Stem cell therapy’s avant-garde: Eight companies leading the way

The field of stem cell therapy has gained traction over recent years, with companies transitioning from experimental stages to delivering practical treatments. Stem cells, with their ability to differentiate into various cell types, hold great potential in regenerative medicine. 

Some notable advancements in stem cell therapy include its application in treating neurodegenerative diseases such as Parkinson’s disease. Companies like BlueRock Therapeutics have pioneered the development of dopamine-producing neural cells derived from stem cells, potentially altering the disease course. Stem cell therapy has also shown potential in repairing damaged heart tissue or in cancer treatment research. And major players such as Vertex Pharmaceuticals are advancing stem-cell-derived islet cell therapies for type 1 diabetes. 

Here are eight companies worth keeping an eye on in the stem cell landscape. 

Aspen Neuroscience 

• Founded: 2018 
• Headquarters: San Diego, California, U.S. 
• Lead candidate: ANPD001 

Founded in 2018 and headquartered in San Diego, Aspen Neuroscience is a clinical-stage biotech developing personalized, autologous stem-cell therapies for neurodegenerative diseases, starting with Parkinson’s disease (PD). 

Its lead candidate, ANPD001, is an autologous, induced pluripotent stem cells (iPSC)-derived dopaminergic neuronal precursor cell (DANPC) therapy aimed at replacing the dopamine-producing neurons lost in PD. The process begins with a small skin biopsy from the patient; these cells are reprogrammed into iPSCs, differentiated into DANPCs, and surgically implanted into the putamen. Because the cells are autologous, the therapy avoids immune rejection and removes the need for long-term immunosuppression. 

In April 2024, Aspen dosed the first patient in its phase 1/2a trial, the first multi-patient, multi-center autologous neuronal replacement therapy trial in Parkinson’s disease in the U.S. 

By early 2025, the first two cohorts had completed dosing, and in May 2025 the company reported six-month data for the first three patients: ANPD001 was well tolerated, with no serious safety issues, and patients showed early, clinician- and patient-reported improvements in motor symptoms and daily living, without immunosuppression. In late 2025, the company raised $115 million in a series C financing round. 

BlueRock Therapeutics 

• Founded: 2016 
• Headquarters: Cambridge, Massachusetts, U.S. 
• Lead candidate: BRT-DA01 

Founded in 2016 and headquartered in Cambridge, BlueRock Therapeutics (a subsidiary of Bayer AG) develops regenerative medicines derived from pluripotent stem cells, with programs spanning neurology, cardiology, immunology, and ophthalmology. 

Its lead program, bemdaneprocel (BRT-DA01), is an allogeneic pluripotent stem-cell–derived dopaminergic neuron progenitor therapy designed to replace the dopamine-producing neurons lost in Parkinson’s disease. 

The therapy completed a phase 1 trial in which it met the primary endpoint of safety and tolerability. In 2025, BlueRock reported 36-month follow-up results showing persistent graft survival (confirmed via PET imaging) and meaningful motor improvements in the high-dose cohort. 

Building on these results, bemdaneprocel advanced to a registrational phase 3 trial, exPDite-2. This makes it one of the first pluripotent stem-cell-derived therapies and the most advanced allogeneic neuronal replacement therapy to reach a pivotal study in Parkinson’s disease. 

Beyond its Parkinson’s program, BlueRock is expanding its pipeline with OpCT-001, a pluripotent stem-cell-derived photoreceptor therapy that recently entered early clinical testing for inherited retinal diseases.  

BrainXell 

• Founded: 2015 
• Headquarters: Madison, Wisconsin, U.S. (therapeutics division in San Diego) 
• Lead candidate: BXT-110 

BrainXell is an emerging player in regenerative neurology, building on a decade of expertise in producing high-purity neurons and glia from human iPSCs. Originally focused on supplying subtype-specific human central nervous system (CNS) cells for research and drug discovery, the company expanded into therapeutic development with the launch of BrainXell Therapeutics, its San Diego–based division dedicated to translating this manufacturing expertise into cell therapies for neurological disorders. 

Its lead program, BXT-110, comprises iPSC-derived midbrain dopaminergic progenitors (mDAPs) designed to replace the neurons lost in Parkinson’s disease. Recent 2025 preclinical data showed robust differentiation and encouraging in vivo results in rodent models, including >60% graft survival and functional behavioral improvements within 12 weeks post-transplantation.  

In parallel, BrainXell continues to advance its broader iPSC platform, including 3D neurovascular models and high-purity oligodendrocyte progenitor cells, reinforcing its position at the intersection of CNS model development and next-generation cell replacement therapies. While still preclinical, the company’s technical depth and newly reported data make it a notable early-stage company to watch in the landscape. 

Fate Therapeutics 

• Founded: 2007 
• Headquarters: San Diego, California, U.S. 
• Lead programs: FT522, FT596, FT819 

Fate Therapeutics is developing universal, off-the-shelf immunotherapies derived from clonal iPSC master lines. The company’s approach enables standardized, industrial-scale manufacturing of uniform NK and T cell products, designed to overcome the variability, complexity, and cost barriers associated with donor-sourced or autologous cell therapies. 

Fate’s iPSC-derived natural killer (NK) cell programs have shown promising activity in early clinical studies. FT522, a CD19-directed chimeric antigen receptor (CAR) NK therapy, has demonstrated good safety and signs of biological activity in patients with B-cell lymphomas, notably without requiring preparative chemotherapy. FT596, another engineered NK cell therapy targeting multiple tumor markers, showed a favorable safety profile along with objective tumor responses across several types of B-cell cancers. 

Fate is also advancing the field of iPSC-derived CAR T cells in autoimmunity. In 2025, the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to FT819, an off-the-shelf CAR T therapy for systemic lupus erythematosus (SLE) and lupus nephritis.  

Heartseed 

• Founded: 2015 
• Headquarters: Tokyo, Japan 
• Lead candidate: HS-001 

Heartseed is a Japanese biotech company focused on regenerative therapies for severe heart failure, built around allogeneic iPSC-derived ventricular cardiomyocytes. Its lead program, HS-001, consists of highly purified cardiomyocyte spheroids designed to remuscularize the failing left ventricle by engrafting into the myocardium, beating in synchrony with the patient’s own heart, and improving contractile function. 

HS-001 is being evaluated in a phase 1/2, open-label, dose-escalation trial in patients with advanced ischemic heart failure undergoing planned coronary artery bypass graft surgery. The cells are injected directly into the ventricular wall during open-heart surgery, with safety and changes in cardiac function as key endpoints. Enrollment was completed in early 2025. 

Heartseed is also advancing HS-005, a next-generation program that uses the same cardiomyocyte spheroids delivered via a catheter-based endocardial injection system rather than open-heart surgery. The phase 1/2 study will evaluate this less invasive approach in patients with heart failure with reduced ejection fraction (HFrEF) due to ischemic heart disease or dilated cardiomyopathy. 

The company previously entered a global collaboration with Novo Nordisk around HS-001. That partnership was discontinued in 2025 as Novo reassessed its iPSC strategy, leaving Heartseed to carry the program forward on its own. 

Orca Bio 

• Founded: 2016 
• Headquarters: Menlo Park, California, U.S. 
• Lead candidate: Orca-T 

Orca Bio is a late-stage biotech developing high-precision allogeneic cell therapies for blood cancers and autoimmune diseases. Its products are built using a proprietary platform that sorts donor blood at single-cell resolution to generate defined mixtures of stem and immune cells, with the goal of delivering curative transplants with fewer complications than conventional allogeneic hematopoietic stem cell transplantation. 

The company’s lead therapy, Orca-T, is an investigational allogeneic T-cell immunotherapy composed of highly purified CD34⁺ hematopoietic stem and progenitor cells, regulatory T cells, and conventional T cells from matched related or unrelated donors. Orca-T is designed to rebuild the patient’s blood and immune system while lowering the risk of graft-versus-host disease (GVHD). 

In the pivotal phase 3 trial, Orca-T was compared head-to-head against standard allo-HSCT in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndromes (MDS), and mixed-phenotype acute leukemia (MPAL).  

The study met its primary endpoint: patients treated with Orca-T had a significantly higher rate of survival free from moderate-to-severe chronic GVHD than those receiving conventional transplants, while maintaining high rates of disease control.  

On the back of these results, in October 2025 the FDA accepted Orca Bio’s biologics license application (BLA) for Orca-T and granted it priority review for select hematologic malignancies, with a target decision date in April 2026. If approved, Orca-T would become the first allogeneic T-cell immunotherapy for this setting. 

Sana Biotechnology 

• Founded: 2018 
• Headquarters: Seattle, Washington, U.S. 
• Lead program: UP421 

Sana Biotechnology develops engineered pluripotent stem cells and donor-derived cells designed to function as off-the-shelf medicines. Its central innovation is the hypoimmune (HIP) platform, which uses gene editing to make cells invisible to the immune system.  

Sana’s most advanced application of this platform is in type 1 diabetes (T1D). In January 2025, the company announced the first human results for UP421, a hypoimmune-modified, donor-derived islet cell therapy. In the first treated patient, transplanted cells survived without immunosuppression and produced detectable insulin, confirmed by measurable C-peptide and MRI evidence of graft survival at 28 days. Follow-up data released in mid-2025 reinforced these findings: six months after transplant, the patient continued to show insulin production, including a responsive rise in C-peptide during a mixed-meal tolerance test. 

These results were significant enough to be published in the New England Journal of Medicine in August 2025. This publication represented the first peer-reviewed evidence that engineered allogeneic cells, transplanted without immune suppression, can survive and function in a human with T1D. 

Sana has since prioritized its diabetes pipeline, streamlining earlier work in glial progenitor cells and other hypoimmune cell types to focus resources on advancing the first-in-human islet program and preparing for a stem-cell-derived islet candidate expected to enter the clinic in 2026. 

Stratus Therapeutics (formerly Garuda Therapeutics) 

• Founded: 2021 
• Headquarters: Cambridge, Massachusetts, U.S. 
• Lead platform: Stratus Prime 

Stratus Therapeutics, known as Garuda Therapeutics until its rebranding in 2025, is developing an off-the-shelf hematopoietic stem cell (HSC) platform aimed at replacing traditional donor-dependent bone marrow transplantation. Instead of relying on matched donors or cord blood units, the company is engineering self-renewing, HLA-compatible HSCs that can be administered on demand, offering a standardized and potentially more accessible way to restore blood and immune function. 

The company’s technology, now formalized under the Stratus Prime platform, is designed to generate transgene-free HSCs at scale, with HLA profiles selected to match broad swaths of the patient population. In theory, this could eliminate the need for donor searches, streamline the transplant process, and expand access to patients with conditions such as bone marrow failure, β-thalassemia, inherited hematologic diseases, or those requiring immune reconstitution after cancer therapy. 

The rebranding came with a $50 million series A-1 financing in March 2025, adding to the $62 million series B it raised in 2023. As of 2025, Stratus remains preclinical, and reliably producing long-term repopulating, multilineage HSCs outside the human body is a technical challenge. The cells must demonstrate durable engraftment, appropriate immune compatibility, and stable behavior across all blood lineages. Still, the potential reward is substantial. If successful, Stratus’s platform could radically broaden access to stem-cell transplants and redefine how hematologic and immune disorders are treated.  

Stem cell therapeutics, a complete toolbox 

Taken together, the companies in this article show how stem cell therapy has evolved from a regenerative idea into a broader toolbox, from neuronal and cardiac repair to engineered immune cells, universal grafts for blood and immune reconstitution, and immune-evasive islet cells for diabetes.  

The field is also moving from proof-of-concept into pivotal trials and first regulatory decisions. BlueRock Therapeutics has initiated the first phase 3 study of an allogeneic pluripotent stem-cell-derived therapy in Parkinson’s disease. Orca Bio’s engineered graft Orca-T has delivered phase 3 data showing improved survival.