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 unique ability to differentiate into various cell types, hold great potential in regenerative medicine.
One of the most notable advancements in stem cell therapy is 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.
In this article, we delve into stem-cell-oriented biotech companies to know about.
Table of contents
Aspen Neuroscience
Aspen Neuroscience was founded in 2018, and is headquartered in San Diego. It is a clinical-stage company focused on developing personalized stem cell therapies to treat neurodegenerative diseases, with an initial emphasis on Parkinson’s disease.
Aspen Neuroscience’s lead candidate, ANPD001, is an autologous induced pluripotent stem cell (iPSC)-derived neuron replacement therapy aimed at treating Parkinson’s disease. This therapy is designed to replace the lost dopamine neurons that are characteristic of the disease, potentially reversing its effects.
Aspen’s technology lies in the use of autologous iPSCs. The process begins with taking a small sample of a patient’s skin cells, which are then reprogrammed into iPSCs. These iPSCs have the ability to differentiate into any cell type. For Parkinson’s therapy, the iPSCs are differentiated into dopamine-producing neurons. This autologous approach ensures that the cells are genetically identical to the patient’s own cells, minimizing the risk of immune rejection.
Aspen leverages bioinformatics, machine learning, and genomic testing to ensure the quality and safety of the reprogrammed cells. Its proprietary artificial intelligence (AI)-based genomics tests are used to evaluate the quality of the cells at every stage of the manufacturing process. As of 2024, the company has raised a total of $272 million across multiple funding rounds.
In 2023, the stem cell therapy company received the U.S. Food and Drug Administration (FDA) clearance for its investigational new drug (IND) application for ANPD001, allowing the initiation of a phase 1/2a clinical trial for which the company dosed its first patient in April.
BlueRock Therapeutics
BlueRock Therapeutics, founded in 2016 and headquartered in Cambridge, develops regenerative medicines using iPSC technology. BlueRock is a wholly-owned subsidiary of Bayer and works on stem cell therapies aimed at treating neurological, cardiovascular, and immunological diseases.
BlueRock utilizes iPSC technology to generate specialized cell types and focuses on the replacement and repair of damaged cells. For Parkinson’s disease, this involves implanting neurons derived from iPSCs to replace those lost due to the disease.
BlueRock’s lead program, bemdaneprocel (BRT-DA01), is an investigational cell therapy designed to treat Parkinson’s disease. This therapy uses dopamine-producing neurons derived from human pluripotent stem cells, which are surgically implanted into the patient’s brain. These cells have the potential to re-establish neural networks damaged by Parkinson’s, aiming to restore motor function and improve the quality of life for patients.
ThepPhase 1 clinical trial showed that the bemdaneprocel therapy was well-tolerated, with no major safety issues, and demonstrated the feasibility of transplantation and cell survival in the brain. These promising results support the initiation of phase 2 trials expected to begin enrolling patients in the first half of 2024.
Cellino
Cellino, founded in 2017, is also based in Cambridge. The company focuses on developing a scalable, automated biomanufacturing system for personalized cell therapies.
Cellino’s primary focus is on creating personalized regenerative medicines using iPSCs. The company aims to treat a range of diseases, including Parkinson’s disease, diabetes, and heart disease, by producing patient-specific cell therapies.
Cellino combines label-free imaging, high-speed laser editing, and machine learning to automate the reprogramming, expansion, and differentiation of cells. This platform allows for the parallel processing of thousands of patient samples in a single facility. By leveraging these technologies, Cellino aims to overcome the challenges of traditional autologous cell therapy processes, which are often labor-intensive, variable, and expensive.
- Label-free imaging: This technique helps identify and monitor cells without the need for fluorescent or chemical labels, ensuring that the cells remain unaltered.
- High-speed laser editing: Precise laser technology is used to edit and reprogram cells at the single-cell level.
- Machine learning: AI algorithms are employed to optimize and control the cell manufacturing process, ensuring high consistency and quality across batches.
These technologies collectively could enable the efficient and scalable production of iPSC-derived cell therapies, making personalized medicine more accessible and feasible on a larger scale. In 2022, Cellino raised $80 million in a series A financing round led by Leaps by Bayer.
Fate Therapeutics
Founded in 2007 and headquartered in San Diego, California, Fate Therapeutics is a clinical-stage company developing programmed cellular immunotherapies for cancer and immune disorders. The company leverages its proprietary iPSC technology to create off-the-shelf stem cell therapies designed to be universally available and immediately accessible, eliminating the need for patient-specific customization.
Fate Therapeutics creates clonal master iPSC lines, which serve as a standardized starting material for manufacturing uniform cell products, facilitating scalable manufacturing.
The stem cell company’s pipeline includes several product candidates that utilize iPSC technology:
- FT522: An iPSC-derived chimeric antigen receptor (CAR) natural killer (NK) cell product in phase 1 clinical trials, designed to treat B-cell lymphomas and other autoimmune disorders.
- FT819: The company’s first off-the-shelf CAR T-cell product, designed for the treatment of B-cell malignancies and other hematologic cancers. FT819 is in phase 1 clinical trials for relapsed/refractory B-cell malignancies and systemic lupus erythematosus (SLE).
In 2024, Fate Therapeutics presented new clinical data from their ongoing trials of FT819, demonstrating its potential in treating both hematologic malignancies and autoimmune disorders.
Garuda Therapeutics
The Cambridge-based company founded in 2021, focuses on developing blood stem cell therapies. Garuda’s platform technology focuses on generating off-the-shelf, self-renewing blood stem cells. This approach addresses several challenges associated with traditional stem cell therapies, such as the need for compatible donors and the high costs and time associated with finding matches.
Garuda Therapeutics’ pipeline includes therapies for hematologic malignancies, sickle cell disease, β-thalassemia, and bone marrow failure diseases. β-Thalassemia is a hereditary blood disorder characterized by the reduced or absent production of hemoglobin, the protein in red blood cells (RBCs) responsible for carrying oxygen throughout the body.
Their approach involves using hematopoietic stem cells (HSCs) to provide consistent, durable and human-leukocyte-antigen(HLA)-compatible stem cell therapies without the need for donor matching. HSCs are a type of multipotent stem cell that gives rise to all the blood cell types, including red blood cells, white blood cells, and platelets. These cells are vital for the maintenance and regeneration of the blood system throughout a person’s life.
HLAs play a critical role in the immune system’s ability to distinguish between self and non-self. These antigens are the major histocompatibility complex (MHC) in humans and are crucial in organ and tissue transplantation because they are responsible for the immune system’s response to foreign cells. By engineering stem cells to be HLA-compatible, Garuda ensures that its therapies can be used across a broad patient population. This eliminates the dependency on donor or patient cells, making the treatments more accessible and reducing the risk of immune rejection.
In 2023, Garuda Therapeutics raised $62 million in a series B funding round, bringing its total funding to $134 million.
Mesoblast
Founded in 2004 and headquartered in Melbourne, Mesoblast is developing allogeneic cellular medicines. Allogeneic cell therapies involve the transplantation of cells from a donor to a genetically distinct recipient. The company specializes in regenerative medicine, utilizing mesenchymal stem cells (MSCs). MSCs are multipotent stromal cells that can differentiate into a variety of cell types, including bone cells, cartilage cells, muscle cells, and fat cells. Mesoblast’s therapeutic focus areas include cardiovascular and spinal orthopedic disorders, oncology, hematology, and immune-mediated and inflammatory diseases.
Mesoblast’s proprietary technology platform is based on mesenchymal lineage cells, which are rare cells that respond to tissue damage by secreting mediators that promote tissue repair and modulate immune responses. These cells are collected from the bone marrow of healthy adult donors and expanded to produce a uniform, highly reproducible cell population. This allows for industrial-scale manufacturing and enables the administration of these cells to patients without the need for donor-recipient matching or immune suppression.
Mesoblast’s product pipeline features several advanced-stage candidates, including:
- Remestemcel-L: In phase 3 trials for steroid-refractory acute graft versus host disease (SR-aGVHD) and moderate to severe acute respiratory distress syndrome (ARDS) due to COVID-19.
- REVASCOR: In phase 3 trials targeting advanced chronic heart failure.
- MPC-06-ID: In phase 3 trials or chronic low back pain due to degenerative disc disease.
- MPC-300-IV: In phase 2 trials investigated for biologic refractory rheumatoid arthritis and diabetic nephropathy.
- MPC-25-IC: In phase 2 for the treatment or prevention of acute myocardial infarction.
Mesoblast went public in 2013 and has secured over $452 million in funding.
Orca Bio
Founded in 2016 and headquartered in Menlo Park, California, Orca Bio is a late-stage biotechnology company specializing in high-precision cell therapies designed to treat cancer, autoimmune diseases, and genetic blood disorders. The company focuses on developing allogeneic cell therapies that aim to safely replace patients’ diseased blood and immune systems with healthy ones
The company’s lead candidate, Orca-T, is an investigational cell therapy that includes infusions of regulatory T cells, CD34+ stem cells, and conventional T cells derived from peripheral blood from donors. CD34+ cells have the capacity to differentiate into various types of blood cells, including red blood cells, white blood cells, and platelets. Orca-T is currently being evaluated in a pivotal phase 3 clinical trial for treating multiple hematologic malignancies. The therapy has shown promising results in clinical trials, offering potentially curative outcomes for patients with blood cancers and reducing the risks associated with conventional bone marrow transplants.
Orca Bio utilizes single-cell precision technologies to create mixtures of immune and stem cells. This process involves sorting blood at the single-cell level to produce cell therapies with a high level of purity and specificity. These therapies are designed to precisely target and eliminate diseased cells while minimizing the risk of complications such as graft-versus-host disease (GvHD). The company’s approach aims to transform allogeneic cell therapy by enhancing the efficacy and safety of stem cell transplants, making them a viable option for a broader patient population.
In 2020, Orca Bio raised $192 million in a series D financing round, bringing its total funding to nearly $300 million. This funding supports the continued development of Orca-T and the company’s other cell therapy programs.
Sana Biotechnology
Sana Biotechnology, founded in 2018, is headquartered in Seattle and focuses on developing engineered pluripotent stem cells as medicines to treat various diseases. Shortly after completing its colossal $700 million series A round, Sana went public in 2021.
Sana employs gene-editing technologies to modify stem cells. These modifications can include correcting genetic defects, enhancing cell function, and ensuring stem cells can effectively integrate and function within the patient’s body.
Sana’s hypoimmune platform involves engineering stem cells to evade the immune system. This modification helps in creating allogeneic cell therapies that can be used universally without the risk of immune rejection. These hypoimmune stem cells can be applied in various therapeutic areas requiring cell replacement therapy.
One of the company’s projects involves using hypoimmune-modified primary islet cells derived from stem cells to treat type 1 diabetes. These cells are engineered to produce insulin, potentially restoring the body’s ability to regulate blood sugar levels.
Another focus for the company is delivering healthy glial progenitor cells (GPCs), derived from stem cells, to treat central nervous system disorders. These therapies aim to repair or replace damaged neural cells and improve patient outcomes in conditions such as multiple sclerosis and other neurodegenerative diseases.
The expanding field of stem cell therapy
The stem cell therapy market was valued at approximately $12.4 billion in 2024, and is projected to reach around $18.5 billion by 2029, growing at a compound annual growth rate (CAGR) of 10.20% during this period. This growth is fueled by increased awareness and demand for regenerative treatments, advancements in stem cell technologies, and supportive regulatory frameworks.
Innovations in stem cell technology, including the development of iPSCs and MSCs, have broadened the scope of therapeutic applications. These advancements enable the creation of off-the-shelf cell therapies that are more accessible and scalable. The increasing incidence of chronic and degenerative diseases, such as cardiovascular diseases, neurodegenerative disorders, and cancer, drives the demand for advanced therapeutic options like stem cell therapy that can repair and regenerate damaged tissues.
While the potential of stem cell therapy is vast, the field still faces several challenges, including high treatment costs, regulatory hurdles, and ethical considerations.