Simpler. Faster. Less Expensive.

American Gene Technologies® is creating the foundation for a fundamentally new approach to medicine with a gene therapy “cure platform” that harnesses the power of lentivirus vectors to cure diseases. Think of it like Apple’s iOS platform for the iPhone on which other app developers can build. With American Gene gene therapy platform, scientists and researchers will be able accelerate the path to real cures. 


  • The platform significantly cuts the time and costs of developing new drugs for a variety of diseases.
  • And it simplifies the process for all drug developers to create new life-saving therapies.

Amid a constantly changing competitive landscape, American Gene Technologies always finds ways to create tools and technologies that improve and accelerate the path to real cures.

Harnessing the Power of Lentivirus Vectors to Cure Diseases

American Gene Technologies 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 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 Cure program is currently in Phase 1 clinical trials. Details on the ongoing trial can be found here. American Gene Technologies 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 immuno-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.


American Gene Technologies’ 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 American Gene Technologies 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, American Gene Technologies is very active in developing new technologies to improve 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.