Human PAH expression cassette for treatment of PKU by liver-directed gene replacement therapy

NZ837294APending Publication Date: 2026-09-25GENZYME CORP
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Patent Information

Application Number
NZ837294
Authority / Receiving Office
NZ · NZ
Patent Type
Applications
Current Assignee / Owner
Priority Date
2020-12-04
Filing Date
2021-09-30
Publication Date
2026-09-25

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Abstract

Provided herein are expression cassettes for expressing a transgene in a liver cell, wherein the transgene encodes a PAH polypeptide. Also provided are methods to treat phenylketonuria (PKU) and / or to reduce levels of phenylalanine in an individual in need thereof. Further provided herein are vectors (e.g., rAAV vectors), viral particles, pharmaceutical compositions and kits for expressing a PAH polypeptide in an individual in need thereof.
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