# Autologous T-cells Expressing a Chimeric Antigen Receptor Directed to B-Cell Maturation Antigen (BCMA) in patients with Generalized Myasthenia Gravis (MG)

> **NIH NIH R44** · CARTESIAN THERAPEUTICS, INC. · 2020 · $835,858

## Abstract

Project Summary/ Abstract
Myasthenia Gravis (MG), which affects 60,000 people in the U.S., is an autoimmune disease caused by
autoantibodies that attack the neuromuscular junction. For many MG patients, there is no safe, selective, and
effective therapy, and MG remains a chronic, debilitating, and potentially fatal disease. Today, despite knowledge
that the disease is mediated by autoantibodies produced by aberrant plasma cells (PCs), treatment of
generalized MG still relies on indiscriminate immunosuppression with systemic steroids or steroid-sparing
immunomodulators. Targeting a PC-restricted antigen would eliminate aberrant autoantibody-producing plasma
cell clones, reduce pathogenic autoantibody, and could improve disease symptoms. One such target antigen is
B-Cell Maturation Antigen (BCMA), which is expressed on all healthy PCs and on malignant PCs (i.e., multiple
myeloma (MM)). Clinical trials with autologous anti-BCMA Chimeric Antigen Receptor (CAR) T-cells
permanently modified by gene transfer show unprecedented efficacy in relapsed/refractory MM, but permanent
genetic modification of T-cells leads to uncontrolled proliferation and unpredictable pharmacokinetics in vivo.
The resulting toxicity can be severe and lethal. In order to preserve CAR T-cell efficacy but significantly reduce
toxicity, Cartesian Therapeutics pioneered development of anti-BCMA CAR T-cells (named as Descartes-08)
with defined, controllable pharmacokinetics by transfecting T-cells with CAR mRNA instead of by gene transfer.
This project is intended to determine the feasibility of treating generalized MG with mRNA-transfected CAR T-
cells. The hypothesis to be tested is that dosing Descartes-08 at or below the maximal tolerated dose (MTD)
will achieve a high rate of clinical remission in patients with severe generalized MG.

## Key facts

- **NIH application ID:** 10010086
- **Project number:** 1R44NS115426-01A1
- **Recipient organization:** CARTESIAN THERAPEUTICS, INC.
- **Principal Investigator:** METIN KURTOGLU
- **Activity code:** R44 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $835,858
- **Award type:** 1
- **Project period:** 2020-04-15 → 2022-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10010086

## Citation

> US National Institutes of Health, RePORTER application 10010086, Autologous T-cells Expressing a Chimeric Antigen Receptor Directed to B-Cell Maturation Antigen (BCMA) in patients with Generalized Myasthenia Gravis (MG) (1R44NS115426-01A1). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10010086. Licensed CC0.

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