ASGCT Presentations Include Preclinical Data for TN-401 PKP2 and DWORF Gene Therapy Programs and AAV Capsid Engineering Innovations
Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a biotechnology company with a mission to discover, develop and deliver curative therapies that address the underlying causes of heart disease, announced that it will present preclinical data for its TN-401 and DWORF gene therapy programs, as well as data on its capsid engineering capabilities at the upcoming American Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting taking place May 16–19, 2022, in Washington, D.C. and virtually.
TN-401 is Tenaya’s gene therapy candidate being developed for the potential treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations. Mutations of the PKP2 gene are the leading genetic cause of ARVC and can result in severe disease, including significant arrhythmia and sudden cardiac death in adults and children. These mutations are estimated to affect more than 70,000 patients in the U.S. alone. Data being presented at ASGCT will detail the impact of a single dose of PKP2 gene therapy on arrhythmias, disease progression and survival in a Pkp2-deficient mouse model of ARVC. Tenaya will support the establishment of a global natural history study of ARVC caused by PKP2 mutations in 2022 and expects to submit an Investigational New Drug application (IND) to the U.S. Food and Drug Administration for TN-401 in 2023.
The company’s DWORF gene therapy is being developed for the potential treatment of genetic dilated cardiomyopathy (DCM). DWORF is a muscle specific micro-peptide first discovered by Tenaya co-founder Eric Olson, Ph.D. that acts on the SERCA pathway, which is widely considered to be a promising target in heart failure. Data to be shared at ASGCT will review initial tolerability and efficacy of adeno-associated viral (AAV) delivered DWORF in a DCM mouse model. Tenaya’s DWORF program is currently at candidate selection stage.
Tenaya believes its capsid engineering efforts will ultimately help support successful clinical development of its product candidates by enhancing the efficacy and safety of gene therapies. Data being presented for Tenaya’s capsid engineering efforts will showcase the work being done to enhance the specificity and expression of genes delivered to cardiomyocytes using novel capsids.
Details of the presentations are as follows:
Tuesday, May 17, 2022
5:30 p.m. – 6 :30 p.m. ET
Cardiovascular and Pulmonary Diseases session
Poster Presentation: Cardiac AAV: PKP2 Gene Therapy Reduces Ventricular Arrhythmias, Reverses Adverse Right Ventricular Remodeling, Improves Heart Function, and Extends Survival in a Pkp2-Deficient Mouse Model of Arrhythmogenic Right Ventricular Cardiomyopathy (Abstract 630)
Lead Author: Jane Yang, Ph.D., Senior Scientist, Tenaya Therapeutics
Poster Presentation: Developing a DWORF Micropeptide Gene Therapy for Heart Failure (Abstract 622)
Lead Author: Huanyu Zhou, Ph.D., Senior Scientist, Tenaya Therapeutics
Poster Presentation: Engineering Novel AAV Capsids for Cardiac Gene Delivery (Abstract 626)
Lead Author: Ze Cheng, Ph.D., Scientist II, Tenaya Therapeutics
To view full event programming, please visit the ASGCT 25th Annual Meeting website.
About Tenaya Therapeutics
Tenaya Therapeutics is a biotechnology company committed to a bold mission: to discover, develop and deliver curative therapies that address the underlying drivers of heart disease. Founded by leading cardiovascular scientists from Gladstone Institutes and the University of Texas Southwestern Medical Center, Tenaya is developing therapies for rare genetic disorders as well as for more prevalent heart conditions through three distinct but interrelated product platforms: Gene Therapy, Cellular Regeneration and Precision Medicine. For more information, visit www.tenayatherapeutics.com.
This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely historical are forward-looking statements. Words such as “will”, “potential,” “expects” and “believes,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, Tenaya’s plans to present preclinical data on its TN-401 and DWORF gene therapy programs at the ASGCT 25th Annual Meeting; the therapeutic potential of TN-401 as a treatment for ARVC caused by PKP2 gene mutations; Tenaya’s plans to support the establishment of a global natural history study of ARVC caused by PKP2 mutations and expectations regarding the timing of the IND filing for TN-401; the therapeutic potential of Tenaya’s DWORF gene therapy program as a treatment for DCM; and Tenaya’s belief that its capsid engineering efforts will enhance the efficacy and safety of gene therapies. The forward-looking statements contained herein are based upon Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including but not limited to: the availability of data at the referenced times; risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early stage company; Tenaya’s ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; the timing, progress and results of preclinical studies for TN-401, DWORF and Tenaya’s other programs; Tenaya’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Tenaya’s manufacturing and operations, including preclinical studies and planned clinical trials; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Tenaya’s manufacturing, commercialization and marketing capabilities and strategy; the loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s reliance on third parties; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya assumes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.