Compositions, systems, and methods for reducing low density lipoprotein through gene silencing targeting a target

An epigenetic DNA targeting system using CRISPR-Cas/guide RNA technology represses multiple genes to reduce LDL levels, overcoming the limitations of existing therapies by enhancing efficacy and safety in managing familial hypercholesterolemia.

JP2025524469A5Pending Publication Date: 2026-07-01TUNE THERAPEUTICS INC
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
TUNE THERAPEUTICS INC
Filing Date
2023-06-23
Publication Date
2026-07-01

AI Technical Summary

Technical Problem

Current treatments for familial hypercholesterolemia, such as statin therapy and antibody therapy, face challenges including toxicity and high costs associated with repeated drug administration, necessitating the development of new and improved methods to manage elevated LDL cholesterol levels.

Method used

An epigenetic modification DNA targeting system comprising multiple DNA targeting modules, each with a fusion protein containing a DNA-binding domain and a transcription repressor domain, is used to repress the transcription of genes regulating LDL without causing gene disruption or DNA cleavage, utilizing CRISPR-Cas/guide RNA systems to target specific gene regulatory elements.

Benefits of technology

The system effectively reduces LDL levels by repressing the transcription of multiple genes, achieving a greater reduction than individual gene repression, thereby addressing the limitations of existing therapies and providing a safer, more cost-effective approach for managing cardiovascular disease risk.

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Abstract

In some aspects, an epigenetic modification DNA targeting system, such as a CRISPR-Cas / guide RNA (gRNA) system for transcriptional repression of genes, is provided to promote a cell phenotype that results in a reduction of low density lipoprotein (LDL). In some embodiments, the epigenetic modification DNA targeting system binds to or targets a target site of at least one gene that regulates LDL or its regulatory element. In some embodiments, the system is a multiplexed system that binds to or targets target sites in at least two genes or their regulatory elements. Also provided herein are methods and uses related to the provided epigenetic modification DNA targeting system related to the treatment of cardiovascular disease and familial hypercholesterolemia.
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