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The Cellution to Autoimmunity? The promise and perils of CAR-T.

Autoimmune diseases (AID) encompass a group of more than 80 distinct diseases affecting up to 50 million individuals in the U.S. alone, all of which are characterized by an abnormal immune response against self, usually involving autoreactive B and T cells. These diseases range in severity from minor nuisance (e.g., mild Sjogren’s Syndrome) to life-threatening (e.g., severe lupus nephritis), and their management collectively costs the U.S. healthcare system more than $100B each year. Management typically involves the use of broad-acting immunosuppressive drugs which can be highly effective at controlling symptoms and preventing disease progression in some patients. However, available treatments are not curative, don’t work for all patients, require lifelong treatment, and are often associated with significant side effects due to chronic immunosuppression. Accordingly, there is a great need for new treatments that are safer and more effective.

For drug developers in the autoimmune space, the holy grail has long-been to restore immune tolerance without the need for broad-acting immunosuppressive drugs.

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