Global Stem Cell Therapy Market Outlook, 2031

The global stem cell therapy industry covers treatments that use stem cells or cells derived from them to repair, replace or regenerate damaged tissues, or to modulate the immune system. The field includes haematopoietic stem cell transplantation, long used to treat blood cancers and inherited blood disorders; mesenchymal stromal cell therapies for inflammatory and degenerative conditions; cell products derived from embryonic stem cells and induced pluripotent stem cells, which can become almost any cell type; tissue-specific stem cell products for skin, cornea and cartilage repair; and gene-modified stem cell therapies such as Casgevy and Lyfgenia for sickle cell disease. Approved products include Mesoblast's Ryoncil, the first mesenchymal stromal cell therapy approved in the United States in December 2024 for steroid-refractory acute graft-versus-host disease in children; Gamida Cell's Omisirge for improving cord blood transplantation; CellTrans's Lantidra for type 1 diabetes; Holoclar for corneal damage in Europe; and several products approved in Japan and South Korea. Major participants include Vertex, Mesoblast, Sumitomo Pharma, BlueRock Therapeutics owned by Bayer, Novo Nordisk, Lineage Cell Therapeutics, Cuorips, Astellas, Takeda and many academic transplant centres and cord blood banks.

Stem cell therapy reached a historic milestone in 2026. In February and March 2026, Japan's health ministry approved the world's first therapies derived from induced pluripotent stem cells: Sumitomo Pharma's Amchepry (raguneprocel), dopaminergic neural progenitor cells for Parkinson's disease, and Cuorips' ReHeart, iPSC-derived heart muscle cells for heart failure, both under Japan's conditional and time-limited approval pathway. The approvals built on Nobel Prize-winning iPSC research by Shinya Yamanaka and HLA-matched cell banks developed at Kyoto University. In type 1 diabetes, Vertex reported in June 2025 that most patients receiving its stem cell-derived islet therapy zimislecel became insulin-independent, and Bayer's BlueRock is running a phase 3 trial of stem cell-derived dopamine neurons for Parkinson's disease. In the United States, the FDA's approval of Ryoncil in December 2024 ended years of setbacks for Mesoblast. Other developments have been less positive: Takeda withdrew its mesenchymal cell product Alofisel from Europe in December 2024 after a confirmatory trial failed, and regulators continue to act against clinics offering unproven stem cell treatments. The field faces challenges in manufacturing, immune rejection, tumour risk, high costs and demonstrating durable benefit, but growing clinical evidence and new pathways for regenerative medicine are driving investment.

Market Dynamics Market Drivers • Unmet Need in Degenerative and Chronic DiseasesMany conditions, including Parkinson's disease, heart failure, type 1 diabetes, spinal cord injury, macular degeneration, osteoarthritis and stroke, involve loss of cells that existing drugs cannot replace. Stem cell therapies aim to restore function by replacing lost cells or promoting repair. Ageing populations and rising chronic disease prevalence increase the potential patient population and the value of regenerative treatments.
• Scientific Progress and Supportive Regulatory PathwaysAdvances in stem cell biology, differentiation protocols, gene editing to create hypoimmune cells, and manufacturing have made it possible to produce specific cell types at scale. Regulatory pathways such as Japan's conditional approval system for regenerative medicine, the US regenerative medicine advanced therapy designation and the EU's advanced therapy framework support development. The first iPSC approvals in 2026 have validated the field.

Market Challenges • Manufacturing Complexity and High CostsProducing stem cell therapies requires complex, controlled processes, specialised facilities and extensive quality testing, and products must be consistent, pure and free of undifferentiated cells that could form tumours. Scaling manufacturing while maintaining quality is difficult and expensive, and the resulting therapies are costly. Reimbursement for high-priced regenerative therapies is uncertain in many countries.
• Safety, Immune Rejection and Unproven TreatmentsDonor-derived cells can be rejected by the immune system, requiring immunosuppression or HLA matching, and pluripotent stem cell products carry theoretical risks of tumour formation. Long-term safety and durability are still being established. Clinics offering unproven stem cell treatments, sometimes causing serious harm, damage public trust and have prompted regulatory crackdowns in the United States and elsewhere.

Market Trends • Induced Pluripotent Stem Cell-Derived TherapiesiPSC technology allows production of specific cell types, including neurons, heart cells, pancreatic islets, retinal cells and immune cells, from reprogrammed adult cells. Japan's approvals of Amchepry and ReHeart in 2026 were the first for iPSC-derived products, and many more programmes are in trials worldwide. HLA-matched cell banks and gene-edited hypoimmune cells aim to allow off-the-shelf use without strong immunosuppression.
• Stem Cell-Derived Islets for Type 1 DiabetesStem cell-derived insulin-producing islet cells offer the potential to cure type 1 diabetes by restoring the body's insulin production. Vertex's zimislecel produced insulin independence in most treated patients in its trial and is moving toward regulatory submission, while Vertex, Sana Biotechnology and others are developing versions that avoid immunosuppression through encapsulation or gene editing.



Segment Analysis Haematopoietic stem cell therapies account for the largest share of stem cell treatment, while mesenchymal and pluripotent stem cell-derived therapies are the fastest-growing product segments. Haematopoietic stem cells, collected from bone marrow, peripheral blood or umbilical cord blood, are used in tens of thousands of transplants each year to treat leukaemia, lymphoma, myeloma, aplastic anaemia and inherited disorders, making them the largest and most established segment. Gene-modified haematopoietic stem cell therapies, including Casgevy, Lyfgenia and treatments for rare immune and metabolic disorders, add high-value products. Mesenchymal stromal cells, derived from bone marrow, adipose tissue or umbilical cord, are used for their immunomodulatory and repair-promoting effects, with Ryoncil approved in the United States and several products approved in Japan, South Korea and India. Pluripotent stem cell-derived therapies, including those from embryonic stem cells and induced pluripotent stem cells, produce specific cell types such as dopaminergic neurons, cardiomyocytes, pancreatic islets and retinal pigment epithelium, and the first iPSC products were approved in Japan in 2026. Tissue-specific adult stem cell products, such as limbal stem cells for corneal repair and cultured skin and cartilage cells, form a smaller segment. Neural stem cell therapies are studied for spinal cord injury, stroke and neurodegenerative disease.

Allogeneic stem cell therapies are increasingly favoured for scalable products, while autologous therapies remain important in transplantation and some regenerative applications. Allogeneic stem cell therapies use cells from donors, allowing large-scale manufacturing and off-the-shelf availability, and most mesenchymal and pluripotent stem cell-derived products, including Ryoncil, Amchepry and ReHeart, are allogeneic. They may require immunosuppression, HLA matching or gene editing to avoid rejection, and Japan's HLA-homozygous iPSC banks allow one cell line to match a large share of the population. Allogeneic haematopoietic stem cell transplantation from matched or partially matched donors is widely used for blood cancers. Autologous stem cell therapies use the patient's own cells, avoiding rejection, and include autologous haematopoietic stem cell transplantation for myeloma and lymphoma, gene-modified autologous stem cell therapies such as Casgevy, and some regenerative products for cartilage and heart repair. Autologous iPSC therapies are technically possible but expensive and slow to produce. Hypoimmune gene-edited cells, designed to evade immune recognition, aim to combine the scalability of allogeneic products with reduced need for immunosuppression.

Haematological and oncological conditions account for the largest current use of stem cell therapy, while neurological, cardiovascular, metabolic and musculoskeletal diseases are the fastest-growing application areas. Haematological and oncological conditions, including blood cancers treated with stem cell transplantation, inherited blood disorders such as sickle cell disease and thalassaemia, and graft-versus-host disease after transplantation, account for most current stem cell therapy use. Neurological conditions, including Parkinson's disease, where Amchepry was approved in Japan and BlueRock's therapy is in phase 3, spinal cord injury, stroke, amyotrophic lateral sclerosis and multiple sclerosis, are major research areas. Cardiovascular diseases, including heart failure, where ReHeart was approved in Japan, and ischaemic heart disease, are a growing focus. Metabolic diseases, especially type 1 diabetes, have seen breakthroughs with stem cell-derived islets. Musculoskeletal conditions, including osteoarthritis, cartilage defects, bone fractures and tendon injuries, are treated with mesenchymal cell products in some countries. Ophthalmology, including macular degeneration, retinitis pigmentosa and corneal damage, has several programmes and approved products. Autoimmune and inflammatory diseases, including Crohn's fistulas and graft-versus-host disease, use mesenchymal cells. Wound healing, liver disease and ageing-related conditions form further areas.

Hospitals and transplant centres are the main end users of stem cell therapies, while specialised regenerative medicine clinics, research institutes and cord blood banks play important roles. Hospitals and transplant centres perform haematopoietic stem cell transplants and administer approved regenerative and gene-modified stem cell therapies, and they are the main end users. Specialised treatment centres are required for gene therapies such as Casgevy and for surgical delivery of cell therapies into the brain or heart, as with Amchepry. Academic medical centres and research institutes, such as Kyoto University's Center for iPS Cell Research and Application, conduct clinical trials and develop new therapies. Cord blood banks, public and private, store umbilical cord blood for transplantation and research. Contract development and manufacturing organisations produce cell therapies for developers. Regenerative medicine clinics in Japan, South Korea and other countries provide treatments under specific regulatory frameworks, while unregulated clinics in some countries offer unproven therapies. Pharmaceutical and biotechnology companies develop products and partner with hospitals for trials. Payers, including national health insurance in Japan, influence access through reimbursement decisions on newly approved products.

Regional Analysis North America leads the stem cell therapy market by value because of its large transplant sector, biotechnology industry and early approvals of gene-modified stem cell therapies, while Japan has become the global leader in approving iPSC-derived therapies. North America, led by the United States, is the largest stem cell therapy market by value, with a large haematopoietic transplant sector, the first approvals of gene-modified stem cell therapies for sickle cell disease in 2023, the approval of Ryoncil in December 2024 and many leading developers, including Vertex, BlueRock, Sana and Lineage. The FDA's regenerative medicine advanced therapy designation supports development, while enforcement against unproven stem cell clinics continues. Europe is a major market, with strong transplant networks, approved products such as Holoclar, advanced therapy regulation and research in the UK, Germany, Sweden and Spain, although some products have been withdrawn, including Alofisel in 2024. Asia-Pacific is the fastest-growing region. Japan became the first country to approve iPSC-derived therapies in 2026, Amchepry for Parkinson's disease and ReHeart for heart failure, under its conditional approval pathway, and has several other approved regenerative products. South Korea has approved several stem cell products, China has large clinical research programmes and is developing its regulatory framework, and India, Australia and Singapore have growing activity. Latin America and the Middle East have developing transplant and regenerative medicine sectors, with some countries also facing unregulated clinics.

Key Developments March 2026: Japan granted conditional, time-limited approval to Sumitomo Pharma's Amchepry for Parkinson's disease and Cuorips' ReHeart for heart failure, the world's first approved therapies made from induced pluripotent stem cells.
June 2025: Vertex published data showing that most patients with severe type 1 diabetes treated with its stem cell-derived islet therapy achieved insulin independence.
December 2024: Mesoblast's remestemcel-L became the first mesenchymal stromal cell therapy approved in the United States, for children with steroid-refractory acute graft-versus-host disease.
December 2024: Takeda withdrew its stem cell therapy for complex perianal fistulas in Crohn's disease after a confirmatory phase 3 trial failed to show benefit.
June 2023: CellTrans's donor-derived pancreatic islet cell therapy was approved for adults with type 1 diabetes and recurrent severe hypoglycaemia.
April 2023: Gamida Cell's omidubicel, a modified cord blood product, was approved to speed recovery of blood cells after stem cell transplantation in patients with blood cancers.

Considered in this report
• Historic Year: 2020
• Base Year: 2025
• Estimated Year: 2026
• Forecast Year: 2031

Aspects covered in this report
• Global Stem Cell Therapy Market with its value and forecast along with its segments
• Various drivers and challenges
• Ongoing trends and developments
• Top profiled companies
• Strategic recommendation

By Cell Type
• Haematopoietic Stem Cells
• Mesenchymal Stromal Cells
• Pluripotent Stem Cell-Derived Cells
• Tissue-Specific Adult Stem Cells

By Therapy Type
• Allogeneic
• Autologous

By Application
• Haematological and Oncological Conditions
• Neurological Disorders
• Cardiovascular Diseases
• Metabolic Diseases
• Musculoskeletal Disorders
• Ophthalmic Disorders

By End User
• Hospitals and Transplant Centres
• Specialised Treatment Centres
• Research Institutes
• Cord Blood Banks