Cabaletta Bio, Inc. products

DSG3/1-CAART is being developed to treat mucocutaneous pemphigus vulgaris (mcPV), a B cell-mediated autoimmune disease that causes blistering of the skin and mucous membranes. mcPV is caused by autoantibodies that attack the cell adhesion proteins DSG3 and DSG1. DSG3/1-CAART is designed to selectively target and kill the B cells that express the pathogenic autoantibodies while preserving the healthy B cells critical to immune function. We anticipate that much of what we learn from our DSG3-CAART program, including clinical trial design in the DesCAARTesTM trial, will inform and potentially accelerate the development of DSG3/1-CAART.

Our lead product candidate, DSG3-CAART, is being developed to treat mucosal pemphigus vulgaris (mPV), a blistering skin disease that affects mucous membranes. mPV is caused by autoantibodies that attack the cell adhesion protein desmoglein 3, or DSG3. DSG3-CAART is designed to selectively target and kill the B cells that produce DSG3 antibodies while preserving the healthy B cells critical to immune function. In September 2019, the FDA cleared our investigational new drug application for our clinical study of DSG3-CAART in patients with mucosal pemphigus vulgaris. We received Orphan Drug Designation for the treatment of PV from the FDA in January 2020 and Fast Track Designation for improving healing of mucosal blisters in patients with mPV from the FDA in May 2020. The DesCAARTes™ trial is actively recruiting patients at multiple clinical sites across the U.S.

Precision Approach to Address the Underlying Cause of Disease: Our second candidate, MuSK-CAART, is designed to treat myasthenia gravis (MG), an autoimmune disease affecting the neuromuscular junction that can lead to motor impairment, muscle weakness, and respiratory failure.

In addition to our work on autoimmune disease treatments, we are investigating the viability of CAART as an adjunct in cases where the immune system produces antibodies against current therapies (rather than against the patient’s cells). We believe our approach has the potential to destroy those antibodies by ablating B cells, similar to the method we have used to combat autoimmune disease. FVIII-CAART is a discovery-stage candidate for Hemophilia A patients who have an anti-Factor VIII immune response. FVIII-CAART is designed to destroy the antibodies and B cells that neutralize the FVIII clotting factor – potentially restoring the effectiveness of FVIII infusions.