Center of Excellence for Cell Therapy in Autoimmunity (CECTA)

Advancing engineered CAR and CAAR T cell therapies across multiple autoimmune diseases through clinical trials, preclinical development, and industry partnerships.
Ongoing Dermatologic Diseases Other Pemphigus & Pemphigoid Systemic Diseases Systemic Lupus Erythematosus (SLE) Systemic Sclerosis (Scleroderma) Vasculitis Academia–Industry Partnerships Animal Models Autoantibodies B Cell Biology Biological & Mechanistic Clinical Trials Collaboration & Innovation Experimental Platforms & Models In Vitro Models Precision Medicine T Cell Biology Therapeutic Development Translational & Clinical

Funding Year: 2024

Project Overview

The Colton Center of Excellence in Cellular Therapies for Autoimmunity (CECTA) supports the development and clinical translation of innovative cell therapies for autoimmune diseases, combining active clinical trials with laboratory-based discovery science. Active and completed trials span pemphigus vulgaris, lupus nephritis, systemic sclerosis, and ANCA-associated vasculitis, using anti-CD19 CAR T cells and disease-specific CAAR T approaches. Preclinical work has developed CAR and CAAR T cells targeting VH1-69 B cell receptors in immune thrombotic thrombocytopenic purpura, with demonstrated in vitro and in vivo efficacy, a new iTTP mouse model, and an IgG4-targeted CAR in development for IgG4-related disease. CAP T cells targeting autoimmune T cells are also in development, with commercialization discussions underway.

Impact & Innovation

Building the clinical and scientific infrastructure for cell therapy in autoimmunity.

 

CECTA is actively running and opening trials across pemphigus vulgaris, lupus nephritis, systemic sclerosis, and AAV — while simultaneously advancing next-generation CAR, CAAR, and CAP T cell platforms with broad applicability across autoimmune diseases.

  • Demonstrates clinical proof-of-concept for anti-CD19 CAR T cell therapy across multiple autoimmune diseases, with early responses observed in lupus nephritis and systemic sclerosis patients
  • Generates strong IP through three patent submissions — including VH1-69 CAR for iTTP, ADAMTS13 CAAR cells, and compositions for celiac disease — with commercialization discussions underway for CAP T cell technology
  • Advances the Consortium’s From Mechanistic Insight to Translation pillar by operating as a cross-disease clinical and preclinical cell therapy platform, with active industry partnerships with Cabaletta Bio, Kyverna, and Novartis
Research Approach

A framework designed for discovery

CECTA combines active clinical trial execution with laboratory-based CAR, CAAR, and CAP T cell development to advance cell therapies across multiple autoimmune diseases. The work integrates clinical mechanistic insights from treated patients with preclinical platform development and industry partnerships to accelerate translation.

Clinical trials of anti-CD19 CAR T cells (KYV-101) in lupus nephritis and systemic sclerosis; Cabaletta Bio-sponsored DSG3-CAART and CABA-201 trials in pemphigus vulgaris; Novartis-sponsored anti-CD19 CAR T trial in AAV; preclinical development of VH1-69-targeting CAR and CAAR T cells and a new iTTP mouse model; IgG4-targeted CAR development for IgG4-related disease; and CAP T cell development targeting autoimmune T cells.

Clinical trial data from patients with pemphigus vulgaris, lupus nephritis, systemic sclerosis, and AAV; in vitro and in vivo efficacy and safety datasets for VH1-69 CAR/CAAR T cells; new iTTP mouse model datasets recapitulating clinical disease features; and IgG4-targeted CAR in vitro functional testing datasets.

Advancement of multiple cell therapy platforms from preclinical validation through clinical trial execution across autoimmune diseases, with translational goals including regulatory approval pathways, industry co-development partnerships, and commercialization of novel CAP T cell technology. Three patent applications have been submitted, and NHLBI R01 HL178849-01 ($500,000/year, 5 years) is pending review.

Investigators & Institutions

Powering the science

Principal Investigators

David Porter, MD, Colton Consortium Member

Jodi Fisher Horowitz Professor in Leukemia Care Excellence, Department of Medicine, Perelman School of Medicine, University of Pennsylvania

James L. Riley, PhD, Colton Consortium Member

Professor, Department of Microbiology, Perelman School of Medicine, University of Pennsylvania

Colton Centers & Institutions

Colton Center for Autoimmunity at the University of Pennsylvania

Perelman School of Medicine

Research Outputs

From insight to impact

Industry partnerships:

  • Cabaletta Bio (pemphigus vulgaris trials)
  • Kyverna (KYV-101 lupus nephritis and CARTIMMUNE trials)
  • Novartis (anti-CD19 CAR T in AAV, lead investigator Dr. Merkel).

Commercialization discussions:

  • Penn Center for Innovation arranging meetings with Curie Bio and VC firms for CAP T cell technology.

Manuscripts in preparation: CAR/CAARs for iTTP; CAPs.

  • Compositions and Methods for Celiac Disease
  • Compositions and Methods of VH1-69 Chimeric Antigen Receptor for Immune Thrombotic Thrombocytopenic Purpura.
  • Pending patent: Compositions and Methods of ADAMTS13 Chimeric Autoantibody Receptor Cells.