Compositions and methods for modulating apoe

WO2024259232A3 Publication Date: 2025-04-17LEAL THERAPEUTICS INC
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Patent Information

Application Number
PCT/US2024/034006
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-02-21
Filing Date
2024-06-14
Publication Date
2025-04-17

AI Technical Summary

Technical Problem

Current treatments for neurodegenerative diseases such as Alzheimer’s and dementia with Lewy Bodies are inadequate in addressing the accumulation of amyloid plaques and neurofibrillary tangles, as existing therapies fail to effectively modulate the key proteins involved in these processes.

Method used

The development of isolated nucleic acids, specifically antisense oligonucleotides, that bind to mRNA transcripts of genes like APOE to modulate their expression, reducing the production of apolipoprotein E, thereby decreasing amyloid plaque formation and associated neurodegenerative effects.

Benefits of technology

This approach effectively decreases the levels of functional apolipoprotein E, leading to reduced amyloid plaque formation and potentially treating or preventing neurodegenerative diseases by targeting the underlying genetic mechanisms.

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Abstract

Aspects of the disclosure relate to compositions and methods for modulating levels, transcription, splicing, and / or translation of one or more RNA transcripts (e.g., mRNA transcripts) in a cell or subject. The disclosure is based, in part, on isolated nucleic acids that bind to mRNA transcripts of genes involved in neurodegenerative diseases and disorders, for example an apolipoprotein E gene (APOE), which encodes a protein involved in lipid homeostasis and amyloid plaque formation in the CNS. In some embodiments, compositions of the disclosure are useful for treating neurodegenerative diseases or disorders, such as Alzheimer's disease (AD) or dementia with Lewy Bodies.
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