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ST-601
- Development stage
- Preclinical
- Lead developer
- Biogen
- Modality
- Gene Silencing → Gene Therapies, Gene Addition/Replacement → Gene Therapies, Zinc Finger Nucleases → Other Nucleases → Programmable Nucleases → Gene Editing → Gene Therapies
- Administration
- Intracerebral, Intraparenchymal
01
Overview
ST-601 is an investigational genomic medicine designed to treat Parkinson's disease and other synucleinopathies by targeting the underlying genetic cause of protein aggregation. Developed using proprietary zinc finger protein transcription factor (ZFP-TF) technology, ST-601 is engineered to specifically bind to the promoter region of the **SNCA** gene, which encodes the **alpha-synuclein** protein. By acting as a transcriptional repressor, ST-601 downregulates the expression of SNCA, thereby reducing the production of alpha-synuclein and preventing the formation of pathological aggregates known as Lewy bodies. The therapeutic is typically delivered to the central nervous system via an adeno-associated virus (AAV) vector. Originally part of a major neurodegeneration collaboration between Sangamo Therapeutics and Biogen, the rights to the program were returned to Sangamo in 2023, where it continues in preclinical development.
- Other names
- Sangamo ST-601ZFP-TF alpha-synuclein repressor
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