Tenaya Therapeutics to Showcase Advancements in Gene Therapy at ASGCT 27th Annual Meeting

Tenaya Therapeutics, Inc. (NASDAQ: TNYA), based in South San Francisco, is a clinical-stage biotechnology firm dedicated to developing transformative therapies for heart disease. The company is set to present seven abstracts at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, scheduled for May 7-11, 2024, in Baltimore, MD. These presentations will spotlight Tenaya's expanding capabilities in genetic medicine focused on heart disease.

Tenaya has developed comprehensive internal capabilities for target validation agnostic to modality, and for designing and manufacturing adeno-associated virus (AAV)-based genetic medicines targeting heart diseases. The company's pipeline features two gene therapies in clinical stages for cardiomyopathies, alongside early-stage projects in gene therapy, gene editing, and cardiac cell regeneration, all employing AAV as a delivery mechanism. The ASGCT presentations will include recent advancements in capsid engineering, gene editing endeavors, and manufacturing process improvements aimed at boosting the safety and efficacy of AAV-based gene therapies.

Capsid Engineering and Promoters

Tenaya's capsid engineering initiatives involve identifying and evaluating both known and novel capsids. These are tested in various species to understand their transduction and expression within specific heart cells, as well as their ability to avoid liver targeting. At the ASGCT event, new data from studies on mice and non-human primates will be shared, comparing AAV9, AAVrh10, and AAVrh74 capsids for their cardiac cell tropism and transgene expression.

Two additional posters will provide insights into creating libraries of promoters for cardiac-specific gene expression and detailing efforts to design more compact promoters to accommodate larger gene therapy or gene editing treatments.

Gene Editing

Tenaya scientists will present preclinical findings on gene editing of the R14del variant of the phospholamban (PLN) gene, which could be a potential treatment for PLN-R14del-associated dilated cardiomyopathy. The company has developed a gene editing therapy using a single AAV vector that delivers a proprietary self-inactivating CRISPR-Cas9 system along with a PLN-R14del-specific single guide RNA. Earlier data demonstrated that this gene editing vector corrected the PLN-R14del mutation, preserving heart function, reducing fibrosis, and extending survival in a mouse model. The upcoming presentation will replicate these findings using self-inactivating vectors to minimize off-target edits without compromising efficacy.

Product Development and Manufacturing

Tenaya's clinical supply of TN-201 and TN-401 gene therapies was manufactured under Good Manufacturing Practice regulations at the company's Genetic Medicines Manufacturing Center using a proprietary Sf9 recombinant baculovirus (Sf9/rBV) production process. Abstracts related to enhancing yield and scalability of Sf9/rBV manufacturing processes will be presented at ASGCT.

The company has also advanced the HEK293 manufacturing platform to the 200L scale. Researchers will present data on a novel small molecule additive that boosts productivity in HEK293-based AAV manufacturing processes, potentially improving scalability, productivity, and cost-efficiency.

To explore the full event programming, visit the ASGCT 27th Annual Meeting website. After the conference, Tenaya’s presentations will be accessible in the "Our Science" section of the company's website.

About Tenaya Therapeutics

Tenaya Therapeutics is committed to discovering, developing, and delivering potentially curative therapies for heart disease. The company leverages integrated proprietary capabilities to identify and validate targets, design AAV-based genetic medicines, and conduct in-house manufacturing. Tenaya's pipeline includes TN-201 for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor initially developed for heart failure with preserved ejection fraction (HFpEF). The company also has multiple early-stage programs in preclinical development.

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