The biotechnology company Cabaletta Bio has recently announced that the company investigational new drug (IND) application for its experimental CD19-CAR T-cell therapy CABA-201 has been cleared by the US Food and Drug Administration (FDA). This application is in regard to a phase I/II trial which will evaluate the drug as a treatment for generalized myasthenia gravis, a rare disease. IND applications for this therapy have previously been cleared for other diseases as well, such as systemic sclerosis, myositis, and lupus.
The small scale, early-stage trial will include two cohorts of six patients each, with one being patients that test positive for the acetylcholine (AChR) receptor and one being patients that do not. These patients will receive a one-time does of CABA-201 in the form of an infusion. Prior to this infusion, a preconditioning process, including administration of cyclophosphamide and fludarabine, will be administered.
Cabaletta Bio is focused on the development of targeted cell therapies in order to treat or potentially cure autoimmune disorders.
About Myasthenia Gravis
Myasthenia gravis is a long-term autoimmune disease that is most characterized by weakness of the skeletal muscles which can affect a patient’s ability to move and breathe. As an autoimmune disease, myasthenia gravis is ultimately the result of abnormal immune system behavior in which the system attacks normal body tissue. In this disease, the immune system produces antibodies that inhibit regular neuromuscular function. Symptoms of the illness include muscle weakness that worsens after activity and gets better after resting. This weakness may affect the face, eyes, neck, breathing, and limbs. It may cause speech and swallowing difficulties, eyelid drooping, shortness of breath, and blurred vision. In severe cases, patients may require breathing assistance with a ventilator. Treatment options for myasthenia gravis include immunosuppressive medications, thymus gland removal, IVIG, and plasmapheresis. Most patients can effectively manage the condition with treatment. To learn more about myasthenia gravis, click here.
