Modified Adeno-Associated Virus Capsid Proteins and Methods Thereof

JP2025518145A5Pending Publication Date: 2026-05-29MOUNT SPEC INVESTMENTS PTY LTD
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
MOUNT SPEC INVESTMENTS PTY LTD
Filing Date
2023-05-31
Publication Date
2026-05-29

AI Technical Summary

Technical Problem

Current recombinant adeno-associated virus (AAV) vectors face challenges in efficiently transducing retinal cells and inducing a reduced immunogenic response, particularly due to the presence of neutralizing antibodies and physical barriers in the eye.

Method used

Development of modified recombinant AAV particles with a modified capsid protein, specifically an insertion sequence set forth in SEQ ID NO: 1 or its functional equivalent, which enhances transduction efficiency and reduces immunogenicity by improving virus stability and tropism.

Benefits of technology

The modified AAV particles demonstrate increased transduction efficiency and reduced immunogenicity compared to unmodified AAV particles, effectively improving gene therapy outcomes for eye disorders such as age-related macular degeneration.

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Abstract

The present invention relates to a modified recombinant adeno-associated virus (AAV) capsid protein and AAV particles thereof. In one aspect, the modified recombinant AAV particles provide an increase in transduction of retinal cells as compared to the effect of unmodified recombinant AAV particles. The present invention also relates to a nucleic acid encoding the modified AAV capsid protein and AAV particles thereof.
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