# University of Pennsylvania Clinical Autoimmunity Center of Excellence

> **NIH NIH UM1** · UNIVERSITY OF PENNSYLVANIA · 2022 · $78,890

## Abstract

The overall goals of the University of Pennsylvania Clinical Autoimmunity Center of Excellence (Penn
ACE) are 1) to perform cutting-edge clinical and translational research to advance our understanding of human
autoimmunity through Clinical and Collaborative Projects, 2) to encourage the next generation of physician-
scientists to pursue clinical and translational research in autoimmunity through Scientific Communications and
Mentoring and Enrichment Programs overseen by an Administrative Core, and 3) to provide the financial and
regulatory guidance and infrastructure to support collaborative scientific interactions within and between
academic institutions nationwide through an ACE Funds Management Core.
 The clinical and translational research program of the Penn ACE centers around the theme of “B cells
as drivers of autoimmunity”, focusing on three debilitating and potentially life-threatening autoimmune diseases
(pemphigus vulgaris (PV), multiple sclerosis (MS), and type 1 diabetes (T1D)) for which prospective
randomized clinical trials of anti-CD20-mediated B cell depletion have demonstrated significant clinical benefit.
Thus, B cells are key drivers of autoimmunity in these conditions, but risk of serious and potentially fatal
infections compromises the utility of chronic B cell depletion as a long-term treatment strategy. Each of the
proposed Clinical and Collaborative Projects, as well as the Administrative Core, addresses a key question or
barrier to progress in the field. The Primary Clinical Project will execute a phase 1b open-label dose escalation
study to evaluate the safety, tolerability, and potential efficacy of a novel gene-engineered cellular
immunotherapy known as DSG3-CAART to achieve targeted, antigen-specific B cell depletion and lasting
disease remission in PV. The Alternate Clinical Project will utilize a prospective randomized trial of ocrelizumab
withdrawal in MS to evaluate whether B cell depletion can be safely withdrawn in MS patients without
compromising efficacy and will identify potential biomarkers that predict long-term tolerance induction. A
Collaborative Project will perform deep phenotyping, immune repertoire analysis, and target antigen discovery
for expanded B cell clones in the pancreas, secondary lymphoid organs, and blood of patients with T1D, which
may not only identify novel therapeutic strategies for T1D, but also establish a framework for how similar
experimental approaches can be used to elucidate the pathophysiology and targets of B cells in a broad range
of other autoimmune diseases. Finally, an Administrative Core will implement a scientific communications and
mentoring program to foster collaborative research within and beyond the Penn ACE and encourage future
generations of physician-scientists to pursue careers in autoimmune disease clinical care and research.

## Key facts

- **NIH application ID:** 10386830
- **Project number:** 5UM1AI144288-04
- **Recipient organization:** UNIVERSITY OF PENNSYLVANIA
- **Principal Investigator:** AMIT BAR-OR
- **Activity code:** UM1 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2022
- **Award amount:** $78,890
- **Award type:** 5
- **Project period:** 2019-05-01 → 2024-04-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10386830

## Citation

> US National Institutes of Health, RePORTER application 10386830, University of Pennsylvania Clinical Autoimmunity Center of Excellence (5UM1AI144288-04). Retrieved via AI Analytics 2026-05-22 from https://api.ai-analytics.org/grant/nih/10386830. Licensed CC0.

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