing solutions have the potential to address cost, time, and logistics challenges. One recent example involves the use of a methylcellulose-based gene therapy foam containing the appropriate viral vector for modifying HSCs in freshly isolated bone marrow aspirate concentrate at the bedside. The treated aspirate is then injected back into the marrow.21
Exosomes as Alternatives to Cell Therapies
Given the many challenges associated with stem cell therapies, many researchers in academia and industry are pursuing the development of exosome treatments as a cell-free alternative.7 Exosomes are the EVs secreted by stem cells containing numerous molecules involved in intracellular communication and many healthy and diseased physiological processes. Using exosomes eliminates the need for managing live cells as drug products while achieving much of the key functionalities of stem cell therapies.
Exosomes are also being designed as drug delivery vehicles.22 This application is attracting attention because exosomes have minimal immunogenicity and can travel through the bloodstream undetected by the immune system. Using various technologies, it is possible to load small molecule, peptide, and RNA-based active drug substances.
Bioprinting and Organoids
Organoids are three-dimensional cellular constructs, typically dispersed on some sort of scaffold, that mimic the structure and function of specific organs.6 They can be generated in various ways from different types of stem cells. One approach receiving significant attention is 3D bioprinting, in which cells and other biomaterials are deposited using 3D printers to form tissues or organs.7 Promising biomaterials used as scaffolds include hydrogels and nanofibers. They are biocompatible and biodegradable and can be integrated with body tissues.8 The bioinks for 3D printing also include transcription factors and other components that facilitate cellular differentiation and cell viability.3
Organoids have also been produced via 3D culture techniques.3 Examples include trophoblast, intestinal, islet, and cortical organoids, blood vessels, and endodontics. An advantage of this method is the ability to establish complex tissue structures that closely mimic those of natural organs.
Beyond their potential for disease treatment through transplantations, organoids are invaluable in drug discovery and development because they serve as a bridge between in vivo testing and traditional 2D systems.23 They are already being employed to investigate organ development, in disease modeling, and for the screening of drug candidates for both toxicity and efficiency.
In addition to 3D organoid cultures for autism spectrum disorder, schizophrenia, and lung diseases,5 CAR-T cell organoids have been developed.3 3D brain organoids within an organ-on-a-chip system, meanwhile, have been used for modeling brain development and diseases. Organoids generated from iPSCs have also been used to investigate metabolic conditions like obesity and liver diseases, colorectal cancer, and cardiac disorders. Organoids derived from patient cells help with the development of personalized cell therapies.23
Future Directions
The global stem cell therapy market is estimated to be expanding at a compound annually growth rate of 12–17% over the next five years.24,25 Currently ASCs account for the vast majority of the market. But iPSC treatments are growing at the fastest rate. From an application perspective, regenerative medicine strongly predominates, but drug discovery and development uses are growing more rapidly. Slightly more than half of current stem cell therapies are allogeneic, but autologous treatments are growing more quickly, largely due to the approval of adoptive cell therapies derived from stem cells. Automation and digitalization are helping to address scalability and manufacturing challenges, while gene editing technologies are facilitating the development of targeted and personalized therapies.
Going forward, wider application of gene editing technologies, further leveraging of biomaterials and bioengineering, and increased use of artificial intelligence (AI) and automation will continue to be key elements of stem cell therapy advances, including in the areas of immune modulation and precision mediicne.1,3 Expansion of developmental cell therapies to more indications beyond blood, central nervous system, and ophthalmic disorders, including immune, cardiac, and endocrine cell conditions, will continue.12 As initial clinical trials reach the readout stage, strong efficiency data is anticipated in areas such as diabetes, epilepsy, PD, and AMD. Developers will then need to focus on achieving practical, cost-effective, larger-scale GMP manufacture of their promising products and designing pivotal trials that provide the evidence that will satisfy regulators regarding both safety and efficacy.
One trend that should be of concern for legitimate cell therapy developers is the shift in FDA policy regarding the thousands of clinics (~2,750 in 2021) offering non-approved stem cell treatments.26 Only in October 2025 did a Supreme Court decision to reconsider an earlier court ruling confirm the agency’s right to regulate unproven stem cell therapies — a result that took seven years to achieve. Under the leadership of Robert F. Kennedy, Jr., however, it appears that the FDA will likely not exercise this authority. The agency may also be loosening stem cell regulations further, despite hundreds of patients having been harmed by unproven treatments.
Stem cell therapies developed in accordance with rigorous GMP requirements and clearly proven to be safe and effective have great potential to treat many different diseases and significantly impact patient lives. Hopefully the actions of Trump Administration appointees will not be detrimental to the continued growth and success of these novel, promising therapies.
References
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16. “Fate Therapeutics Receives Regenerative Medicine Advanced Therapy (RMAT) Designation from FDA for FT819 to Treat Moderate to Severe Systemic Lupus Erythematosus (SLE).” Fate Therapeutics. 14 Apr. 2025.
17. “XellSmart Secures FDA Clearance for Three Phase I INDs of Allogeneic iPSC-Derived Cell Therapies Targeting Major CNS Diseases: Parkinson’s Diseases, Spinal Cord Injury and ALS.” XellSmart Biomedical. 2 Jun. 2025.
18. “Beam Therapeutics Announces Publication of BEACON Phase 1/2 Data for Risto-cel in Patients with Sickle Cell Disease (SCD) in The New England Journal of Medicine.” Beam Therapeutics. 1 Apr. 2026.
19. “Stem Cell Anti-Ageing Trial Launches in China Using Cord Tissue Stem Cells.” Cells4Life. 17 Jun. 2026.
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21. Cummings, Carrie L, et al. “Bedside manufacturing of engineered stem cells using gene therapy foam.” Molecular Therapy – Methods & Clinical Development. 33: 101612 (2025).
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