Cartesian Therapeutics announces successful trial of RNA cell therapy in autoimmunity

Betsy Goodfellow | June 23, 2023 | News story | Research and Development Cartesian Therapeutics, Neurology, RNA, autoimmune, neurology 

Clinical stage biotechnology company, Cartesian Therapeutics, has announced results from its clinical trial using RNA CAR T (rCAR T) therapy to treated patients with generalised myasthenia gravis (MG), an autoimmune neurological disease.

The data from this trial was published in The Lancet Neurology and demonstrates a long-lasting clinical improvement in patients with MG.

The study included 14 patients with generalised MG, who received 6 infusions of rCAR T therapy as an outpatient treatment without lymphodepletion. The patients were assessed for an average of six months. The trial showed the rCAR T was well tolerated with no dose-limiting toxicities, cytokine release syndrome or neurotoxicity.

The drug had marked and long-lasting clinical improvements across four MG disease scoring systems, with average improvements at six months approximately three times greater than the thresholds considered clinically meaningful. Most patients sustained long-term clinical benefit, with three patients achieving complete or near-complete eradication of disease symptoms.

Dr James Howard, professor of Neurology at University of North Carolina – Chapel Hill, US and a senior author on the paper, commented: “Currently, the mainstay of myasthenia therapy is chronic use of broad immunosuppression, which has many drawbacks. The prospect of inducing potent, durable responses and reducing or eliminating use of immunosuppressive therapy is very appealing to the myasthenia community. Based on the compelling results of this paper, we are now enrolling patients with MG into a randomised placebo-controlled study, the first study of its kind for an engineered adoptive cell therapy.”

Samantha Masterson, president and CEO of Myasthenia Gravis Foundation of America, added: “We are grateful to our community of MG patients and physicians for enabling clinical development of novel therapeutics such as rCAR T. A safe, personalised therapy with durable clinical benefit would be a welcome addition to the growing toolkit of MG treatments.”

Betsy Goodfellow

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