Drug Rescue

Through our CardioSafe 3D and LiverSafe 3D drug rescue programs, we believe we can recapture substantial value from the prior investment by pharmaceutical companies and others who have developed and established the efficacy potential of new drug candidates that have been terminated prior to FDA approval due to heart or liver safety concerns. Using our hPSC technology, together with medicinal chemistry, we are focused on generating new, proprietary, small molecule variants (Drug Rescue Variants) of once-promising drug candidates for our internal development. We believe focusing on drug rescue candidates previously optimized for efficacy may give us a valuable “head start advantage” in our efforts to produce new, proprietary Drug Rescue Variants faster and less expensively than the drug rescue candidates discovered and developed previously using only conventional live animal models and in vitro cellular assays.

The initial goal of each drug rescue program will be to produce, with our medicinal chemistry collaborator, a portfolio of Drug Rescue Variants of the once-promising but discontinued drug candidate. We will then use our CardioSafe 3D and/or LiverSafe 3D, assay systems to identify the lead Drug Rescue Variant in the portfolio that demonstrates an improved therapeutic index compared to the original drug candidate (that is, equal or improved efficacy with reduced toxicity or metabolism issues). We will then validate that the lead Drug Rescue Variant demonstrates reduced toxicity and/or metabolism issues in both our proprietary assay systems and in the in vitro and in vivo preclinical testing models that the pharmaceutical company used to determine efficacy and safety for the original blocked drug candidate.

Strategic Partners

Successful partnerships are critical to the achievement of our strategic objectives. Developing and nurturing long term research and business collaborations have enabled us to strengthen our core competencies and develop new skills as the stem cell industry continues to grow at an exciting pace. We seek strategic collaborations wherever there is a tight fit between our hPSC platform and the potential for leveraging the capabilities and technologies of partners to enable joint success. We are open to diverse collaboration models framed around mutually beneficial commercial and research and development opportunities.

We seek to partner proactively with like-minded research teams and companies, with the goal of creating a vibrant, effective and innovative drug development and drug recue ecosystem to drive our programs towards new therapeutic modalities, cost effectively and efficiently. We welcome strategic partnering requests, technology licensing offers or other collaborative proposals from the research and business communities at large.

Cato Research is a global contract research organization (CRO) that offers a wide range of integrated services to pharmaceutical, biotechnology, and medical device companies. They specialize in complex development programs requiring innovative regulatory and clinical strategies, and have experience implementing and conducting successful clinical trials and development programs. Cato Research provides us regulatory and drug development expertise for our AV-101 development programs and also acts as a source of potential drug rescue candidates and strategic collaborations.

The Centre for Commercialization of Regenerative Medicine (CCRM) is a Canadian, not-for-profit organization supporting the development of foundational technologies that accelerate the commercialization of stem cell- and biomaterials-based products and therapies. CCRM is supported by the Centres of Excellence for Commercialization and Research (CECR) Program. CCRM brings together leading RM experts from the University of Toronto and McMaster University, with researchers from the Hospital for Sick Children, the University Heath Network, the Ottawa Hospital Research Institute and Mount Sinai Hospital, to accelerate Regenerative Medicine (RM) research and development, and create a commercialization pipeline that rapidly brings RM technologies to market. These researchers are working to harness the power of stem cells, biomaterials and molecules through innovative technologies to treat, and perhaps cure, diseased cells, tissues and organs.

The California Institute for Regenerative Medicine is accelerating the development of new therapies for chronic disease and injury by funding stem cell research programs throughout California. The Institute was established in 2004 after Californians passed Proposition 71, the California Stem Cell Research and Cures Initiative. The statewide ballot measure, which provided $3 billion in funding for stem cell research at California universities and research institutions, called for the establishment of a new state agency to make grants and provide loans for stem cell research, research facilities and other vital research opportunities. The mission of CIRM is to support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease and injury.


ChanTest has developed the world’s most complete library of validated human ion channel-expressing cell lines to serve all the ion channel needs of its pharmaceutical and biotech customers. Services range from early functional screens for profiling drug candidates or ranking within profiles during the drug-discovery process – to a complete set of in vitro GLP service products for cardiac risk assessment. Since inception in 1998, the company has tested 12,000+ compounds for more than 250 global pharmaceutical and biotech companies, and its scientists have published 300+ papers in peer-reviewed journals.


The McEwen Centre for Regenerative Medicine’s mission is to be a catalyst for regenerative medicine by facilitating collaboration, supporting research, and promoting awareness of the field. The Centre includes 15 scientists at five Toronto hospitals, as well as the University of Toronto. Currently, these scientists are working to accelerate the development of more effective treatments for conditions such as heart disease, diabetes, respiratory disease and spinal cord injury. The McEwen Centre is supported by philanthropic contributions and research grants, and is based at University Health Network in Toronto, Canada. They also collaborate with many other research institutions throughout North America, Europe and the Asia/Pacific region.


Internationally regarded for its dedication to medical science, Mount Sinai is home to an array of leading research institutes, centers, and laboratories, all of which work toward rapidly translating advances in basic science into innovative patient care. With a history rich in clinical milestones and an unequaled passion for patient-focused research, Mount Sinai has pioneered breakthroughs benefiting untold numbers of lives.


National Jewish Health is known worldwide for treatment of patients with respiratory, cardiac, immune and related disorders, and for groundbreaking medical research. Founded in 1899 as a nonprofit hospital, National Jewish Health remains the only facility in the world dedicated exclusively to these disorders. Since 1998, U.S. News & World Report has ranked National Jewish Health the #1 respiratory hospital in the nation. NJH maintains a proactive approach to medicine, with its research focusing on environment-gene interaction and making strides in predicting, preventing, treating and tracking many diseases. Our research is truly transforming the lives of millions. By using an individual’s DNA to predict and prevent disease, to guide the right choice of therapy to limit side effects and bring higher success rates for treatments and to be able to track the effects of these therapies, National Jewish Health is working to improve healthcare for the future.


The largest source of funding for medical research in the world, National Institutes of Health (NIH) is made up of 27 institutes and centers. For over a century, NIH scientists have paved the way for important discoveries that improve health and save lives. The medical research agency has created hundreds of thousands of high-quality jobs by funding thousands of scientists in universities and research institutions in every state across America and around the globe. To date, VistaGen has been awarded over $8.5 million from NIH.


Synterys, Inc. is a San Francisco Bay Area medicinal chemistry and collaborative drug discovery provider focused on the needs of virtual and small drug discovery companies. They are headquartered in the US with fully modern overseas facilities in Taiwan. With an internal focus on high-quality synthetic organic medicinal chemistry, they are virtually integrated with outstanding computational chemistry, DMPK and manufacturing partners to provide all the tools necessary for the rapid advancement of discovery projects. The company’s highly experienced senior management team and staff have multiple years of experience in large pharmaceutical and small biotech companies and a proven track record of drug discovery success with numerous clinical candidates invented.


TET Systems was founded by the inventors of the Tet Technology and holds the Tet Technology IP portfolio. The Tet Technology provides efficient, precise and reversible control over both timing and level of gene expression in eukaryotic cells. The company has licensees from the pharmaceutical, biotechnology, plant sciences and other industries located on 4 continents (Asia, Australia, Europe and North America). They also have academic customers who market their inventions including the Tet Technology under a commercial license.


Providing care to the community for more than 200 years, UHN is a major landmark in Canada’s healthcare system and a teaching hospital of the University of Toronto. Building on the strengths and reputation of each of the three hospitals, Princess Margaret Hospital, Toronto General Hospital, and Toronto Western Hospital, UHN brings together the talent and resources needed to achieve global impact and provide exemplary patient care, research and education. Approximately 2,700 scientists, technical staff, students and trainees at University Health Network are working together to solve fundamental and applied research problems, with much of the research focused on four priority platforms: Genes, Proteins, and People; Medical Technology Innovation; Health Informatics; and Regenerative Medicine.


Since its founding in 1925, the Wisconsin Alumni Research Foundation (WARF) has served the University of Wisconsin-Madison scientific community by patenting the discoveries of UW-Madison researchers and licensing these technologies to leading companies in Wisconsin, the United States, and worldwide. They promote, encourage and aid scientific investigation and research at the University of Wisconsin-Madison and the Morgridge Institute for Research through helping commercialize the technology arising from that research for the benefit of humankind; and investing the proceeds of our commercialization efforts and use the added value from our investments to promote, encourage and aid further scientific investigation and research at and within the University of Wisconsin-Madison and the Morgridge Institute for Research.