A clinical-stage biotechnology company focused on the discovery and development of engineered T cell therapies for autoimmune diseases

The Final Act for Autoimmune Disease

What if autoimmune diseases could be cured with a one-time treatment? At Cabaletta Bio, we believe it is possible.

On a proven foundation of CAR T technology and an established track record of clinical operational excellence in cell therapy in autoimmunity, we are developing innovative engineered T cell therapies for patients with autoimmune diseases. Our proprietary Cabaletta Approach to B cell Ablation (CABA™) platform is designed to provide deep, durable, and potentially curative responses with one-time administration, with the possibility of eliminating the need for chronic immune suppressive therapies. The CABA™ platform has potential applicability across many autoimmune diseases, including systemic lupus erythematosus, pemphigus vulgaris and myasthenia gravis, and it encompasses two approaches. 

The Chimeric Antigen Receptor T cells for Autoimmunity (CARTA) approach is designed to enable transient eradication of all B cells in patients with autoimmune diseases, with subsequent repopulation of normal B cells, thus resetting the immune system and restoring immune health. Our lead CARTA candidate, CABA-201, is being developed in systemic lupus erythematosus and other autoimmune diseases with high unmet need.

Cabaletta’s proprietary Chimeric AutoAntibody Receptor T (CAART) cell approach is designed for diseases driven by specific autoantibodies, to enable permanent elimination of only disease-causing B cells with preservation of the healthy immune system. Our most advanced CAART candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ trial, which is a Phase 1 study for the treatment of patients with mucosal-dominant pemphigus vulgaris (mPV). The MusCAARTes™ Phase 1 trial is underway for our MuSK-CAART candidate in MuSK-associated myasthenia gravis, and we have advanced several other high-priority targets into discovery and preclinical testing.

Our Mission

To harness the immune system through innovative cell and gene therapies to deliver accessible cures for patients.

Our Vision

To develop and launch the first targeted cellular therapies for patients with autoimmune diseases. When we succeed, we will be:

  • Champions to patients
  • Valued partners to physicians
  • Preferred collaborators to scientists and partners
  • Family to each other
  • Value-added providers to payors
  • Trusted, transparent, and reliably providing a fair return to our investors

Our Values

  • Prioritize the patient
  • Listen to the data
  • Serve the team
  • Improve every day
  • Pursue excellence and persevere
  • Be authentic, vulnerable, and courageous
  • Embrace differences, demand equity and inspire trust
  • Do the right thing

Orchestrating the Future of Autoimmune Therapy

We aim to leverage our CABA™ platform to rapidly advance new engineered T cell therapies for patients with autoimmune diseases.

Our strategy is to:

  • Build on a foundation of clinically validated CAR T manufacturing processes with innovative advances to enhance the profile of products for patients.
  • Advance a portfolio of high-potential targets based on our proprietary CABA™ platform.
  • Demonstrate potential for CD19-CAR T to reset the immune system in patients with systemic lupus erythematosus and other autoimmune diseases.
  • Achieve clinical proof-of-concept for DSG3-CAART in patients with mucosal pemphigus vulgaris and MuSK-CAART in patients with MuSK myasthenia gravis.
  • Continue to expand our established intellectual property position.
  • Leverage our leadership position in T cell therapy as well as our robust talent and team-oriented culture to build a fully integrated cell therapy company focused on autoimmune diseases.

Composing New Defenses Against Pathogenic B-Cells

Our engineered T cell therapy products are designed to reprogram a patient’s immune system through one of two mechanisms: 1) Transient elimination of B cells, with repopulation of healthy B cells; or 2) Specific permanent killing of only autoantigen-specific B cells that cause disease with preservation of normal B cells. We aim to eliminate the cause of autoimmune disease while leaving the healthy immune system intact.

How It Works

Robust Clinical-Stage Cell Therapy Pipeline

Our CABA™ platform has potential applicability across a broad range of autoimmune diseases. Our CABA-201 candidate is being developed for systemic lupus erythematosus as well as other autoimmune diseases with high unmet needs. Our announced CAART pipeline includes candidates that target pemphigus vulgaris (both mucosal-dominant and mucocutaneous subtypes), MuSK myasthenia gravis and PLA2R-associated membranous nephropathy, with our DSG3-CAART and MuSK-CAART candidates currently under evaluation in the DesCAARTes™ and MusCAARTes Phase 1 trials, respectively.

Our Candidates

Our Story

Since Cabaletta Bio was founded in 2017, we have been working toward a singular goal: Developing and launching the first curative targeted cellular therapies for patients with autoimmune diseases.

Over the past five years, our robust track record of execution has yielded two clinical-stage programs that are elegantly designed to target only the cells responsible for disease. We are encouraged by the safety profile demonstrated by our DSG3-CAART in the DesCAARTes™ Phase 1 trial to date, and based on emerging translational data, we are employing combination strategies to increase DSG3-CAART cell activity. Our learnings have accelerated the potential to observe signs of clinical efficacy with our next CAART candidate, MuSK-CAART for patients with MuSK-associated myasthenia gravis, which is currently being evaluated in the Phase 1 MusCAARTes™ trial. Our robust clinical execution is demonstrated through our positive regulatory interactions since 2018, with three IND applications cleared within the routine 30-day period, implementation of multiple clinical trial protocol modifications, two Fast Track Designations and two Orphan Drug Designations granted to pipeline candidates, as well as our successful manufacturing of novel cell therapy products with academic and industry partners. 

As we have established our position in the field of autoimmunity, we continue to evaluate novel approaches that have the potential to cure autoimmune diseases. We were excited to see the breakthrough results generated by Professor Georg Schett, M.D. and his colleagues at Friedrich-Alexander-Universität Erlangen-Nürnberg, published in Nature Medicine in September 2022, showing the potential for CD19-CAR T to transform the course of systemic lupus erythematosus (SLE). In five patients with SLE, one-time treatment with a 4-1BB-containing CD19-CAR T cell therapy induced deep and durable clinical responses in all five patients within three months after treatment, with favorable tolerability. Healthy B cells repopulated in all patients within five months of treatment, and responses remained durable with up to 17 months of follow-up. Similar results were seen in a case report on a patient with anti-synthetase syndrome, a subtype of myositis, as published in The Lancet in February 2023. These findings demonstrate the potential for CD19-CAR T to “reset the immune system,” eliminating the cause of autoimmune disease with restoration of the healthy immune system.

Building on these results, we have initiated development of CABA-201, a 4-1BB-containing CD19-CAR T therapy, for the treatment of systemic lupus erythematosus and other severe autoimmune diseases. We are employing a fully human CD19 binder that has high similarity to the construct employed in the academic study, and through an exclusive translational research partnership with Professor Schett, we will employ our robust translational engine to generate early and actionable insights from his trials that may inform our clinical development. 

Accelerated by our team’s deep expertise in cell therapy, robust clinical experience in autoimmunity, and demonstrated track record of strong execution, we are uniquely positioned to employ CD19-targeting strategies in order to further our mission to develop therapies that deliver deep, durable and potentially curative responses for patients with autoimmune diseases.

Meet Our Conductors

Cabaletta Bio was founded by leading experts in autoimmune diseases and CAR T technology. Today, we are led by a team of physicians, scientists, and pharmaceutical industry veterans broadly experienced in developing and manufacturing novel cell therapies and bringing them to market.

Our Leaders

Our Directors

Scientific Advisory Board