Kyverna Therapeutics says it has gained alignment with the FDA on a single-arm trial and path to submission for its CAR-T cell therapy, miv-cel, following a positive pre-BLA meeting.
According to Kyverna, the company has initiated its rolling BLA submission in stiff person syndrome, seeking priority review under the program's RMAT designation, and anticipates completing the submission in Q4 2026. Kyverna will include additional analysis of its completed natural history study presented at the 2026 American Academy of Neurologyannual meeting and planned one-year follow-up data from its phase 2 registrational trial, KYSA-8.
Miv-cel is an autologous, CD19-targeting CAR-T cell therapy with CD28 co-stimulation, designed for potency and tolerability. In April, the company announced positive primary analysis results from KYSA-8, where a single dose of miv-cel delivered rapid, statistically significant and clinically meaningful improvements across all primary and secondary endpoints at 16 weeks, with the majority of patients regaining function, and all 26 patients discontinuing chronic immunotherapies. According to Kyverna, these outcomes have not been previously observed in SPS — a rare, progressive neurologic autoimmune disease characterized by muscle stiffness and painful muscle spasms, impacting mobility and gait.
The company is also testing the therapy in myasthenia gravis and progressive multiple sclerosis.
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