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December 8, 2025BloodOpen Access

Safety and preliminary efficacy of a novel loop-optimized, BCMA/CD19 bispecific CAR-T therapy for refractory or relapsed autoimmune diseases patients

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Authors

JPJing PanAZAng ZhangSLSha Li

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Overview

Pilot study demonstrates safety and preliminary efficacy of bispecific CAR-T therapy in systemic lupus erythematosus and systemic sclerosis patients, suggesting a promising treatment approach.

Key Points

  • This research aims to evaluate the safety and efficacy of a novel bispecific CAR-T therapy targeting BCMA and CD19 in refractory autoimmune diseases.
  • Developed a bispecific CAR structure using proprietary nanobodies.
  • Conducted a pilot study evaluating safety and efficacy in patients with systemic lupus erythematosus, systemic sclerosis, and primary Sjögren's syndrome.
  • Measured clinical outcomes using established criteria like SLEDAI, LLDAS, SRI-4, EUSTAR-AI, and ESSDAI.
  • Among 12 treated patients, the overall response rate for systemic lupus erythematosus was 83.3%, with 4 achieving complete remission.
  • All systemic sclerosis patients showed positive response rates at 1 month, with significant reductions in modified Rodnan scores.
  • Safety outcomes indicated low incidence of CRS and no severe adverse events.

Cite This Study

Pan et al. (2025) studied this question.

synapsesocial.com/papers/69362f634fa91c937236ddb2https://doi.org/10.1182/blood-2025-2370
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