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US 2022/0275367 A1Published application
Compositions and methods for treating huntington's disease
AssigneeVoyager Therapeutics, Inc.
InventorsDinah Wen-Yee Sah, Pengcheng Zhou, Jeffrey S. Thompson, Christina Gamba-Vitalo, Jenna Carroll Soper, Steven M. Hersch, Todd Carter, Jacob J. Cardinal, Lori B. Karpes, Bin Liu
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Abstract
The present disclosure relates to pharmaceutical compositions of adeno-associated viral (AAV) particles encoding siRNA molecules and methods for treating Huntington's Disease (HD).
Claims51
A pharmaceutical composition for use in the treatment of Huntington's Disease, said composition comprising adeno-associated virus (AAV) particles in a pharmaceutically acceptable formulation;
- wherein at least one of the AAV particles comprises an AAV viral genome comprising a polynucleotide sequence selected from the group consisting of SEQ ID NO:
- 1352-1379, 1388, and 1426-1438 or variants of the foregoing having at least 95% identity thereto;
- and wherein the concentration of the AAV viral genome in the pharmaceutical composition is from 1×1011 to 1×1012 VG/mL.
Dependencies are inferred from the claim text.