About Us

Located in South San Francisco, VistaGen is a clinical-stage biopharmaceutical company committed to developing and commercializing innovative product candidates for patients with depression, cancer, other diseases involving the central nervous system (CNS), as well as certain neurodegenerative diseases.

AV-101 (L-4-chlorokynurenine or 4-Cl-KYN)

VistaGen’s lead program, AV-101, is currently undergoing a National Institutes of Health (NIH) sponsored Phase 2 study for the treatment of major depressive disorder (MDD), an area of growing global concern. According to The World Health Organization, depression is the leading cause of disability worldwide, and is a major contributor to the global burden of disease, affecting 350 million people worldwide. VistaGen’s AV-101 holds the promise of providing the therapeutic anti-depressant benefits of ketamine, a classic drug whose efficacy has catalyzed the development of a new generation of drugs for MDD, without its limiting side effects.

Beyond MDD, VistaGen believes that AV-101 has potential utility in additional CNS indications, including neuropathic pain and epilepsy, and in neurodegenerative diseases such as Parkinson’s disease and Huntington’s disease. 

Stem Cell Technology and Drug Rescue

With the mature, functional human heart and liver cells produced using its pluripotent stem cell technology, VistaGen has developed two novel, customized human cellular bioassay systems, CardioSafe 3D and LiverSafe 3D, to predict possible heart and liver toxicity of new drug candidates in vitro, long before they are ever tested in animal or human studies. CardioSafe 3D and LiverSafe 3D are also being used to advance the Company’s internal drug rescue programs, focused on producing new chemical entities (NCEs). Such NCEs are intended to be novel, safer variants of drug candidates previously optimized and tested for efficacy by pharmaceutical companies and others but terminated before FDA approval due to unexpected heart or liver toxicity concerns. The Company’s initial drug rescue programs are focused on NCEs for the treatment of cancer.

Stem Cell Regenerative Medicine

VistaGen believes that stem cell technology-based regenerative medicine has the potential to transform the U.S. healthcare system over the next two decades by altering the fundamental mechanisms of disease, and help slow rapidly rising healthcare costs. The Company plans to explore nonclinical opportunities to apply its stem cell technology platform to develop novel human disease models for discovery of small molecule drugs and biologics with regenerative and therapeutic potential. VistaGen’s key objectives are to explore regenerative medicine opportunities through pilot, nonclinical proof-of-concept studies, after which the Company intends to assess any potential opportunities for further development and commercialization of therapeutically and commercially promising regenerative medicine programs, either on its own or with strategic partners.