Sangamo Announces AAV Capsid Data

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

We believe that genomic medicines hold the key to transforming the treatment landscape for severe diseases.

Our mission is to shift the focus from temporary symptom relief to the development of lasting cures. By harnessing the potential of genomic medicine, we aim to create a brighter future for patients and their families.

Technology Platforms

Using our highly versatile technology platforms we are designing and developing genomic medicines to potentially transform the lives of patients suffering from severe diseases.

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.

Learn more about our zinc finger platform

Zinc Finger Technology

Delivery Platform

Depending on the therapeutic application, these technologies can be deployed either on human cells outside the body (ex vivo) or directly on cells and tissues inside the body (in vivo).

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).

Learn more about our delivery platform

ZF technology is used to create genomic medicines

CAR-Treg Platform

Sangamo is aiming to pioneer the next generation of cell therapy using regulatory T cells, or Tregs. Our vision is to develop Treg therapies with the potential for long-term remission for major autoimmune diseases.

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.

Learn more about our CAR-Treg platform

Therapeutic Applications

Using our highly versatile zinc finger and delivery platforms, we aim to develop genomic medicines to transform the lives of patients suffering from severe diseases. Our technology can be utilized to potentially address a wide range of therapeutic applications. Our primary focus areas are neurology, autoimmunity and transplantation and oncology.

Clinical programs

Today, Sangamo is in the clinic with gene therapy in hemophilia A and Fabry disease, and autologous cell therapy in renal transplant.

View our pipeline
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