Stem cell research and regenerative medicine are growing markets in the life science sector, and the top stem cell companies are working hard to make advancements that can help patients.
Stem cells serve as an internal repair system in the body. They can divide without limit to replenish other cells as long as the body is still alive. Unsurprisingly, there’s a lot of promise for medical advancements, and there are already stem cell treatments approved by the US Food and Drug Administration (FDA). Still, there is a lot of room for further advancements, and plenty of lab work still needs to be done before stem cell products can be used as cell-based therapies or regenerative medicines.
A report from Grand View Research projects that the global stem cell market will reach US$28.89 billion by 2030. The research firm sees “the rising number of stem cell banks, growing focus on increasing therapeutic potential of these, and extensive research for the development of regenerative medicines” as drivers of this market. The stem cell market and the value of regenerative medicine stocks is expected to grow further via increased government funding for cancer research and the development of cellular therapies to treat various cancers.
Grand View Research reports that in 2024 the pharmaceutical and biotechnology companies segment captured the largest revenue share for the stem cell therapy market at 54.19 percent.
1. AstraZeneca (NASDAQ:AZN)
Market cap: US$228.3 billion
First on this list of the top NASDAQ stem cell company stocks is multinational pharma and biotech firm AstraZeneca, which also specializes in other therapeutic areas, including oncology, cardiovascular diseases, the respiratory and central nervous systems and pain control.
While it may be the largest company on this list by market cap, the pharma giant has been slower than its peers in staking a position in the cell therapy market. Its particular focus in oncology is the treatment of solid tumors. The company is also looking at stem cell therapies to treat life-threatening chronic kidney disease and liver disease.
AstraZeneca acquired biotech firm Neogene Therapeutics in 2023, bringing the company’s expertise on T-cell receptor therapies into AstraZeneca’s fold.
Its long-term strategy in this area is to develop ‘off-the-shelf’ allogenic CAR-T therapies to increase the availability and accessibility of the treatments.
2. Amgen (NASDAQ:AMGN)
Market cap: US$160.05 billion
A global leader in biotech, Amgen is heavily invested in gene-based research and uses advanced human genetics to develop and manufacture therapeutics targeting a variety of diseases with unmet medical needs.
The biotech firm has a multi-year fund agreement with Canada’s Center for Commercialization of Regenerative Medicine (CCRM), which specializes in developing and commercializing regenerative medicine-based technologies and cell and gene therapies. Under the multi-year agreement, both CCRM and Amgen will fund early stage regenerative medicine-based technologies and therapies with research conducted in institutions that are part of CCRM’s global network.
In May 2024, the US FDA approved Amgen’s Imdelltra for the treatment of adult patients with extensive-stage small cell lung cancer, making the drug the first and only T-cell engager therapy available for treating this condition. The therapy activates the patient’s own T cells to attack particular tumor cells.
3. Gilead Sciences (NASDAQ:GILD)
Market cap: US$137.13 billion
Global biopharmaceutical company Gilead Sciences specializes in developing breakthrough medicines to prevent and treat serious diseases such as HIV, viral hepatitis and cancer.
One of the stem cell therapies in its product portfolio is Yescarta, a CAR-T cell therapy for blood cancer and the first such therapy for certain types of non-Hodgkin’s lymphoma. Yescarta aids a patient’s immune system in fighting the disease. Gilead currently has at least eight cell therapy candidates in its product pipeline, including three in Phase 3 clinical studies targeting high-risk lymphomas and myeloma.
4. Sanofi (NASDAQ:SNY)
Market cap: US$134.81 billion
French multinational pharmaceutical company Sanofi develops therapeutic products for diabetes and cardiovascular diseases, oncology, immunology, multiple sclerosis, rare diseases, rare blood disorders and vaccines. Its product portfolio includes Mozobil, a hematopoietic stem cell mobilizer that helps bone marrow release stem cells into the bloodstream for transplant into the body.
In 2021, the firm bolstered its regenerative medicine business through the US$1.9 billion acquisition of Kadmon, adding FDA-approved stem cell transplant product Rezurock to its portfolio. Sanofi has since partnered with privately held biotech company Scribe Therapeutics to develop CRISPR-based cell therapies targeting cancers.
The company also has a licensing agreement with Innate Pharma (NASDAQ:IPHA) as the two companies collaborate on developing natural killer cell therapeutics in oncology. One investigational drug candidate under this collaboration is SAR’579, which is in Phase 1/2 studies for the treatment of blood cancers with high unmet needs, including relapsed or refractory acute myeloid leukemia, B cell acute lymphoblastic leukemia and high-risk myelodysplasia. SAR’579 received FDA Fast Track Designation for the treatment of acute myeloid leukemia.
5. Vertex Pharmaceuticals (NASDAQ:VRTX)
Market cap: US$123.34 billion
Vertex Pharmaceuticals has developed a number of approved treatments for cystic fibrosis, and has a pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes.
Vertex and its partner CRISPR Therapeutics (NASDAQ:CRSP) co-developed drug Casgevy, a CRISPR/Cas9 genome-edited cell therapy, received FDA approval in December 2023 for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises. Casgevy is the first ever treatment based on CRISPR technology to be approved for the US market. In 2024, the drug received validation from the European Medicines Agency and Health Canada for this indication as well.
In addition, Vertex has two active clinical trials for type 1 diabetes cell therapy: X-880, an investigational allogeneic stem cell-derived, insulin-producing islet cell therapy in Phase 1/2/3 clinical trials; and VX-264, designed to be a surgically implanted device in phase 1/2 clinical trials.
6. BioNTech (NASDAQ:BNTX)
Market cap: US$28.67 billion
Immunotherapy company BioNTech is advancing novel therapies for diseases such as cancer. The company combines computational discovery and therapeutic drug platforms to rapidly develop new biopharmaceutical products. The company is best known today for its mRNA vaccine development and in-house manufacturing capabilities, as well as its COVID-19 vaccine, created with partner Pfizer (NYSE:PFE).
BioNTech is engaged in collaborative partnerships aimed at assembling mRNA vaccine candidates for a range of infectious diseases. BioNTech’s portfolio of oncology product candidates includes individualized and off-the-shelf mRNA-based therapies, CAR-T cell therapies, bispecific checkpoint immuno-modulators, targeted cancer antibodies and small molecules.
The most advanced of its CAR-T product candidates is BNT211, which comprises two drug products designed to induce an immune response against various CLDN6-positive solid tumors, such as ovarian cancer, sarcoma, testicular cancer, endometrial cancer and gastric cancer. BNT211 is in a Phase 1 trial in patients with CLDN6-positive relapsed or refractory advanced solid tumors.
7. BeiGene (NASDAQ:ONC)
Market cap: US$26.72 billion
Biotechnology company BeiGene specializes in the development of drugs for cancer treatment across a broad range of areas, including esophageal squamous cell carcinoma, non-small cell lung cancer, mantle cell lymphoma, non-Hodgkin’s lymphoma and ovarian cancer. Its clinical development pipeline includes 12 advanced Phase 3 programs.
BeiGene has a strategic partnership with Shoreline Biosciences for the development and commercialization of a genetically engineered natural killer cell therapy.
BeiGene’s bruton tyrosine kinase inhibitor Brukinsa gained FDA approval in early 2023 for patients with chronic lymphocytic leukemia or small lymphocytic leukemia, both forms of non-Hodgkin’s lymphoma.
The company’s monoclonal antibody Tevimbra garnered FDA approval in early 2024 for the treatment of advanced or metastatic esophageal squamous cell carcinoma after prior chemotherapy. Near the end of the year, the European Commission gave the green light to the drug for first-line treatment of advanced/metastatic esophageal squamous cell carcinoma and gastric or gastroesophageal junction cancer, while the FDA approved it for first-line treatment of gastric and gastroesophageal junction cancers in combination with chemotherapy.
8. BioMarin Pharmaceutical (NASDAQ:BMRN)
Market cap: US$13.1 billion
BioMarin Pharmaceutical develops and commercializes therapies for patients with serious, life-threatening genetic diseases and medical conditions.
The global biotech company focuses on diseases with critical unmet medical needs, as well as products with an opportunity to be first to market or to offer significant benefits over existing products. BioMarin’s portfolio includes eight commercial products and multiple clinical and preclinical product candidates across its therapeutic areas.
9. Moderna (NASDAQ:MRNA)
Market cap: US$12.98 billion
Biotechnology and pharmaceutical company Moderna is a pioneer in the field of mRNA. The company’s assets include a diverse clinical portfolio of vaccines and therapeutics, plus a large intellectual property portfolio in areas such as mRNA and lipid nanoparticle formulation; it also has an integrated manufacturing plant that allows for both clinical and commercial production.
These assets, along with Moderna’s network of domestic and overseas government and commercial collaborators, allowed for the rapid development of one of the world’s most effective COVID-19 vaccines.
The other therapeutics and vaccines in the company’s pipeline are targeting infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases and autoimmune diseases. Moderna has 23 development programs underway across these areas.
10. Bio-Techne (NASDAQ:TECH)
Market cap: US$10.3 billion
Bio-Techne develops, manufactures and sells reagents, diagnostic instruments and bioprocessing services for research and clinical applications, including drug discovery. The company’s revenue segments include diagnostics and spatial biology, and protein sciences.
Bio-Techne offers tailored technologies and services for its life science customers developing and producing immune cell, regenerative and gene therapies. The company expanded its Advanced Cell Diagnostics branded RNAscope probe portfolio in February 2025.
‘With over 12,000 citations in clinical and translational research combined, our expanded portfolio of gold-standard RNAscope probes enables customers to accelerate biomarker validation and ultimately improve lives,’ said Dr. Matt McManus, president of Bio-Techne’s diagnostics and spatial biology segment.
FAQs for stem cell stocks
What are stem cells?
Stem cells are the building blocks of life, with special capabilities that are particularly important in both the early and later stages of a human’s lifecycle. Human stem cells have the ability to develop into a variety of different cell types in the body, including organ-specific cells, as well as muscle tissue and bone marrow cells; they can even renew themselves.
What is stem cell therapy?
Stem cell therapy is the use of stem cells to treat or prevent a disease or condition, including some cancers such as leukemia, lymphoma, multiple myeloma and neuroblastoma. Cell therapy involves the direct administration of cells into the body to promote the body’s natural ability to heal itself.
What is stem cell banking?
Stem cell banking is the process of collecting, processing and cryogenically storing potentially life-saving stem cells for future use in therapies and regenerative medicine.
Securities Disclosure: I, Melissa Pistilli, hold no direct investment interest in any company mentioned in this article.