We are utilising Tregs that are engineered with a CAR to treat a variety of inflammatory and autoimmune diseases” What is Sangamo working on with regard to CAR Tregs? Similarly to CAR T-cell therapies used in oncology, you could treat the patient once with these cells and, because they are living, they would not metabolise like a normal drug does instead they are able to proliferate and expand, giving a durable effect, which has the potential – at least theoretically – to be curative. Moreover, because these are living cells, we believe the effect has the potential to be durable and long-lasting. The promise of CAR Tregs is that they are living drugs whose effect, using the CAR technology, we can target to a specific location in the body.įor instance, in multiple sclerosis – a disease where the patient’s own immune system attacks their central nervous system (CNS) – we can localise the Tregs solely to the CNS and prevent that pathology, offering a new and improved way of treating the disease that does not require generalised immunosuppression. These are all diseases that are highly prevalent in the population and are generally treated with drugs that are broadly immunosuppressive while some of these drugs are beneficial and do ameliorate some symptoms, these patients experience side effects including increased susceptibility to infections. Preventing transplant rejection is one example, but other prevalent diseases such as multiple sclerosis, inflammatory bowel disease and rheumatoid arthritis could also benefit. The interest in CAR Tregs is that they could be generally applicable to numerous autoimmune and inflammatory diseases. They can control the activity of other immune cells and are therefore able to limit inflammatory responses, such as autoimmune inflammation.
Jason Fontenot (JF): Tregs are a specialised subset of immune cells that have immunosuppressive properties.
What are Tregs and how are they applicable to immune-mediated disease? In this article, Sangamo Therapeutics’ Chief Scientific Officer, Jason Fontenot, explains why CAR Tregs may become transformational therapeutics for immune-mediated diseases, highlighting the challenges his company has overcome to get the first ever engineered Treg therapy into clinical trials. Recognising the potential of CAR T cells to act as highly targeted therapeutics, several biotechnology companies – including Sangamo Therapeutics – have established development pipelines of CAR T regulatory cells (Tregs) that aim to tackle immune‑mediated diseases, such as autoimmunity and transplant rejection. In recent years there has been significant development within the cell therapy field, as chimeric antigen receptor (CAR) T-cell therapy demonstrated an ability to transform the treatment of patients with haematological malignancies.
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