Compositions and methods for treating complement-mediated diseases

By using a recombinant AAV vector to express an engineered fH protein variant, the problem of frequent drug administration required by existing treatments has been solved, enabling long-term treatment of complement-mediated diseases and reducing interference with the host's defense system.

CN115976105BActive Publication Date: 2026-07-10THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
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Patent Information

Authority / Receiving Office
CN · China
Patent Type
Patents(China)
Current Assignee / Owner
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
Filing Date
2016-09-23
Publication Date
2026-07-10

AI Technical Summary

Technical Problem

Existing treatments for complement-mediated diseases require frequent administration and may impair host defenses, making it difficult to achieve long-term, sustained therapeutic effects.

Method used

The engineered human complement regulator H (fH) protein variant, containing a specific short common repeat (SCR), was expressed using a recombinant vector and delivered to the subject via an AAV vector to ensure that an effective plasma level was maintained in vivo for at least one week or one month.

Benefits of technology

It enables long-term treatment of complement-related diseases, reduces the frequency of drug administration, minimizes interference with the host's defense system, and provides more stable therapeutic effects.

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Abstract

The present invention relates to compositions and methods for treating complement-mediated diseases. Provided are recombinant vectors having an expression cassette comprising a modified human factor H (hfH) gene, wherein the hfH gene encodes a hfH protein variant comprising SCR1-4, 19-20, and one or more of: SCR7, SCR17, and / or SCR18. Also provided are pharmaceutical compositions comprising the vectors and their use in treating AMD and / or other complement-related diseases.
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