Delivery of tau repressors using the blood-brain barrier penetrant capsid

AU2025234423A1Pending Publication Date: 2026-09-10SANGAMO THERAPEUTICS INC
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Patent Information

Application Number
AU2025234423
Authority / Receiving Office
AU · AU
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-11-12
Filing Date
2025-03-12
Publication Date
2026-09-10

AI Technical Summary

Technical Problem

Current treatments for tauopathies, such as Alzheimer's disease and frontotemporal dementia, face challenges in effectively delivering therapeutic agents across the blood-brain barrier and targeting specific brain regions affected by tau pathology, leading to inadequate reduction of tau protein levels and potential immune responses.

Method used

Development of recombinant adeno-associated virus (rAAV) particles encapsulating a zinc finger protein (ZFP) that targets and represses microtubule-associated protein tau (MAPT) gene expression, allowing for one-time administration and widespread distribution within the central nervous system, including key brain regions.

Benefits of technology

The ZFP approach achieves significant and sustained reduction of tau protein levels in targeted brain regions, inhibiting tau aggregation and propagation, offering a promising treatment for tauopathies with reduced immune response and improved therapeutic efficacy.

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Abstract

Provided are compositions for delivery of zinc finger fusion proteins that inhibit expression of tau in the nervous system using the blood-brain barrier penetrant AAV capsid proteins comprising SEQ ID. NO: 1235, and methods of using the compositions to treat neurodegenerative diseases such as Alzheimer's disease, frontotemporal dementia, and other tauopathies.
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