Cartesian Therapeutics Receives FDA Rare Pediatric Disease Designation for Descartes-08 for the Treatment of Juvenile Dermatomyositis
Cartesian Therapeutics, Inc. recently announced the US FDA has granted Rare Pediatric Disease Designation to Descartes-08 for the treatment of juvenile dermatomyositis (JDM). Descartes-08, Cartesian’s lead mRNA cell therapy candidate, is an autologous mRNA-engineered chimeric antigen receptor T-cell therapy (mRNA CAR-T) product candidate targeting B-cell maturation antigen (BCMA). Descartes-08 is designed to be administered without preconditioning chemotherapy and does not use integrating vectors.
“We are pleased that the FDA recognizes the potential of Descartes-08 to serve as a meaningful therapeutic option for this underserved pediatric patient population,” said Carsten Brunn, PhD, President and Chief Executive Officer of Cartesian. “Leveraging our novel mRNA platform, we are committed to our mission of expanding the reach of cell therapy to patients with autoimmune diseases. We remain on track to file an Investigational New Drug application for a Phase 2 pediatric basket study focused on neurology and rheumatology autoimmune indications, including JDM, by year-end.”
The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children ages 18 years or younger and fewer than 200,000 people in the US. Under the FDA’s Rare Pediatric Disease Designation and Voucher Program, if Decartes-08 is approved for marketing in JDM, Cartesian may qualify for a priority review voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product.
Juvenile Dermatomyositis (JDM) is a rare pediatric autoimmune disorder marked by pathognomonic skin rash and muscle inflammation affecting multiple organ systems including the joints, heart, lungs, kidneys, eyes, and gastrointestinal systems. The symptoms of JDM can range from mild to life-threatening and symptoms often include fatigue, joint pain, muscle weakness and fever. JDM impacts approximately 4,000 people in the US.
Cartesian Therapeutics is a clinical-stage company pioneering mRNA cell therapies for the treatment of autoimmune diseases. The Company’s lead asset, Descartes-08, is an mRNA CAR-T in Phase 2b clinical development for patients with generalized myasthenia gravis and Phase 2 development for systematic lupus erythematosus, with a Phase 2 basket trial planned in additional autoimmune indications. The Company’s clinical-stage pipeline also includes Descartes-15, a next-generation, autologous anti-BCMA mRNA CAR-T. For more information, visit www.cartesiantherapeutics.com.
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