Our RESETTM clinical trials with CABA-201 (investigational CD19-CAR T cell therapy) in lupus, myositis, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris are now recruiting.

About

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 since 2018, 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 across many autoimmune diseases, and it encompasses two approaches.

The Chimeric Antigen Receptor T cells for Autoimmunity (CARTA) approach is designed to transiently, but fully, eliminate B cells, thus potentially enabling an “immune system reset.” Our lead CARTA candidate, CABA-201, is a fully human CD19-CAR T cell product candidate being evaluated in the RESETTM Phase 1/2 trials in autoimmune diseases with high unmet need, including in systemic lupus erythematosus, myositis, systemic sclerosis, and generalized myasthenia gravis.

Cabaletta’s initial Chimeric AutoAntibody Receptor T (CAART) cell approach is designed to eliminate only disease-causing B cells. The DesCAARTes™ and MusCAARTes™ trials are underway in patients with mucosal-dominant pemphigus vulgaris and MuSK myasthenia gravis, respectively.

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 therapeutic candidates based on our proprietary CABA® platform.
  • Demonstrate potential for CD19-CAR T to reset the immune system in patients with systemic lupus erythematosus, myositis, systemic sclerosis, generalized myasthenia gravis 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 by continuing to attract and motivate world class talent within our team-first culture to build the leading fully integrated cell therapy company focused on autoimmune diseases.

Composing New Defenses Against Autoimmune Disease

Our engineered T cell therapy products are designed to reset 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 elimination of only disease-causing B cells. We aim to eliminate the cause of autoimmune disease while maintaining the healthy immune system.

How It Works

Robust Clinical-Stage Cell Therapy Pipeline

Our CABA® platform has potential applicability across a broad range of autoimmune diseases. Our lead candidate, CABA-201, is advancing into the RESETTM (REstoring SElf-Tolerance) Phase 1/2 clinical trials for systemic lupus erythematosus, myositis, systemic sclerosis, and generalized myasthenia gravis, with the potential for application in other autoimmune diseases with high unmet need. Our clinical-stage CAART pipeline includes candidates that target mucosal pemphigus vulgaris and MuSK myasthenia gravis.

Our Candidates

Our Story

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

Since then, we have been focused on advancing clinical studies of novel cell therapy programs in autoimmune disease. Our initial CAART platform has yielded two clinical-stage programs in pemphigus vulgaris and MuSK myasthenia gravis, honing our capabilities to implement studies requiring collaboration between autoimmune treating physicians and cell therapy specialists. As we advanced these elegant product candidates, we were excited to see emerging data that suggested the potential of CD19-CAR T cell therapy approaches to reset the immune system in patients with autoimmune disease. Informed by our expertise in autoimmunity, we conducted a global search to identify the optimally designed product candidate for patients with autoimmune diseases to potentially replicate the promising efficacy and safety observed in these academic reports. This led us to develop CABA-201, a fully human CD19-CAR T with a 4-1BB co-stimulatory domain, which is ideally suited for application in autoimmune disease.

Since announcing CABA-201, we have had four INDs cleared for Phase 1/2 clinical trials in systemic lupus erythematosus, myositis, systemic sclerosis, and generalized myasthenia gravis. As we aim to advance our rapid and broad development program with CABA-201, our progress is accelerated by our team’s deep expertise in cell therapy, robust clinical experience in autoimmunity, and demonstrated track record of strong execution. We remain focused on furthering 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