CAR-T Therapy for Myasthenia Gravis (MG)

Myasthenia Gravis (MG) is a chronic autoimmune neuromuscular disorder caused by autoantibodies (primarily anti-acetylcholine receptor antibodies, AchRAb) that impair communication between nerves and muscles, leading to fluctuating weakness, ptosis, dysphagia, and in severe cases life-threatening respiratory failure. For patients with refractory MG who fail conventional immunosuppressants, IVIG, FcRn inhibitors, and complement inhibitors, treatment options remain limited.

BIOOCUS’s dual-target CD19/BCMA CAR-T therapy represents a breakthrough autologous cell therapy that precisely eliminates pathogenic B cells and plasma cells responsible for autoantibody production, offering the potential for deep and durable remission in difficult-to-treat MG.

CAR-T therapy for MG is an advanced, personalized immunotherapy in which a patient’s own T cells are genetically engineered to express chimeric antigen receptors (CARs) targeting both CD19 (on B cells) and BCMA (on plasma cells). After expansion in the laboratory, these dual-target CAR-T cells are infused back into the patient, where they selectively deplete the autoreactive immune cells driving MG, thereby reducing AchRAb levels and restoring neuromuscular function.

This dual-target approach addresses both the cellular and humoral components of the autoimmune response, providing a more comprehensive reset of the immune system compared to single-target or traditional therapies.

• Rapid and sustained symptom improvement: Significant reduction in muscle fatigue, ptosis, diplopia, dysarthria, and limb weakness.
• Deep B-cell and plasma-cell depletion: Marked and lasting decrease in AchRAb and total B-lymphocyte counts.
• Improved clinical scores: Substantial gains in Quantitative Myasthenia Gravis (QMG) and Activities of Daily Living (ADL) scores.
• Favorable safety profile: Well-tolerated with no cytokine release syndrome (CRS) or neurotoxicity observed in treated cases.
• Potential for long-term remission: Reduces or eliminates the need for chronic immunosuppression.
• Personalized & precise: Uses the patient’s own cells, minimizing rejection risk.

1. Leukapheresis– Collection of the patient’s T cells via peripheral blood apheresis.
2. Genetic Engineering– T cells are modified to express CD19 and BCMA CARs and expanded in GMP facilities.
3. Conditioning (if required)– Brief lymphodepletion to optimize CAR-T engraftment.
4. CAR-T Infusion– Single intravenous infusion of CD19 CAR-T (0.5×10⁶/kg) + BCMA CAR-T (1×10⁶/kg).
5. Monitoring & Follow-up– Regular assessment of CAR-T expansion, AchRAb levels, B-cell counts, QMG/ADL scores, and organ function for up to 12+ months.

• Cutting-edge dual-target CD19/BCMA CAR-T platform specifically optimized for autoimmune diseases.
• World-class GMP manufacturing and experienced immunotherapy team with expertise in both hematologic malignancies and refractory autoimmune conditions.
• Comprehensive international patient support: multilingual coordination, airport transfer, accommodation, and long-term follow-up.
• Proven track record in CAR-T for autoimmune disorders (including SLE) and personalized treatment planning.
• Full-spectrum care from pre-treatment evaluation to post-infusion monitoring.