Faraz Ali, MBA, on Continued Innovation With Cardiovascular-Targeted AAV Gene Therapy


The chief executive officer of Tenaya Therapeutics discussed the company’s research on capsids, promoters, and manufacturing improvements.

“We are committed to platform enhancements that will enable the field of precision genetic medicines for cardiovascular disease...”

Tenaya Therapeutics is currently developing 2 clinical-stage products: TN-201, an investigational adeno-associated virus (AAV)-vector based gene therapy intended to treat hypertrophic cardiomyopathy caused by mutations in the MYBPC3 gene and TN-401, an investigational AAV-based gene therapy intended to treat arrhythmogenic right ventricular cardiomyopathy (ARVC) via delivery of a functional copy of plakophilin-2 (PKP2), the disease-targeted gene in approximately 40% of patients with ARVC.1,2 In addition to these therapeutic candidates, however, the company is also working on research pertinent to the development of future genomic medicines for the treatment of cardiovascular diseases. This work includes research aimed at selection of better AAV capsids for gene therapy delivery, smaller and more efficient promoters, improvements to manufacturing processes, and the development of a gene editing approach. This supplemental research was the focus of the company’s 7 poster presentations at the American Society of Gene & Cell Therapy (ASGCT) 27th Annual Meeting, held May 7 to 10, 2024, in Baltimore, MD.

In an interview with CGTLive® at the conference, Faraz Ali, MBA, the chief executive officer of Tenaya Therapeutics, spoke about these research efforts and how they might help push the field of cardiovascular gene therapy further. He pointed out that AAV9, the capsid used in both TN-201 and TN-401, is currently viewed as the ideal capsid choice for targeting cardiomyocytes, but that the company is working on capsid engineering efforts to potentially find a better option for next generation therapeutic candidates. Ali also discussed the company’s work on developing stronger and smaller promoters to improve the genetic carrying capacity of AAV capsids and the company’s work on manufacturing processes intended to increase the scale and lower the costs of producing gene therapy products. He also briefly touched on the company’s research on gene editing approaches for a rare genetic cardiomyopathy.

Click here to view more coverage of the 2024 ASGCT Annual Meeting.

1. Tenaya Therapeutics announces TN-201 IND clearance and anticipated 2023 milestones. News release. Tenaya Therapeutics, Inc. January 9, 2023. Accessed May 14, 2024. https://investors.tenayatherapeutics.com/news-releases/news-release-details/tenaya-therapeutics-announces-tn-201-ind-clearance-and
2. Tenaya Therapeutics receives orphan drug designation from the U.S. Food and Drug Administration for its gene therapy for genetic arrhythmogenic right ventricular cardiomyopathy. News release. Tenaya Therapeutics. November 28, 2022. Accessed May 14, 2024. https://investors.tenayatherapeutics.com/news-releases/news-release-details/tenaya-therapeutics-receives-orphan-drug-designation-us-food-and

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