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mHTT-ZFP
- Development stage
- Preclinical
- Lead developer
- Sangamo Therapeutics
- Modality
- Gene Therapies
- Administration
- Parenteral
01
Overview
mHTT-ZFP (also known as TAK-686) is an investigational allele-selective zinc finger protein transcription factor (ZFP-TF) gene regulation therapy designed for the treatment of Huntington's disease. Developed by Sangamo Therapeutics in collaboration with the CHDI Foundation and licensed to Takeda, the therapy utilizes an adeno-associated virus (AAV) vector to deliver engineered zinc finger proteins directly to the brain. These ZFPs are designed to selectively bind to the expanded CAG repeat tract in the mutant huntingtin (mHTT) gene, repressing its transcription and thereby lowering the production of the toxic mutant huntingtin protein while sparing the expression of the healthy wild-type huntingtin allele.
- Other names
- AAV-ZFP30645-ZFNAAV-ZFP-30645-ZFNAAV-ZFP 30645-ZFNallele-selective zinc finger protein transcription factor
02
Targets
HTT (Huntingtin protein)
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