American Gene Technologies (AGT) practices the art and science of genetic medicine. Our broad knowledge of lentivirus vectors and substantial toolbox allows for rapid drug development, testing and screening to identify clinical candidates. Our priority areas for clinical drug development include:
- A Functional Cure for HIV disease that combines immunotherapy with lentivirus delivery of genetic medicine to reconstitute effective and natural immune control of this dangerous virus;
- A novel strategy for curing phenylketonuria (PKU) – the second most common genetic disease in the U.S. and Europe; and,
- Innovative approaches to immuno-oncology that use genetic medicine to modify tumors so these sites of disease become potent stimulators of the immune system causing tumor destruction, removing the genetic modification and avoiding long-term consequences of altered T cells
The HIV Functional Cure program is on track for an Investigational New Drug (IND) application submission to the FDA in mid-2018 that will quickly be followed by the start of AGT’s first clinical trial. AGT overcame a longstanding obstacle in its progress toward a cure for PKU and is now selecting the best vector candidates from among many strong lentivirus configurations to gear up for steps toward a future clinical trial. The immune-oncology program already has strong early indicators of success from animal testing and we are screening for the best candidate genetic medicine to take forward in a plan for treating hepatocellular (liver) cancer.
AGT’s robust programs of discovery and drug development also include efforts on research and development of genetic medicines for treating Parkinson’s disease and familial dysautonomia – a rare, inherited orphan disease that is characterized by early onset blindness and other neurological problems. As priority programs move toward clinical testing, and as AGT continues to grow, these and other areas of investigation will expand and refine the genetic medicine strategies while advancing toward the clinic alongside other focus areas.
In addition to research and development on specific diseases, AGT is very active in developing new technologies to improving lentivirus vectors and genetic medicines. Our Transient VectorTM is delivered in a lentivirus particle but creates a non-integrated form that can be amplified to increase the number of gene copies without integration and enable degradation after treatment. Other discoveries are improving lentivirus vectors to target specific cells or tissue, increasing the efficiency of gene delivery and testing the boundaries for creating complex genetic medicines to approach complex human diseases.