Our Science
The future of heart disease treatment is bold.
As an emerging leader in gene therapy for inherited heart conditions, we are advancing a pipeline that targets the root causes of disease—not just the symptoms. These are not incremental steps. They represent a paradigm shift in how we think about treating—and ultimately attempting to cure —cardiomyopathies. And they are made possible by the deep scientific expertise, relentless innovation, and unwavering commitment of our team and collaborators.
Drug Development Capabilities
Early on in Tenaya’s company history, we invested in proprietary integrated capabilities to enable modality agnostic target identification and validation anchored in human genetics and the use of human disease models. Our distinct suite of integrated capabilities broadly enable target identification and validation, design of AAV-based genetic medicines and in-house manufacturing to support our efforts to discover and develop disease-modifying treatments focused on heart disease.
Our long-term goal is to build a leading fully integrated biopharmaceutical company focused on precision medicines for heart disease including, but not restricted to, gene replacement therapy, gene editing, small molecules, gene silencing, and regenerative medicine approaches We have invested in our interrelated capabilities, including the use of human-induced Pluripotent Stem Cell (iPSC) and engineered heart tissue disease models, machine learning and phenotypic screening, capsid engineering and novel promoter constructs, to enable the discovery, design, delivery and development of therapeutics that are best suited to a given cardiovascular condition.
Disease Models
We have internalized the ability to create and integrate proprietary in vitro and in vivo models within our research organization. For our in vitro hiPSC-CM disease models, we use multiple methods to induce phenotypes within cell lines that simulate human diseases and then use these models for high throughput target identification and drug discovery. For our in vivo disease models, we developed three-dimensional engineered heart tissues which allow us to recapitulate contractile function. We have established rodent heart disease models, both genetic and non-genetic, and can dose animals, perform heart surgeries, and use non-invasive imaging to assess the impact of our therapies under development.
Capsid Engineering
We have established in-house adeno-associated virus (AAV) capsid engineering capabilities and have successfully screened over one billion variants from more than 30 diverse, proprietary AAV libraries in multiple in vitro, in vivo and in silico models to discover novel AAV capsids that can target the different types of cells in the heart. These capsids are designed, and have shown in preclinical studies, to have desirable properties including the ability to more selectively target the heart versus other organs as well as lower susceptibility to neutralizing antibodies. We believe our capsid engineering efforts will be critical in supporting the successful clinical development of our product candidates and enabling those product candidates, if approved, to reach more patients.
Promoters and Regulatory Elements
We have created novel promoters and regulatory elements to support our gene therapy and cellular regeneration programs. We use these innovations to help ensure more precise and more robust expression of therapeutic payloads in the different cell types of the heart as compared to what can be achieved with currently available methods. We believe our innovations can support successful clinical development in part by improving the efficacy and safety profile of our product candidates.
Drug Delivery
We are actively exploring different routes of administration (ROAs) as well as different infusion- and injection-based methods for delivering our AAV-based therapies. We have designed a new catheter to support more targeted delivery and more efficient uptake of therapeutic payloads in the heart.
Manufacturing
For the production of our lead gene therapy candidates, we maintained complete ownership of process development, analytical development, and quality control and have already produced all necessary clinical trial material needed for our ongoing clinical trials at our Good Manufacturing Practice (cGMP)-certified Genetic Medicines Manufacturing Center in Union City, California.
Collaborations
Tenaya’s mission is to discover, develop, and deliver therapies that address the underlying drivers of heart disease. We were founded by leading cardiovascular scientists from, and have established academic collaborations with, Gladstone Institutes and University of Texas Southwestern Medical Center, and have forged additional partnerships aimed at helping advance our groundbreaking therapies. We are always open to strategic collaborations to push new boundaries in the treatment of heart disease.
For partnering inquiries, please contact us at partnering@tenayathera.com.
