Overview
Stem cell therapies are moving out of the lab and into the clinic, with early trials showing real signal and regulators opening faster paths to approval. These companies pushing the field toward standard care.
Stem cells can differentiate into any of the body’s cell types, making them an invaluable source of treatments in regenerative medicine. Stem cell therapies have, for example, the potential to repair heart tissue damaged by a heart attack, regenerate neurons in Parkinson’s disease, or replace lost insulin-secreting cells in diabetes.
Such therapies could “potentially revolutionize treatment options for people suffering from a number of degenerative disorders,” says Francisco Silva, Vice President of Research and Development, for BioRestorative Therapeutics. BioRestorative is developing stem cell therapies for chronic pain and metabolic disorders. “Regenerative medicine is now at the cusp of becoming part of the standard of care and a valuable therapeutic tool,” says Silva.
Researchers obtain stem cells for regenerative medicine from embryos or certain adult tissues, such as bone marrow, or from adult somatic cells—typically skin or blood—that are coaxed into an embryonic-like state in the lab. They then differentiate the stem cells into precursors of the desired cell type, such as dopamine-producing neurons, which are then implanted in the brain or other organ of the patient.
Two recent early-stage clinical trials led by academic researchers grabbed scientists’ attention last year as they were the first to show that stem cell therapy has clear potential to treat people with Parkinson’s disease. The researchers showed that the therapies were safe and reduced the motor symptoms of the neurodegenerative disorder. Multiple other stem-cell trials are underway.
Through these trials, “we are witnessing the emergence of a new type of stem cell medicine that is well-substantiated and scientifically proven,” says Aitor Aguirre, PhD, Chief of the Division of Developmental and Stem Cell Biology at Michigan State University. That’s in contrast to unapproved stem cell treatments of previous decades, which brought the field into disrepute.
Moreover, the clinical trial environment for regenerative medicine therapies is improving. FDA guidance published in September 2025 enables regenerative medicine companies to use flexible trial designs and an expedited approval program. Regulatory agencies are more open to working with companies developing stem cell therapies and aiding in the path towards approval, too. “This approach has provided companies with paths for quicker clinical development and approval without compromising patient safety,” says Silva.
We’ve curated companies that are advancing stem cell therapies through clinical trials, several of which have recently reported preliminary results.
Aspen Neuroscience
Sector: Stem cell therapies for neurological disorders
HQ: San Diego, CA
Year Founded: 2018
Origin Story: Founded by stem-cell biologist Jeanne Loring, who was the founding director of the Center for Regenerative Medicine at Scripps Research Institute.
Key Leaders: Damien McDevitt, PhD, President and Chief Executive Officer; Kim Raineri, Chief Technology Officer; Revati Shreeniwas, MD, Chief Medical Officer; Xiaokui Zhang, PhD, Chief Scientific Officer
Number of Employees: ~123
Stage: Private, Series C
Financial Snapshot: ~$338.5 million in several rounds
Notable Investors: Balyasny Asset Management, Cormorant Asset Management, Frazier Lifesciences Acquisitions, Kite Pharma, Prebys Ventures, Domain Associates, Axon Ventures, OrbiMed, Google Ventures, LYFE Capital, Revelation Partners, ARCH Venture Partners
Key Products: Aspen’s lead product is ANPD001, an iPSC patient-derived, dopaminergic neuronal precursor cell therapy that’s in a phase 1/2 trial for treating Parkinson’s disease.
Recent Highlights: The company in May 2025 released clinical results showing that ANPD001 improved motor function in three patients, assessed by clinician- and patient-reported scores, and had a good safety and tolerability profile.
BlueRock Therapeutics
Sector: Cell and gene therapy
HQ: Cambridge, MA
Year Founded: 2016
Origin Story: Founded by Lorenz Studer and Viviane Tabar from Memorial Sloan Kettering Cancer Center, and the University of Toronto’s Gordon Versant.
Key Leaders: Seth Ettenberg, PhD, President and CEO; Amit Rakhit, MD, Chief Development and Medical Officer; Stefan Irion, MD, Chief Scientific Officer
Number of Employees: ~459
Stage: Private, owned by Bayer
Financial Snapshot: Acquired by Bayer for $240 million upfront and run as an independent company
Notable Investors: Versant Ventures and Leaps by Bayer
Key Products: The company’s lead product is bemdaneprocel, a human embryonic stem cell donor-derived dopaminergic neuron progenitor that’s in a phase 3 trial for Parkinson’s disease. A second product, OpCT-001, is derived from iSPCs and is in a phase 1 trial for primary photoreceptor diseases.
Recent Highlights: In October 2025, the company showed that bemdaneprocel improved motor symptoms and had a favorable safety profile.
Neurona Therapeutics
Sector: Stem cell therapies for neurological disorders
HQ: South San Francisco, CA
Year Founded: 2008
Origin Story: Cory Nicholas and Arnold Kriegstein, researchers at the University of California, San Francisco, and Gautam Banik.
Key Leaders: Cory Nicholas, PhD, Co-Founder and Chief Executive Officer; Catherine Priest, PhD, Chief Development Officer; Gautam Banik, PhD, Chief Technology Officer; Eduardo Dunayevich, MD, Chief Medical Officer
Number of Employees: ~98
Stage: Private, Series F
Financial Snapshot: Around $300 million in several rounds. The company’s most recent deal was an upsized and oversubscribed $102 million financing in 2025.
Notable Investors: Viking Global Investors, Cormorant Asset Management, Fidelity Management and Research Company, Soleus Capital, YK Bioventures, Iaso Ventures
Key Products: Neurona’s lead product NRTX-1001 is an allogeneic therapy comprising hPSC-derived inhibitory GABAergic interneurons that’s in phase 1/2 trials for treating drug-resistant focal epilepsy.
Recent Highlights: Clinical results presented in December 2025 showed that a one-time dose of NRTX-1001 reduced seizures by 58–93%.
Vertex Pharmaceuticals
Sector: Global company with marketed products including stem cell therapies
HQ: Boston, MA
Year Founded: 1989
Key Leaders: Reshma Kewalramani, MD, Chief Executive Officer and President; Mark Bunnage, D.Phil, Chief Scientific Officer; Carmen Bozic, MD, Chief Medical Officer; E. Morrey Atkinson, PhD, Chief Technical Operations Officer
Number of Employees: ~5,000 in the U.S.
Stage: Public
Financial Snapshot: The company’s market cap was $121.24 billion in March 2026
Key Products: Vertex’s lead stem-cell product is zimislecel, an allogenic hPSC-derived, insulin-producing islet cell therapy for type 1 diabetes that’s in a phase 1/2 trial. Trikafta/Kaftrio and Alyftrek, as well as Casgevy are produced for treating sickle cell disease and beta thalassemia, and Journavx is used for treating pain
Recent Highlights: Results published in the New England Journal of Medicine in 2025 showed that zimislecel eliminated severe hypoglycemic events with some patients no longer needing to take insulin.
BioCardia
Sector: Cardiovascular regenerative medicine
HQ: Sunnyvale, CA
Year Founded: 1994
Origin Story: Founded by Simon Stertzer, a cardiologist who helped bring coronary angioplasty to the United States in the 1970s.
Key Leaders: Peter Altman, PhD, President and CEO; Debby Holmes-Higgin, MPH, Vice-President of Clinical
Number of Employees: ~34
Stage: Public (BCDA)
Financial Snapshot: The company’s market cap was about $13.37 million in March 2026.
Key Products: The company’s lead program is CardiAMP, a patient-derived, mononuclear cell therapy derived from mesenchymal stem cells for treating ischemic heart failure that’s in a phase 3 trial. A donor version of this therapy, known as CardiALLO, is in a phase 2 trial.
Recent Highlights: The company released data in March 2025 showing that in 115 people with ischemic heart failure with reduced ejection fraction, CardiAMP increased survival, reduced cardiac events, and improved quality of life, but did not improve heart function measured by the six-minute walking test – the study’s primary outcome measure.
Lineage Cell Therapeutics
Sector: Cell therapy
HQ: Carlsbad, CA
Year Founded: 1990 (known as BioTime)
Key Leaders: Brian Culley, MBA, Chief Executive Officer; Rami Skaliter, PhD, Chief Executive Officer of subsidiary Cell Cure Neurosciences;
Number of Employees: ~77 employees
Stage: Public
Financial Snapshot: The company’s market cap was approximately $429.56 million in March 2026
Key Products: The company’s lead product is OpRegen, a hESC-derived, allogeneic, retinal pigment epithelial cell therapy for treating eye disorders that happen due to age-related macular degeneration. It’s in a phase 2 clinical trial and is being developed in partnership with Roche/Genentech. Another product, OPC1, is a hESC-derived oligodendrocyte progenitor cell therapy for spinal cord injuries that’s in phase 2 studies.
Recent Highlights: Phase 1/2a results released in June 2025 showed that in patients with geographic atrophy, a single dose of OpRegen improved patients’ visual acuity and the structure of their retina also improved.
Gameto
Sector: Reproductive health
HQ: New York, NY
Year Founded: 2020
Origin Story: After Dina (Radenkovic) Turner and Martin Varsavsky founded the company, they worked with a lab at Harvard’s Wyss Institute to research cell engineering. Their work together inspired the science to mature eggs outside a woman’s body, and led to the development of their product, Fertilo.
Key Leaders: Dina (Radenkovic) Turner, Co-founder and Chief Executive Officer; Christian Kramme, PhD, Chief Scientific Officer; Gus Haddad, MD, Chief Medical Officer
Number of Employees: ~49
Stage: Series C
Financial Snapshot: $127 million across several rounds
Notable Investors: Overwater Ventures; Insight Partners; RA Capital; Two Sigma Ventures; BOLD Capital Partners; Future Ventures; Ingeborg Investments; Arcadia Investment Partners; PAGS Group; Pontiva Healthcare Partners; Portfolia
Key Products: Gameto’s lead product is Fertilo, an iPSC-derived system of ‘ovarian support cells’ designed to improve the in-vitro maturation of human oocytes and hence boost IVF outcomes. Fertilo is in phase 3 trials in the United States and approved in some countries including Australia, Argentina, Peru, and Mexico.
Recent Highlights: A February 2026 paper in Cell Stem Cell described how transcription factor-mediated hiPSC differentiation produces ovarian support cells— that is, Fertilo—which improve the oocyte maturation rate, which in turn increases the number of IVF-mediated pregnancies and births.
Longerveron
Sector: Regenerative medicine
HQ: Miami, FL
Year Founded: 2014
Origin Story: Founded by Joshua Hare, director of the University of Miami’s Interdisciplinary Stem Cell Institute
Key Leaders: Stephen Willard, Chief Executive Officer; Joshua Hare, MD, Chief Science Officer and Executive Chairman; Nataliya Agafonova, MD, Chief Medical Officer; Devin Blass, Chief Technology Officer
Number of Employees: ~25
Stage: Public
Financial Snapshot: The company’s market cap was $24.11 million as of March 2026.
Key Products: The company’s lead product is laromestrocel (Lomecel-B), which is derived from culture-expanded mesenchymal stem cells sourced from the bone marrow of young adult donors. It’s in clinical trials for mild Alzheimer's disease, aging frailty, and a congenital heart disorder.
Recent Highlights: February 2026 results from a phase 2b clinical trial showed that laromestrocel improved physical function in people with age-related frailty.
Immunis
Sector: Age-related disorders
HQ: Irvine, CA
Year Founded: 2019
Origin Story: Founded by stem cell expert and entrepreneur Hans Keirstead.
Key Leaders: Hans Keirstead, PhD, Chair; Tom Lane, PhD Chief Science Officer; Erin Curry, PA-C, MPH, Director of Medical Affairs
Number of Employees: ~19
Stage: Private, Series A
Financial Snapshot: Closed $25 million Series A-1 financing round in January 2025.
Notable Investors: Elevation Capital, Fairfax Financial Holdings, Spartan Capital Securities, Remiges Ventures, Continuum Health Ventures, BOLD Capital Partners, LifeSpan Vision Ventures, JLS Fund
Key Products: Immunis’ lead product is IMM01-STEM, which is composed of molecules derived from stem cells, such as extracellular vesicles, and growth, cytoskeletal remodeling, and immunomodulatory factors. It’s in a phase 2 trial in people with muscle loss and obesity, or muscle loss and knee arthritis.
Recent Highlights: Preliminary results released in January 2026 showed that IMM01-STEM improved physical function in overweight, older people with muscle loss; their gait speed increased by 26%.
Orca Bio
Sector: Cell therapy
HQ: Menlo Park, CA
Year Founded: 2016
Origin Story: Founded by Ivan Dimov, Nate Fernhoff, and Jeroen Bekaert who met at Stanford University based on research conducted in the lab of Irv Weissman at Stanford.
Key Leaders: Jeroen Bekaert, PhD, President; Nate Fernhoff, PhD, Chief Executive Officer; Scott McClellan, MD, Clinical Development
Number of Employees: ~279
Stage: Private, Series F
Financial Snapshot: Raised $250 million in January 2026 to prepare for possible commercialization of its lead product
Notable Investors: Lightspeed Venture Partners, DCVC, Dream Variation Ventures, Edgewood Ventures, Illinois Municipal Retirement Fund, Kaiser Foundation Hospitals
Key Products: The company’s lead product is Orca-T, an allogeneic stem cell-derived therapy that consists of regulatory T-cells, other T cells and hematopoietic stem cells to replace diseased bone marrow in people with blood cancers. It completed a phase 3 trial and has a PDUFA date of April 6, 2026. A next-gen version of this therapy called Orca-Q, designed not to need a fully matched donor, is in a phase 1 trial.
Recent Highlights: Phase 1 results released in February 2026 showed that compared to standard stem-cell transplants, Orca-T reduced graft-versus-host disease and improved patient survival.
