New preliminary clinical data evaluating an induced pluripotent stem cell (iPSC)-derived CAR T-cell therapy for patients with treatment-resistant systemic sclerosis was presented at ISSCR 2026. The early findings are from the systemic sclerosis cohort of an ongoing Phase 1 basket trial investigating FT819, an investigational off-the-shelf iPSC-derived CAR T-cell therapy in autoimmune diseases.
Systemic sclerosis is a rare autoimmune disease that can affect multiple organs and has the highest mortality rate among rheumatic diseases. Current treatments focus mainly on slowing disease progression, and there are no approved therapies specifically for systemic sclerosis. The study enrolls patients with active, treatment-refractory systemic sclerosis who have experienced prior treatment failure. Unlike some previous studies, this trial does not impose an upper limit on disease duration for eligibility, allowing a broader range of participants.
FT819 is designed as an off-the-shelf product derived from an established iPSC cell bank, which allows doses to be readily available and potentially supports scalable distribution. This differs from conventional autologous CAR T-cell therapies that require individualized manufacturing. According to the presentation, FT819 has shown a reassuring safety profile so far and has been administered successfully in outpatient settings for patients with rheumatic diseases. Funding support came in part from the California Institute for Regenerative Medicine (CIRM), a state agency supporting research in regenerative medicine.
"Improving access is an important part of advancing cell therapy for autoimmune disease," said Dr. Shiff. "An off-the-shelf approach has the potential to reduce many of the manufacturing and logistical challenges associated with personalized cell therapies while expanding access to patients who might otherwise face barriers to receiving these treatments." Preliminary observations indicate signals of clinical improvement; if confirmed by larger studies with longer follow-up, these findings could represent progress toward improving quality of life and reducing disease burden in affected patients.
"While these findings require additional study, the early signals are encouraging for a patient population with significant unmet medical needs," said Dr. Shiff. "Our next steps are to continue evaluating the safety, efficacy, and durability of FT819 in systemic sclerosis and other rheumatic diseases, while identifying which patients are most likely to benefit." Beyond systemic sclerosis, researchers believe this platform may have applications across other autoimmune conditions such as lupus.
