Our Technology Can be Broadly Applied
to the Development of Novel Human Therapeutics, Applications in Plant Agriculture and Laboratory Research Reagents

We are applying our ZFP TF and ZFN product development platform
in three major fields:

• We are developing ZFP Therapeutics for the direct regulation of therapeutic genes in patients and for applications in gene modification.
• We are using our gene regulation and gene modification technology to increase the production of protein pharmaceuticals.
• We are using our gene regulation and gene modification technology for applications in plant agriculture.

These commercial opportunities are based upon our ability to precisely regulate or modify any endogenous gene. An important advantage of this approach is that endogenous cannot be patented and therefore we are unencumbered by the increasing problem of patent restrictions that exist for the cDNA copies of many of these.

Human Therapeutics
ZFP Therapeutics™
ZFP TFs have the potential to be developed as pharmaceutical products to treat a broad spectrum of diseases through the regulation or modification of disease-related genes in patients.

We have active programs in the following areas:

• Diabetic Neuropathy: Sangamo's lead therapeutic program uses a ZFP TF that upregulates the VEGF A gene to protect and regenerate nerves damaged by high blood glucose. Sangamo has four Phase 2 trials ongoing.
• Amyotrophic Lateral Sclerosis: We are developing our ZFP TF that upregulates the VEGF A gene for the treatment of this condition.

ZFP-Targeted Gene Modification
ZFP-restriction enzymes or ZFNs may be used to facilitate correction or disruption of the DNA sequence of a gene that has a disease–related mutation.

We have active preclinical programs in the following areas:

• HIV/AIDS: We are developing ZFNs for the disruption of CCR5 in T-cells. This is designed to provide HIV infected individuals with a reservoir of immunce cells that cannot be infected with the virus and thus are available to fight opportunistic infections.
• Cancer (Glioblastoma multiforme): We are developing ZFNs for the disruption of the glucocorticoid receptor in engineered CD8+ T-cells. .

We have active research programs in the following areas:

• X-linked SCID:(X-linked severe combined immunodeficiency or Bubble Boy Disease)
• Wiskott-Aldrich Syndrome:
• Hemoglobinopathies: such as sickle cell anemia and beta-thalassemia
  

ZFP-Targeted Gene Disruption
ZFP-restriction enzymes or ZFNs may be used to modify or disrupt of the DNA sequence of a gene that has a disease–related mutation.

Enabling Technology Applications
ZFP TF-Engineered Cell Lines for the Manufacturing
of Protein Pharmaceuticals
ZFP TF and ZFN-engineered cell lines can be developed to enhance production yields of protein pharmaceuticals and monoclonal antibodies.

Our partners in this area have included:

• Genentech
• Pfizer
• Amgen
• Novartis
• Medarex
• Novo Nordisk

Agricultural Biotechnology
ZFP TFs can be used to regulate genes and ZFNs to modifiy genes in plants, leading to potential applications in the development of new crops for optimized yield with enhanced nutritional properties.

Our partner in this area is Dow AgroSciences

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