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About

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, we are developing innovative and highly specific engineered T cell therapies for patients with B cell-mediated autoimmune diseases, designed to provide deep, durable, and potentially curative responses. Our proprietary Cabaletta Approach to selective B cell Ablation (CABA™) platform is focused on eliminating pathogenic B cells that express autoantibodies, which cause certain autoimmune diseases, while sparing the normal B cells critical to human health. Our CAAR T cell products are designed to target only those abnormal B cells responsible for autoimmune disease – typically fewer than 1% of all B cells – while sparing healthy B cells and preserving normal immunity. We have identified over two dozen B cell-mediated autoimmune diseases where we believe our CABA™ platform may provide a biological opportunity for cure. Our lead candidate, DSG3-CAART, is being evaluated in the DesCAARTes™ trial, which is a Phase 1 study for the treatment of patients with mucosal pemphigus vulgaris (mPV). We have advanced several other high-priority targets into discovery and preclinical testing, including five announced and two unannounced programs.

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 CAAR T cell therapies for patients with B cell-mediated autoimmune diseases.

Our strategy is to:

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

Composing New Defenses Against Pathogenic B-Cells

Our CAAR T products are designed to reprogram a patient’s T cells to attack pathogenic B cells while preserving beneficial ones, potentially eliminating disease and leaving the healthy immune system intact.

How It Works

Robust Clinical-Stage Cell Therapy Pipeline

Our announced therapeutic candidates include treatments for several B cell-mediated autoimmune diseases, including mPV, MuSK MG, mucocutaneous (mc) PV, PLA2R-associated membranous nephropathy and Hemophilia A with Factor VIII alloantibodies.

Our Candidates

Our Story

A “cabaletta” is a rapid, repetitive and technically challenging concluding section of a two-part operatic aria, designed to highlight the virtuosity of a lead performer. While we are not the first to develop CAR T cell therapies, we are the first to design and develop them to specifically eliminate only certain B cells to treat autoimmune diseases while preserving the normal immune system.

2011

Our scientific advisory board member, Carl June, M.D., publishes the landmark publication “T Cells with Chimeric Antigen Receptors Have Potent Antitumor Effects and Can Establish Memory in Patients with Advanced Leukemia” (Science Translational Medicine, Aug 2011). This prompts our scientific co-founders, Aimee Payne, M.D., Ph.D., and Michael Milone, M.D., Ph.D., to meet and discuss the potential of CAAR T, based on the innovative ideas of Christoph Ellebrecht, M.D.

2013

Our scientific co-founders, Drs. Aimee Payne and Michael Milone begin their partnership at the University of Pennsylvania, combining Dr. Payne’s expertise in B cell-mediated autoimmune diseases with Dr. Milone’s deep and experienced insights into the design and implementation of CAR T products.

2016

Drs. Payne and Milone’s first scientific publication, “Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease” (Science, July 2016), is published.

2017

Steven Nichtberger, M.D., a biotech entrepreneur with nearly two decades of experience in cell therapy, begins conversations with Drs. Payne and Milone in the context of a course he teaches as an adjunct professor at the Wharton School at the University of Pennsylvania. Following a year of interaction and discussions on the optimal strategy to advance the scientific opportunity into a commercially developed product portfolio that could offer potentially curative treatment options to patients, Drs. Payne, Milone and Nichtberger decide to launch Cabaletta Bio.

2019

We received FDA clearance to initiate the first-in-human clinical study of CAAR T technology. The Phase 1 DesCAARTesTM trial is enrolling patients with mucosal pemphigus vulgaris.

2020

The first patient is dosed in the landmark Phase 1 DesCAARTes™ trial for patients with mucosal pemphigus vulgaris. The FDA granted Fast Track Designation for DSG3-CAART for improving healing of blisters in patients with mucosal pemphigus vulgaris. Preclinical in vivo proof of concept data was shown for MuSK-CAART at the AAN Scientific Highlights session in May 2020.

2021

We announced acute safety data from the first cohort of patients in the DesCAARTes™ trial in the first half of 2021. We plan to submit an IND for MuSK-CAART in the second half of 2021.

Meet Our Conductors

Cabaletta Bio was founded by leading experts in B cell-mediated 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