November 2010
AGTII receives funding from the Treasury Department through the Qualifying Therapeutic Development Tax Credit Program (QTDP). The QTDP was created by Congress in March 2010 as part of the Patient Protection and Affordable Care Act. It provides a tax credit or grant equal to 50% of eligible costs and expenses for tax years 2009 and 2010. The funding decision was based on the Company's promising therapeutic lentivirus technology and its anticipated impact on cancer in the United States.
Gene Therapy in the News
Recent successes in gene therapy are illustrating the long term potential of these new technologies to treat serious human disease:
Last year, researchers in Paris reported on a study in which the debilitating neurologic disease X-linked adrenoleukodystrophy (ALD), featured in the movie Lorenzo's Oil, was treated by replacing the defective ABCD1 gene in two patients. The researchers used a lentiviral vector to deliver a functional copy of the ABCD1 gene to the patient's own bone marrow cells, which were then replaced in the patient. At up to 2 years post-treatment there was evidence that the disease progression had slowed or stopped and that the gene-corrected cells had partially repopulated the patient's bone marrow.
Also in 2009, researchers at University of Washington treated the retinas of two colorblind Spider Monkeys with a gene therapy that introduced a gene to correct the genetic defect. The monkeys' eyes were injected with a virus carrying the gene for L-opsin, a protein that is responsible for the ability to detect red and green wavelengths. At first the monkeys did not appear to be able to select food rewards that required the ability to see red and green dots. After five months, however, the monkeys began to select the appropriate red and green dots repeatedly. The monkeys, which had never been able to see red and green, appear to have gained the ability to do so thanks to gene therapy. The study was reported in the journal, Nature, and also described in the online magazine, Wired.
Just this month scientists from the Harvard Medical School and the University of Paris described a gene therapy to correct beta-thalassemia in a young man. Beta-thalassemia is one of a group of diseases that eliminate hemoglobin production in red blood cells, leading to the need for repeated blood transfusions for survival. A lentiviral vector was used to introduce a functional copy of the beta globin gene into the patient's bone marrow cells, which were then injected back into the patient. In the three years since the treatment, tests of the young man's blood cells have revealed stable levels of the newly introduced gene. Expression of beta globin from the new gene has been demonstrated and the patient has been able to forego transfusions for two years. The report was published in the journal, Nature.
Company News
2010
AGTII tests a revised AG1102 Pancreatic Cancer Therapeutic in vitro with promising results. In addition, design work is completed on the Company's AG1103 Liver Cancer Therapeutic. Co-development/Licensing discussions for various indications were inititated with Chinese pharmaceutical companies.
2009
In vivo testing of AGTII AG1101 Prostate Cancer Therapeutic in mice yielded positive results. AGTII scientists completed construction of a novel virus design incorporating the p53 gene and a Bcl-2 siRNA gene in preparation for animal testing. Initial testing of AG1102 Pancreatic Cancer Therapeutic was completed. AGTII formed its Scientific Advisory Board, with the commitment of leading scientists in the United States and Europe.
2008
AGTII scientists designed and tested novel high-expressing multi-gene lentiviral vectors in vitro. Design features include multiple cargo genes, tissue and intracellular targeting elements, and siRNA genes. Multiple variations were successfully tested.
Intellectual property filings
Lai, Z. and Galvin, J. Safe Lentiviral Vectors for Targeted Delivery of Multiple Therapeutic Molecules. WIPO WO 2010/045659, 22 Apr 2010 Several additional patent filings have been completed. Important elements of these filings include:
High-expression, multiple-gene lentiviral vectors.
Tissue and enzyme targeting
Safety features addressing insertional mutagenesis and gene activation issues
Controlled-expression therapeutic protein delivery.
Prostate Cancer, Pancreatic Cancer, Liver Cancer Gene Therapy.
AGTII constructs corresponding to over 100 serious human diseases.
