A More Targeted CAR-T Cell-based Therapy for Myasthenia Gravis Moves into Clinical Trial with University of Florida

A More Targeted CAR-T Cell-based Therapy for Myasthenia Gravis Moves into Clinical Trial with University of Florida. TrialsiteN

Emerging biopharmaceutical cell and gene therapy venture Cartesian Therapeutics announced that it has initiated a Phase I/2 clinical trial targeting its lead CAR-T candidate, Descartes-08, in patients with generalized myasthenia gravis (GMG).

Generalized Myasthenia Gravis (GMG)

A chronic and rare disease impacting almost 200,000 patients in the U.S., EU and Japan, this autoimmune neuromuscular condition causes weakness to different parts of the body—those with ocular myasthenia often experience weakness in only the eye muscles while those with generalized myasthenia gravis have a more severe form of the disease and may experience pervasive weakness in other muscle groups.

The first Car-T investigational candidate for autoimmune disease in clinicals?

The study targets 18 patients in a Phase I/2 study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis. The company announced in its press release that to its knowledge it is the first CAR-T investigational candidate to enter clinical development for an autoimmune disease.

Clinical Investigational Site

The University of Miami Miller School of Medicine is conducting the Phase I/2 study led by principal investigator Volkan Granit, MD—he is also a professor of neurology. Dr. Granit noted, “Patients with severe GMG have limited options and are often dependent on nonselective chronic immunosuppressive therapies (ISTs) that have long-term toxicities.” He continued, “Cartesian’s CAR-T technology selectively targets the primary culprit in the disease: antibody-producing plasma cells. Such selective targeting would be first in GMG and could help patients discontinue use of chronic ISTs.” This approach represents a more targeted therapy in treating myasthenia gravis.

What is Descartes-08

Descartes-08 is a CD8+ CAR-T investigational therapy that targets cells expressing B-cell Maturation Antigen (BCMA), a protein expressed by all plasma cells. This investigational therapy is designed and engineered to “have a defined and predictable half-life, enabling repeat dosing to maximize potency while minimizing risk of toxicity,” reports Metin Kurtoglu, MD, PhD, Chief Medical Officer of Cartesian.

Who is Cartesian Therapeutics?

Founded in 2016, Cartesian is a fully-integrated, clinical-stage biopharmaceutical company developing potent yet safer cell and gene therapies designed to benefit the broadest range of patients with cancer and autoimmune diseases. Cartesian has three programs in clinical development, each under open INDs with the U.S. FDA. The lead investigational therapy, Descartes-08, is a CD8+ CAR T-cell therapy with a defined and predictable half-life, enabling repeat dosing to maximize potential for potency while minimizing risk of toxicity. Descartes-08 is currently in Phase I/II clinical trials to treat patients with multiple myeloma and generalized myasthenia gravis in an outpatient setting. All investigational therapies are manufactured at Cartesian’s wholly-owned, state-of-the-art cGMP manufacturing facility in Gaithersburg, MD. Cartesian’s commanding IP position benefits in part from a broad, exclusive patent license from the National Cancer Institute. The start-up employees under 20.

Lead Research/Investigator

Volkan Granit, MD 

Call to Action: Do you or a loved one have a diagnosis of generalized myasthenia gravis (GMG)? If so, you might want to keep track of this more targeted investigational therapy. TrialSite News will monitor the success of this study. Myasthenia gravis News offers good consistent coverage of ongoing news and events