Tenaya Therapeutics (TNYA) investor relations material

Key trends and industry outlook

• Cardiac gene therapy is advancing with proof-of-concept data, despite some safety setbacks and regulatory scrutiny, but momentum is building due to supportive regulatory commentary and precision medicine trends.

• The field is seeing a shift toward both rare and prevalent cardiac diseases, with companies leveraging novel capsids, local delivery, and improved manufacturing to address safety, efficacy, and scalability.

• Regulatory agencies, especially the FDA, are increasingly open to biomarker-based and surrogate endpoints for accelerated approvals, particularly in high unmet need areas.

• Commercialization is a growing focus, with the need for successful launches, payer engagement, and market uptake now seen as the next major hurdle for gene therapy.

• The innovation cycle in gene therapy is peaking again, with clinical maturity and new products expected to drive renewed investment and industry growth.

Clinical and technical advances

• Companies are developing novel AAV capsids and proprietary plasmid systems to improve cardiac cell targeting, reduce required doses, and minimize off-target toxicity.

• Lead programs target diseases like BAG3 dilated cardiomyopathy, Friedreich’s ataxia, and arrhythmogenic cardiomyopathy, with early clinical and preclinical data showing promising efficacy and safety.

• Non-invasive imaging, cardiac biomarkers, and functional endpoints are central to trial design, enabling rapid assessment of therapeutic impact and supporting regulatory submissions.

• Manufacturing improvements, such as higher yields and lower cost of goods, are making large-scale and prevalent disease indications more commercially viable.

• Local delivery approaches and lower dosing strategies are reducing the need for immunosuppression and improving patient safety profiles.

Commercial and regulatory considerations

• The field is moving beyond clinical proof to focus on commercial readiness, including manufacturing scale, payer engagement, and precision medicine implementation in cardiology.

• Advances in genetic testing and the introduction of precision therapies are expected to transform the cardiovascular treatment landscape, mirroring trends in oncology.

• Regulatory flexibility remains strong, with agencies supporting innovative endpoints and trial designs, especially in rare diseases.

• Portfolio strategies balance rare and common disease programs, with large partners enabling global trials and broader market access.

• Industry sentiment is improving, with valuations rebounding and optimism about upcoming clinical readouts and approvals.