“Myositis, believed to be driven by B cells, is a severe and potentially fatal autoimmune disease for which no curative therapy exists. Current treatment options provide modest efficacy, with a significant portion of diagnosed patients having an inadequate response to treatment, thus, there is a clear need for innovative medicines that can meaningfully change the treatment paradigm," said David J. Chang, M.D., Chief Medical Officer of Cabaletta. “CABA-201 is designed to deeply and transiently deplete CD19-positive B cells, which may enable an immune system reset, and has the potential to deliver durable remission off therapy in patients diagnosed with myositis and other autoimmune diseases where B cells play a role. Orphan Drug Designation is an important recognition for investigational therapies for rare diseases and provides us with potentially valuable benefits as we seek to make a difference in the lives of patients and develop the first targeted, and potentially curative, cell therapy for patients with autoimmune diseases.”

The FDA grants Orphan Drug Designation to drugs or biologics intended to treat or prevent rare diseases or conditions that affect fewer than 200,000 individuals in the United States. This designation qualifies Cabaletta for certain incentives, which may include partial tax credit for clinical trial expenditures, waived user fees and potential eligibility for seven years of marketing exclusivity.