Our scientific efforts are thoughtfully focused on serious conditions for which no adequate treatment exists.

Zinc Finger Platform

Sangamo’s proprietary zinc finger (ZF) platform uniquely confers a number of advantages and has broad therapeutic potential due to its versatility, specificity, clinical utility and compact nature.

Zinc finger technologies are derived from naturally occurring human proteins. They are composed of a DNA-binding domain, which binds with precision to any sequence of DNA, and various functional domains (such as nucleases and transcription factors) based on the therapeutic needs. Due to their compact size, ZF technologies are highly compatible with delivery vectors.

Delivery Platform

Combining ZF technologies with delivery vectors allows for the creation of ZF-based genomic medicines. Our platform can be used to create genomic medicines both ex vivo (cell therapy) and in vivo (genome engineering).

To effectively bring zinc finger technologies into targeted cells, we employ delivery vectors, which serve as vehicles for transporting genetic material. These vectors can originate from inactivated viruses, such as adeno-associated virus (AAV) or lentivirus (LVV).  We are evaluating several potential routes of administration for our neurology-targeted investigational therapies, as delivery of genomic medicines to the central nervous system is a significant obstacle to developing potential therapies treating neurological disorders.

CAR-Treg Platform

Tregs are a subset of immune cells with the ability to control and reduce the body’s immune reactions. They play a crucial role in maintaining immune homeostasis (keeping the immune system balanced) and preventing undesirable immune reactions.