AAV vectors for treating complement pathway 2-related diseases

The CR2-FH fusion protein delivered via AAV vectors addresses the lack of effective treatments for dry AMD by regulating complement activation, reducing inflammation and oxidative stress, and preserving retinal cells.

JP2026522196APending Publication Date: 2026-07-07STARRYGENE THERAPEUTICS CO LTD
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
STARRYGENE THERAPEUTICS CO LTD
Filing Date
2024-05-17
Publication Date
2026-07-07

AI Technical Summary

Technical Problem

Current treatments for dry age-related macular degeneration (AMD) are inadequate, and there is a need for new therapeutic agents that can effectively target complement pathway II-related disorders, particularly those affecting the retina, to prevent vision loss and blindness.

Method used

Development of a CR2-FH fusion protein using adeno-associated virus (AAV) vectors to deliver a CR2-FH fusion protein to complement activation sites, which includes a CR2 moiety with four N-terminal SCR domains and an FH moiety with varying SCR domains, linked by a (G4S)n linker sequence, to regulate complement activation and reduce tissue damage.

Benefits of technology

The CR2-FH fusion protein effectively targets complement activation sites, reducing inflammation and oxidative stress, thereby preserving retinal cells and improving vision in dry AMD models.

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Abstract

This disclosure provides a CR2-FH fusion protein, a polynucleotide encoding it, a vector, a pharmaceutical composition, and its use, which can be used to treat complement pathway II-related diseases, such as dry AMD.
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