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ST-506

Development stage: Preclinical
Lead developer: Sangamo Therapeutics
Modality: Gene Therapies
Administration: Intravenous

Overview

ST-506 is an investigational gene therapy being developed by Sangamo Therapeutics. It is an adeno-associated virus (AAV)-based therapy that utilizes a Zinc Finger Repressor (ZFR) to achieve sustained epigenetic downregulation of prion protein (PrPC) expression in neurons throughout the brain. The therapy is designed for one-time, intravenous administration and is being investigated as a potential treatment for prion disease. A novel engineered AAV capsid, STAC-BBB, is employed to enhance brain expression and reduce liver targeting compared to AAV9.

Targets

Human Receptor 1 (at the blood-brain barrier)PRNP (Cellular Prion Protein)

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