Efficient, effective, targeted. Lentiviral systems for expressing genes in virtually any type of human cell in-vivo.

Effective therapies for chronic diseases including cancer, diabetes, and arthritis have proved elusive. All of these diseases have in common a genetic component, which can provide an opportunity for effective treatment. Interventions targeted at the damaged genetic pathways have improved efficacy and reduced side effects compared to older cytotoxic therapies. Targeting therapies to these damaged pathways is the new challenge in genetic medicine.

AGTII is developing lentiviral vector-based therapeutics to effect targeted treatment of pancreatic, prostate, and liver cancer. These therapeutics will have at least two advantages versus chemical entities. First, the AGTII therapeutics target two or more pathways simultaneously, effecting improved cell killing by addressing the multigenic nature of chronic diseases. Second, increased dose intensity of therapeutic molecules is achievable with gene vectors, further enhancing biologic effect

AGTII's therapeutic lentiviruses have critical properties for a successful therapeutic:

100% bioavailability

Efficient entry and integration into virtually any human cell type, dividing or non-dividing

Simultaneous expression of multiple genes to improve efficacy

Independent control of cargo genes from multiple specific promoters

No immune response

Long-term expression

AGTII's has programs of recent results:

Prostate CancerGo to in vivo experimental results
for prostate cancer therapeutic, AG1101

Pancreatic CancerGo to in vitro experimental results
for pancreatic cancer therapeutic, AG1102

Liver CancerGo to in vitro experimental results
for liver cancer therapeutic, AG1103

Breast Cancer

Broad In Vivo Expression from AGTII Vectors:

A key feature of AGTII’s lentiviral vector technology is the ability to deliver therapeutic genes to nearly all tissues in vivo.  Expression of enhanced Green Fluorescent Protein (eGFP) from AGTII vectors in vivo following intravenous (IV) administration was monitored in tissue sections by fluorescence microscopy in the images below.

Prostate—eGFP	Prostate—Phase Contrast
Kidney—eGFP	Kidney—Phase Contrast
Pancreas—eGFP	Pancreas—Phase Contrast
Heart—eGFP	Heart—Phase Contrast
Lung—eGFP	Lung—Phase Contrast

As the example tissue sections above show, AGTII vectors are capable of cargo gene expression throughout the body’s tissues following intravenous administration.

References:

Lai, Z. and Galvin, J. Safe Lentiviral Vectors for Targeted Delivery of Multiple Therapeutic Molecules. WIPO WO 2010/045659, 22 Apr 2010

AGTII, Unpublished Results 2009-2010