Tenaya Therapeutics Showcases Breakthroughs in Gene Therapy Research

Tenaya Therapeutics Showcases Breakthroughs in Gene Therapy Research

Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a pioneering biotechnology firm located in South San Francisco, is on a mission to revolutionize the treatment of heart disease. The company specializes in discovering, developing, and delivering potentially curative therapies aimed at addressing the root causes of cardiovascular issues.

Recently, Tenaya announced its participation in the upcoming American Society of Gene and Cell Therapy (ASGCT) 28th Annual Meeting. During this renowned event, Tenaya will present five compelling abstracts reflecting its groundbreaking research initiatives.

Abstract Presentations at ASGCT

The abstracts to be featured are a result of Tenaya's commitment to advancing its core competencies in areas like capsid engineering, as well as the identification and optimization of genetic medicines targeting cardiomyocytes. Furthermore, the company is enhancing its manufacturing processes for adeno-associated virus (AAV) gene therapies.

Refining Capsid Engineering Capabilities

One of the abstracts focuses on refining capsid engineering capabilities through high-throughput experimental screening. By leveraging both in vivo and in silico screening methods, Tenaya researchers have efficiently identified several novel capsid candidates. These capsids were tested against AAV serotype 9 (AAV9)—which is known for its effective cardiomyocyte targeting—and some candidates even outperformed AAV9 in murine models, significantly advancing cardiac gene therapy.

Innovations in Prime Editing

Another significant highlight will be Tenaya's development of prime editing techniques specifically aimed at cardiomyocytes. Researchers created a prototype that utilizes a dual-cassette system delivered via two AAV capsids, incorporating cardiomyocyte-specific regulatory elements. In preclinical studies, this prime editing prototype successfully corrected mutations in a murine model of RBM20-related dilated cardiomyopathy (DCM), paving the way for promising therapeutic applications.

Development of TN-501 Gene Editing Therapy

Tenaya will also present outcomes related to its TN-501 gene editing candidate. Aimed at treating PLN-R14del-associated DCM, TN-501 is engineered to specifically target and inactivate the mutant phospholamban allele. Preclinical trials demonstrated that low doses of TN-501 markedly improved cardiac function and reduced fibrosis while being well-tolerated in murine models, underscoring its transformative potential in heart disease treatment.

Advancements in Manufacturing Techniques

The company is also proud of its achievements in establishing scalable manufacturing processes. Tenaya successfully implemented Sf9/rBV-based production at a large scale for its AAV gene therapy programs, while also developing a novel HEK293-based manufacturing process that promises enhanced yields and cost-efficiency. This innovation represents a significant step towards creating affordable AAV gene therapies that can reach a wider patient population.

Event Details

The ASGCT 2025 will take place from May 13 to May 17. Key presentations from Tenaya include:

Key Abstracts

On May 13, during the Poster Abstract Session:

• Abstract #523
  Title: A Humanized RBM20 Mouse Model Exhibits Dilated Cardiomyopathy Phenotypes and Enables Development of In Vivo Prime Editing for Treating Human RBM20 Cardiomyopathy Patients
  Presenting Author: Wenjing Liang, Ph.D.
• Abstract #952
  Title: High Productivity HEK293 AAV Production Platform Enabled by Novel Transfection Reagents
  Presenting Author: Charles Feathers

On May 14, the company will also present:
Abstract #1022
Title: TN-501 Gene Editing Therapy for PLN-R14del-Associated Cardiomyopathy
Presenting Author: Huanyu Zhou, Ph.D.

• Abstract #1028
  Title: Developing In Vivo Prime Editing as a Potential Treatment Option for Heart Disease
  Presenting Author: Lindsey Rollosson
• Abstract #1396
  Title: Engineering Novel AAV Capsids for Cardiac Gene Delivery
  Presenting Author: Ze Cheng, Ph.D.

About Tenaya Therapeutics

Tenaya is dedicated to addressing heart disease through genetic medicine. Its pipeline features a range of innovative therapies, including TN-201 for MYBPC3-associated hypertrophic cardiomyopathy, TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy, and other promising early-stage programs. The company's goal is to make a lasting difference in the lives of patients dealing with rare genetic disorders and prevalent heart conditions.

For more insights into Tenaya’s ongoing research and developments, their website offers comprehensive information.

Frequently Asked Questions

What is Tenaya Therapeutics' mission?

Tenaya Therapeutics aims to discover and develop transformative therapies targeting the underlying causes of heart disease.

Where will Tenaya present their research?

Tenaya will present its research at the ASGCT 28th Annual Meeting in New Orleans.

What is the significance of Tenaya's gene editing therapy?

Tenaya's gene editing therapy aims to provide specific treatment options for genetic heart conditions, potentially reversing disease mechanisms.

How does Tenaya enhance its manufacturing processes?

By implementing novel technologies and scalable methods, Tenaya improves yield and reduces costs for AAV-based therapies.

What programs are in Tenaya's pipeline?

Tenaya's pipeline includes gene therapies for cardiomyopathies and heart failure, showcasing various innovative treatment approaches.

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