Fusion proteins comprising Zinc-finger proteins (ZFPs) are powerful tools for directed genome modification and regulation. Engineered ZFP DNA binding proteins can bind specifically to virtually any DNA sequence and can be fused to an array of effector domains including activation and repression domains as well as catalytic nuclease domains. Engineered zinc finger-based fusion proteins represent a novel modality to treat human diseases.

Sangamo Therapeutics focuses on genomic medicines in multiple therapeutic areas including inherited metabolic diseases, hematology, CNS diseases, oncology, and immunology using ZFPs as therapeutic modalities. The presentation will focus on two novel approaches for the treatment of neurodegenerative diseases and autoimmunity.