Cartesian Therapeutics, Inc., a biotechnology company, recently announced that the FDA has awarded the Regenerative Medicine Advanced Therapy (RMAT) designation to its lead product, Descartes-08, to treat myasthenia gravis (MG).
The FDA’s programs are designed to support drug development and the review processes needed to bring safe and effective products to market for patients with rare diseases.
Myasthenia gravis, a chronic neuromuscular disease, weakens the voluntary muscles such as those in the face, diaphragm, and throat. Muscles that are essential to swallowing, facial movements, and breathing.
About Descartes-08
MG is an autoimmune disease meaning that the defense system gets off target by attacking healthy proteins or cells that are needed for the body to function normally.
Descartes-08 is the company’s leading product. B cell maturation antigen is a prime target. It is autologous, meaning from the same person, not donated.
Cartesian’s CEO, Dr. Carsten Brunn, told Globe Newswire that the company is on track and prepared to report promising data from its Phase 2b trial of MG mid-2024 (NCT04146051). The company previously announced positive 12-month data in January 2024.
Descartes-08 also received the FDA’s Orphan Drug Designation granted to biological products that prevent, treat, or diagnose a rare disease.
About RMAT
To qualify for RMAT, the new technology requires that a product is autologous, and activates its own stem cells to replace damaged cells.
The technology must be intended to modify, treat, reverse, and/or cure a life-threatening condition. It must display clinical evidence of having the potential to meet unmet needs of the disease.
Sponsor companies benefit from these programs through FDA meetings and facilitating drug development.
About Study Results
Descartes-08 was administered minus preconditioning chemotherapy or integrating vectors that transport disease-causing organisms to the host.
Researchers observed depletion of autoantibodies and MG severity scores were improved. Descartes-08 appears to be well-tolerated.
About the Company
Cartesian Therapeutics Inc., Gaithersburg, Md., is a pioneer in mRNA therapies to treat autoimmune diseases. Descartes-08 leads its pipeline with its Phase 2b trial that is in clinical development to treat generalized myasthenia gravis patients.
