Kyverna Therapeutics, Inc. (Kyverna) a California-based clinical-stage biopharmaceutical company, announced on Monday that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for the company's autologous, fully human CD19 chimeric antigen receptor (CAR) T-cell product candidate, KYV-101, intended for the treatment of patients suffering from refractory stiff-person syndrome, a rare autoimmune disease.

KYV-101 is an autologous, fully human CD19 CAR T-cell product candidate for use in B cell-driven autoimmune diseases. The CAR in KYV-101 was designed by the National Institutes of Health (NIH) to improve tolerability and tested in a 20-patient Phase 1 trial in oncology. Results were published by the NIH in Nature Medicine.

KYV-101 is currently being evaluated in sponsored, open-label, Phase 1/2 and Phase 2 trials of KYV-101 in the United States and Germany across two broad areas of autoimmune disease: rheumatology and neurology.

Amanda Piquet, MD, director of the Autoimmune Neurology Program at CU Anschutz Medical Campus, Aurora, Colorado, said, 'Stiff-person syndrome has devastating and life-altering effects on patients suffering from this rare autoimmune disease. I look forward to the data that will emerge from the KYSA-8 trial as this trial could drastically change the treatment landscape for SPS.'