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ST-501

Development stage: Preclinical
Lead developer: Biogen
Modality: AAV Vectors → Viral Vectors → Gene Addition/Replacement → Gene Therapies, Zinc Finger Nucleases → Other Nucleases → Programmable Nucleases → Gene Editing → Gene Therapies
Administration: Intravenous

Overview

ST-501 is a preclinical AAV-based gene therapy developed by Sangamo Therapeutics using a zinc finger protein (ZFP) transcription factor platform to target tau gene expression. It functions as a **genetic transcription inhibitor** and a **tau protein inhibitor**, aiming to suppress tau pathology associated with tauopathies such as Alzheimer's disease and related neurodegenerative disorders. The therapy is not classified as a new molecular entity and is currently being developed in partnership with Biogen. The drug is not assigned orphan drug status and is available for licensing for Alzheimer's disease. Sangamo intends to submit an IND to the US FDA for Alzheimer's disease in Q4 2025[1][5].

Other names: Tau targeting ZFP TF-Sangamo Therapeutics

Targets

DNA

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