Recombinant aav for the gene therapy of wilson disease

EP4684025A1Pending Publication Date: 2026-01-28SKYLINE THERAPEUTICS LTD
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Patent Information

Application Number
EP2024720732
Authority / Receiving Office
EP · EP
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-03-22
Filing Date
2024-03-21
Publication Date
2026-01-28

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  • Figure PCTCN2024082965-FTAPPB-I100001
    Figure PCTCN2024082965-FTAPPB-I100001
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    Figure PCTCN2024082965-FTAPPB-I100002
  • Figure PCTCN2024082965-FTAPPB-I100003
    Figure PCTCN2024082965-FTAPPB-I100003
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Abstract

The present invention provides a polynucleotide and recombinant AAV encoding a functional copper‐transporting ATPase. The present invention also provides a method of treating Wilson disease comprising administering the recombinant AAV to a subject in need thereof.
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