Improvement of myasthenia by b-cell specific ma-siRNA conjugates

By using the combination of anti-BAFF receptor and anti-BCMA-specific receptor antibodies with small RNA, the problem of difficult to effectively reduce pathogenic autoantibodies in the prior art is solved, and the effect of significant reduction in antibody levels and prolonging disease remission time is achieved.

CN119677544APending Publication Date: 2025-03-21ASSOCIATION FRANCAISE CONTRE LES MYOPATHIES (AFM) +1
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Patent Information

Application Number
CN202380055818.0
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2022-07-29
Filing Date
2023-07-28
Publication Date
2025-03-21

AI Technical Summary

Technical Problem

The prior art is difficult to effectively reduce the production of pathogenic autoantibodies and complement activation, resulting in the recurrence or recurrence of autoimmune muscle diseases such as myasthenia gravis.

Method used

The combination of two antibody-RNA complexes, including anti-BAFF receptor antibodies or their binding fragments binding to small RNAs targeting BAFF receptor mRNAs, and anti-BCMA-specific receptor antibodies or their binding fragments binding to small RNAs targeting BCMA receptor mRNAs, forming a biconjugate to target and degrade related mRNAs, thereby inhibiting B cell function.

Benefits of technology

It significantly reduced the autoantibodies level, reduced complement activation, prolonged the time for disease remission, and improved the clinical improvement effect of the mouse model of myasthenia gravis.

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Abstract

The present invention relates to a combination of two antibody-RNA conjugates, the combination comprising: an anti-BAFF receptor antibody or a binding fragment thereof, the anti-BAFF receptor antibody or a binding fragment thereof being bound or conjugated to a small RNA targeting BAFF receptor mRNA; and an anti-BCMA specific antibody or a binding fragment thereof, the anti-BCMA specific antibody or the binding fragment thereof being bound or conjugated to a small RNA targeting the BAFF receptor mRNA. Compositions comprising this combination will be used for the treatment of autoimmune disorders, preferably myasthenia gravis.
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