**Tenaya Therapeutics (TNYA)** Tenaya Therapeutics, is a research-oriented biopharmaceutical company that is focused on developing and producing new therapeutic drugs for the treatment of heart disease. Tenaya is targeting its approach on the underlying causes of heart disease, including rare genetic disorders. The company’s approach includes [[gene therapies]], [[cellular regeneration]], and [[precision medicines]]. Heart disease is the world’s leading cause of death among adults, making its treatment an important niche. Tenaya currently has two primary drug candidates under investigation, TN-201 and TN-401, for the treatment of MYBPC3-associated hypertrophic cardiomyopathy and PKP2-associated arrhythmogenic right ventricular cardiomyopathy, respectively The leading candidate, TN-201, is currently undergoing a Phase 1b human clinical trial. The study is focused on safety and tolerability and will enroll up to 24 adults. Data from the first patient cohort in the study are expected to be released in December, presenting a significant milestone for the stock. Meanwhile, enrollment is ongoing for the second cohort. ### Drug Development Capabilities We have internalized and integrated core capabilities to support our product platforms and our pipeline programs. We believe these capabilities can collectively support more rapid product development, precise delivery of our therapies to the heart, and more efficient and scalable manufacturing of our products. Taken together, these capabilities may ultimately improve the probability of technical and regulatory success of our product candidates that we hope to bring to patients who are fighting different forms of heart disease.  ##### 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 have a dedicated onsite _in vivo_ pharmacology group and vivarium, where we have established approximately 15 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](https://www.tenayatherapeutics.com/wp-content/uploads/ESGCT_2022_poster_Ze_Cheng_final.pdf), 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 We have established the Cardiac Genetic Medicines Manufacturing Center to support the production of our emerging portfolio of gene therapy and cellular regeneration product candidates. Tenaya’s state-of-the-art facility is designed to meet regulatory requirements for AAV-based gene therapies with initial capacity to produce current Good Manufacturing Practice (cGMP) drug product at the 1000L scale, utilizing Tenaya’s proprietary baculovirus-based production platform and suspension Sf9 cell culture system. The Center is located near our research labs in the San Francisco Bay Area to enable seamless collaboration across groups and is staffed by a growing in-house team with expertise in all aspects of gene therapy manufacturing, including process development (PD), analytical development (AD), quality assurance (QA) and quality control (QC). https://investors.tenayatherapeutics.com/static-files/a5220cc4-74c0-49f4-96a9-d8e892648ead