Compositions and methods for treating huntington's disease

By delivering siRNA molecules targeting the Huntington gene, silencing the HTT gene has been solved, and the treatment difficulties of Huntington's disease are achieved selectively reducing HTT expression and preventing disease progression.

CN120569474APending Publication Date: 2025-08-29ATALANTA THERAPEUTICS INC
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Patent Information

Application Number
CN202380076889.9
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2022-09-28
Filing Date
2023-09-28
Publication Date
2025-08-29

AI Technical Summary

Technical Problem

There is currently no effective method for treating Huntington's disease. Abnormal expression of Huntington's protein leads to disease onset and progression, and it is difficult for the prior art to selectively reduce HTT activity.

Method used

The mRNA of the Huntington gene is targeted using small interfering RNA (siRNA) molecules, and is delivered directly to the subject through intrathecal injection, intraventricular injection, striatal injection, parenchymal injection, intracerebellar medulla stool injection, intravenous injection, subcutaneous injection or intramuscular injection, to silencing the HTT gene to prevent mRNA translation and reduce HTT protein expression.

Benefits of technology

Through siRNA-mediated silencing of HTT transcripts, selectively reduces HTT levels and prevents disease onset or progression, providing an effective treatment for Huntington's disease.

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Abstract

The present disclosure provides single-stranded or double-stranded interfering RNA molecules (e.g., siRNAs) that target the Huntington (HTT) gene. The interfering RNA molecule may contain a specific pattern of nucleoside modification and inter-nucleoside linkage modification as a pharmaceutical composition comprising the interfering RNA molecule. The siRNA molecule may be a branched siRNA molecule, such as a two-branched, three-branched or four-branched siRNA molecule. The disclosed siRNA molecules may also be characterized by a 5 '-phosphorus stabilizing moiety and / or a hydrophobic moiety. In addition, the present disclosure provides methods for delivering siRNA molecules of the present disclosure to a subject, such as the central nervous system of a subject identified as having Huntington's disease.
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Citation Information

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