Cartesian Therapeutics Reports Positive Early Clinical Results with Descartes-08, an mRNA-Engineered Anti-BCMA CAR-T Therapy, in Generalized Myasthenia Gravis

On August 24, 2021, Cartesian Therapeutics announced that its investigational therapy, Descartes-08, achieved positive results in a Phase 1b/2a clinical trial for the treatment of patients with generalized myasthenia gravis (gMG). Descartes-08 is an autologous mRNA-engineered chimeric antigen receptor T-cell (CAR-T) therapy targeting B-cell maturation antigen (BCMA). In a cohort of three patients, all demonstrated significant symptomatic improvement, and the therapy was well-tolerated.

Descartes-08 is a CAR-T cell therapy generated using an mRNA-encoded CAR approach. Unlike current anti-B cell therapies, it targets and eliminates pathogenic long-lived plasma cells capable of persisting in the body for decades, holding promise for long-term suppression of autoantibody production.

The press release stated that this non-randomized, single-arm, open-label Phase 1b/2a trial is the first clinical trial of a CAR-T cell therapy for autoimmune diseases. The trial plans to enroll 18 subjects to evaluate the safety and tolerability of Descartes-08 for the treatment of gMG. Trial results indicated that Descartes-08 was well-tolerated, with no reported cytokine release syndrome (CRS) or other serious product-related adverse events (n=3). Furthermore, all patients achieved comprehensive improvement in the Myasthenia Gravis Foundation of America (MGFA) clinical classification and demonstrated significant improvement on the Myasthenia Gravis Composite (MGC) scale, which assesses overall disease severity. Approximately three months after dosing, the Descartes-08 group showed an average improvement of over 50% on the MGC scale.

gMG is a chronic autoimmune disease in which autoantibodies attack specific proteins at the neuromuscular junction, disrupting neuromuscular transmission and leading to muscle weakness and fatigue. Patients with gMG may experience a range of symptoms, from ptosis and diplopia to debilitating muscle weakness and respiratory failure.

▲ Working principle of Cartesian’s RNA Armory technology platform, which utilizes mRNA to express target proteins (Image source: Cartesian official website)

References:

[1] Cartesian Therapeutics Announces Clinical Responses in First Cohort of Phase 1b/2a Trial in Myasthenia Gravis. Retrieved August 24, from https://www.prnewswire.com/news-releases/cartesian-therapeutics-announces-clinical-responses-in-first-cohort-of-phase-1b2a-trial-in-myasthenia-gravis-301361281.html

(Original text abridged)

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