Cell therapy has come a long way in recent years, with many biotech companies now specializing in the development of cell therapies to treat various diseases. In this article we take a look at five of those companies.
Cell therapy refers to the transplantation of human cells in order to replace or repair damaged ones to treat a particular disease. Several different cells can be utilized in cell therapy, including hematopoietic stem cells, skeletal muscle stem cells, neural stem cells, lymphocytes, etc.
Advancements in cell therapy over the last few years are particularly paying dividends in the field of oncology, where, for example, CAR-T cell therapies have been a game changer in the treatment of blood cancer, and are now being explored for the treatment of solid tumors as well.
Here, listed in alphabetical order, are five of the top companies working on the development of cell therapies for cancer and other disease areas.
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Adaptimmune is based in Oxford, in the U.K., and is focused on transforming the lives of people with cancer by making use of patients’ own T cells to target and destroy cancer cells. To do this, the company has a proprietary T-cell receptor (TCR) platform that enables it to identify cancer targets and find and genetically engineer TCRs to recognize and attack the intended targets.
The TCR T-cell therapies that the company is developing are called SPEAR T-cells, which can recognize peptide fragments from both proteins present inside the cell and expressed on the outside of the cell. This allows for a larger number of proteins to be targeted.
Adaptimmune’s clinical pipeline includes candidates being investigated for the treatment of synovial sarcoma, head and neck cancer, esophageal cancer, ovarian cancer, and more. Afamitresgene autocel is the company’s first-generation TCR T-cell therapy targeting MAGE-A4 – an antigen expressed in several solid tumor types, plus the company is also developing next-generation cell therapies, with its first clinical candidate being ADP-A2M4CD8, which is also directed to MAGE-A4.
It was recently announced that Adaptimmune and TCR2 Therapeutics will combine in an all-stock transaction to create a preeminent cell therapy company focused on treating solid tumors.
Gamma delta T cells is Adicet Bio’s focus area, as it hopes to develop allogeneic off-the-shelf genetically modified gamma delta T cells that selectively target tumors. Based in Boston, the company believes that gamma delta T cells have unique attributes that make them particularly well-suited for cancer therapy, with features that combine adaptive – gamma delta TCR-mediated – and innate – NK-cell like – immunity to specifically recognize and eliminate tumor cells without harming normal, healthy cells.
Adicet’s therapies – intended for various hematological and solid tumor cancers, as well as other disease – involve engineering gamma delta T cells with chimeric antigen receptors (CARs) and T cell receptors (TCRs) directed to tumor-specific cell surface or intracellular targets. This can enable precise engagement and killing of tumor cells.
The company currently has seven wholly-owned programs in its pipeline, with one in clinical development, which is its lead candidate, ADI-001, for the treatment of B-cell non-Hodgkin lymphoma (NHL).
Fate Therapeutics is a clinical-stage biopharmaceutical company focused on immunotherapy, bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune disorders. The company uses its proprietary iPSC product platform to generate human iPSCs, which are in turn used to create genetically engineered, clonal master iPSC lines to produce well-defined and uniformly composed NK- and T-cell products.
Fate Therapeutics has a pipeline comprised of immuno-oncology programs that target a broad range of both liquid and solid tumors. It currently has two programs in clinical development for multiple myeloma and B-cell malignancies, as well as two other programs in preclinical development for B-cell lymphoma and solid tumors.
The cell therapy company also has a preclinical candidate for an undisclosed autoimmune disorder, using iPSC-derived NK cells.
U.K.-based company Rinri Therapeutics is working in the niche area of developing regenerative cell therapies for hearing loss, in the hope of reversing hearing loss by replacing the dead or damaged specialized sensory cells of the inner ear that cause sensorineural hearing loss. Sensorineural hearing loss makes up an estimated 90% of all hearing loss in humans and is caused by damage to the sensory cells in the cochlea.
The company’s lead candidate is Rincell-1, which is a novel progenitor cell product derived from stem cells that can differentiate into mature auditory neuron cells. Once delivered to the cochlea, they differentiate into auditory neurons, sending out neurites to connect to the auditory hair cells and back towards the brain stem, eventually reconnecting the circuitry within the inner ear. The therapy has demonstrated long-term safety and efficacy in pre-clinical models, indicating potential levels of functional restoration of hearing that would be clinically significant.
Rinri has raised £18.3 million ($22.5 million) from venture capital funds and non-dilutive funding and, having successfully progressed Ricell-1 through preclinical development, it is now hoping to advance the product towards first-in-human trials.
Umoja Biopharma is focused on pioneering the future of immunotherapy for solid tumors by reprogramming T cells in vivo, rather than ex vivo, using CAR-T cell therapy. Because the company’s platform, VivoVec, is designed to manufacture CAR-T cell therapy inside of the patient’s own body, it should be able to deliver cell therapy at a lower cost and faster speed compared to engineering T cells ex vivo.
Umoja’s UB-VV200 solid tumor program combines three different technologies to attack tumors: the VivoVec delivery system, which enables CAR-T cells to be engineered directly in the patient’s body; a receptor known as RACR, which is added to the CAR-T cells that promotes T-cell persistence and cell survival; and TumorTags, which allow the company’s CAR-T cell therapy to recognize different parts of the tumor microenvironment for more precise targeting.
As well as the UB-VV200 program, the company currently has two other therapeutic programs in preclinical development for hematologic malignancies and gynecologic tumors.