VistaGen Therapeutics, Inc. is a biotechnology company applying stem cell technology for drug rescue and cell therapy. Drug rescue combines human stem cell technology with modern medicinal chemistry to generate new chemical variants (drug rescue variants) of promising drug candidates that have been discontinued during preclinical development (put on the shelf) due to heart or liver safety concerns. We also focus on cell therapy, or regenerative medicine, which includes repairing, replacing or restoring damaged tissues or organs.
Our versatile stem cell technology platform,Human Clinical Trials in a Test Tube™, has been developed to provide clinically relevant indications, or predictions, of potential toxicity of new drug candidates before they are ever tested on humans. Our human pluripotent stem cell-based bioassays more closely approximate human biology than conventional animal studies and nonclinical in vitro techniques and technologies currently used in drug development.
Using mature human heart cells produced from pluripotent stem cells, we leveraged our Human Clinical Trials in a TestTube™ platform to develop CardioSafe 3D™, a three-dimensional (3D)bioassay system for predicting the invivo cardiac effects of new drug candidates before they are tested in humans. We now plan to leverage CardioSafe 3D™ to build a pipeline of new, safer, drug rescue variants of promising drug candidates that have been“put on the shelf” by pharmaceutical companies because of toxicity concerns,despite positive efficacy data signaling their potential therapeutic and commercial benefits.
Our lead drug candidate, AV-101, is in PhaseIb development in the U.S. for treatment of neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system. Neuropathic pain affects approximately 1.8 million people in the U.S. alone. To date, we have been awarded over $8.5 million from the U.S. National Institutes of Health (NIH) for development of AV-101.
We are also developing LiverSafe 3D™,a predictive liver toxicity and drug metabolism bioassay system, and are preparing to initiate pilot preclinical development of cell therapy programs focused on autologous bone marrow transplantation and heart, liver and cartilage repair. Each of these development programs is based on the proprietary human pluripotent stem cell differentiation and cell production capabilities of our Human Clinical Trials in a Test Tube™ platform.
Technology
Our Human Clinical Trials in a Test Tube™ platform is based upon a combination of proprietary and exclusively licensed stem cell technologies, including technologies developed over the last 20 years by renowned Canadian scientist, Dr. Gordon Keller, and Dr. Ralph Snodgrass, our founder, President and Chief Scientific Officer.
The proprietary and licensed technologies underlying our Human Clinical Trials in a Test Tube™ platform enable controlled differentiation of pluripotent stem cells into mature human cells specific to our drug rescue and cell therapy programs.
Controlled differentiation of human pluripotent stem cells into mature, functional human cells could allow the identification of new drug candidates that exhibit human toxicity early in the drug development process, resulting in efficient focusing of resources on those drug candidates with the highest probability of success. We believe this has the potential to substantially reduce drug development costs while producing effective and safer drugs.
The combination of our proprietary and licensed technologies enables the development of 2D and 3D human cell-based bioassay systems that consist of large numbers of normal, non-transformed, human cells that function as “micro-organs”.
The 3D micro-organ cultures induce the cells to grow, mature, and develop 3D cell networks and tissue structures. Compared to traditional flat 2D cell-based assays, these 3D cell networks and structures are believed to more accurately reflect the structures and biology inside the human body and are expected to yield responses to new drug candidates that are more clinically predictive of human drug responses.
We believe that our Human Clinical Trials in a Test Tube™ platform will allow the assessment of the toxicity profile of new drug candidates for a wide range of diseases and conditions with greater speed and precision than nonclinical in vitro techniques and technologies currently used by pharmaceutical companies in the drug development process.
Stem Cell Basics
Stem cells have the capacity to self-renew (divide and give rise to more stem cells) and differentiate or develop into mature, specialized cells that make up tissues and organs in the human body. Pluripotent stem cells can be grown indefinitely in vitro under proper conditions, and can be differentiated (developed) into any of the more than 200 specialized cell types in the human body. Due to these capabilities, they hold potential across a diverse range of medical research and therapeutic applications.
Embryonic stem cells (ES Cells) are pluripotent stem cells derived from excess embryos that develop from eggs that have been fertilized in an in vitro fertilization (IVF) clinic and then donated, with the informed consent of the donors, for research purposes after a clinically-approved IVF procedure. ES Cells are not derived from eggs fertilized in a woman’s body.
Recent developments in stem cell research have made it possible to obtain pluripotent stem cell lines from any individual without the use of embryos. Induced pluripotent stem cells (iPS Cells) are adult cells that have been genetically “reprogrammed” to behave like ES Cells by being forced to express genes necessary for maintaining the key pluripotential property of ES Cells.
Both ES Cells and iPS Cells have the unique capacity to be expanded in an undifferentiated state indefinitely and to differentiate into any cell in the body. We believe these features make them ideal research tools and a vital source of normal cell populations for creating human cell-based bioassays to test potential toxicity of new drug candidates and for cell therapy, or regenerative medicine, applications.
