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Our Technology Can be Broadly Applied
to the Development of Novel Human Therapeutics, Enabling Technology Applications and to Plant Agriculture
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
correction.
- 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 of disease-related
in patients.
We have active programs in the following areas:
- Diabetic Neuropathy: Sangamo's lead therapeutic program uses ZFP TFs that upregulate the VEGF A gene to protect and regenerate nerves damaged by high blood glucose.
- Cardiovascular and Peripheral Vascular Disease: A program partnered with Edwards Lifesciences Corp using ZFP TFs that upregulate the VEGF A gene in ischemic cardiovascular and vascular disease.
- Congestive Heart Failure: We are developing
ZFP TFs that down regulate the expression of Phospholamban for the treatment
of this condition.
- Age-related macular degeneration: We are devleoping ZFP TF activators of PEDF(pigment epithelium-derived factor).
- Cancer: We are developing a ZFP TF-Armed
Virus for the treatment of metastatic cancer.
- Intractable Neuropathic Pain: We are
developing ZFP TFs as a novel treatment for intractable neuropathic
pain.
ZFP-Targeted Gene Correction
ZFP-restriction enzymes or ZFNs may be used to facilitate repair or correction
of the DNA sequence of a gene that has a diseaserelated mutation.
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 diseaserelated mutation.
We have active research 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.
Enabling Technology Applications
ZFP TF-Engineered
Cell Lines for the Manufacturing
of Protein Pharmaceuticals
ZFP TF-engineered cell lines can be developed to enhance production yields
of protein pharmaceuticals and monoclonal antibodies.
Our partners in this area have included:
- Medarex
- Pfizer
- Novartis
- Amgen
- Novo Nordisk
Agricultural Biotechnology
ZFP TFs can be used to regulate in plants, leading to potential applications
in the development of new crops for optimized yield with enhanced nutritional
properties.
Our partners in this area have included:
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