PLN

Abstracts Demonstrate Tenaya’s Continued Innovation Across Core Competencies Facilitating Current Pipeline and Enabling Next Wave of Precision Heart MedicinesSOUTH SAN FRANCISCO, Calif., May 02, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced that it will present seven abstracts focused on the company’s growing capabilities for discovering and advancing genetic medicines for heart disease at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, being held May 7-11, 2024, in Baltimore, MD. Tenaya has established integrated internal capabilities to broadly enable modality agnostic target validation and the design and manufacture of adeno associated virus (AAV)-based genetic medicines focused on the treatment of heart diseases. The company’s pipeline includes two clinical-stage gene therapies for cardiomyopathies, as well as earlier-stage research related to gene therapy, gene editing and cardiac cell regeneration, all using adeno-associated virus (AAV) as a delivery vehicle. Tenaya’s seven presentations at this year’s ASGCT will highlight new capsid engineering insights, the company’s emerging gene editing efforts, and manufacturing process optimizations intended to enhance the safety and efficacy profiles of AAV-based gene therapies. Details of the presentations are as follows: Capsid Engineering and PromotersTenaya has established an active capsid engineering effort encompassing the identification and comparison of known and novel capsids, which are then tested across multiple species to characterize transduction and expression in specific heart cells and/or liver detargeting. At ASGCT, new data from murine and non-human primate studies will be presented comparing previously identified AAV-based capsids, AAV9, AAVrh10 and AAVrh74, for cardiac cell tropism and transgene expression. In two other posters, Tenaya scientists will present new data describing a novel approach to creating a library of promoters for cardiac-specific gene expression, as well as detailing efforts to design more compact cardiac-specific promoters to accommodate larger gene therapy or gene editing therapeutics. Wednesday Poster Session, May 8, 2024, at 12 pm ET AAV9, AAVrh.10, and AAVrh.74 Exhibit Different Cell Type Tropisms in the Heart Following Systemic Delivery and AAV9 Mediates Superior Cardiomyocyte Transgene Expression in Murine and NHP (abstract #2662) Lead author: Ze Cheng, Ph.D., Senior Scientist Chimeric and Rationally Designed Compact Promoters for Cardiac-Specific Gene Expression (abstract #464)AAV DNA shuffle library of GH Loop Variable Regions for Directed Evolution of Cardiotropic Capsids (abstract #482) Lead author: Prasad Konkalmatt, Associate Director, AAV Capsid Engineering Gene EditingTenaya scientists will present preclinical results suggesting that gene editing of the R14del variant of the phospholamban (PLN) gene may hold promise as a treatment for PLN-R14del-associated dilated cardiomyopathy. Tenaya has developed a gene editing therapy utilizing a single AAV vector designed to deliver a proprietary self-inactivating CRISPR-Cas9 and PLN- R14del-mutation-specific single guide RNA. Previously, Tenaya shared data from a mouse model showing that the company’s gene editing vector corrected the PLN-R14del mutation while leaving the wild-type PLN gene intact resulting in preserved heart function, reduced fibrosis and extended survival. This year’s presentation will replicate that set of data in the same model of disease using self-inactivating vectors designed to decrease the risk of off-target edits without any diminishment of efficacy. Friday Poster Session, May 10, 2024, at 12 pm ET Precision Editing of PLNR14del Mutation Using a Self-Inactivating, All-in-One AAV Vector to Rescue PLN-R14del-Associated Cardiomyopathy (abstract #1701) Lead author: Huanyu Zhou, Ph.D., Senior Scientist Product Development and Manufacturing Clinical supply of TN-201 and TN-401 gene therapies was manufactured under current Good Manufacturing Practice regulations at Tenaya’s Genetic Medicines Manufacturing Center using the company’s proprietary Sf9 recombinant baculovirus (Sf9/rBV) production process at the 1000L scale. At ASGCT, Tenaya researchers will present abstracts related to increasing yield and scalability associated with Sf9/rBV manufacturing processes. Tenaya has also internalized the HEK293 manufacturing platform up to the 200L scale. Tenaya researchers will present data on a novel small molecule additive that increases the productivity of HEK293-based AAV manufacturing processes, which may have implications for improved scalability, productivity and costs. Friday Poster Session, May 10, 2024, at 12 pm ET Development of a Scalable High Yield HEK293 Expression Platform for AAV Manufacturing (abstract #1531) Lead author: Charles Feathers, Process Development Manager Utilization of Tenaya’s AAV Productivity Boosting Small Molecule (SMB) for Intelligent Design of HEK293 Cell Line to Improve AAV Productivity (abstract #1530)Development of Highly Productive, Rhabdovirus-free Sf9 Insect Cell Line for Large-scale AAV Production for Cardiovascular Gene Therapies (abstract #1533) Lead author: Jackson Leong, Process Development Associate Scientist, To view full event programming, please visit the ASGCT 27th Annual Meeting website. Following the conference, Tenaya’s presentations will be available in the “Our Science” section of the company’s website. About Tenaya TherapeuticsTenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Leveraging integrated proprietary core capabilities enabling target identification and validation, design of AAV-based genetic medicines and in-house manufacturing the company is advancing a pipeline of novel therapies with diverse treatment modalities for rare genetic cardiovascular disorders and more prevalent heart conditions. Tenaya’s most advanced candidates include TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor being initially developed for heart failure with preserved ejection fraction (HFpEF). Tenaya also has multiple early-stage programs progressing through preclinical development. For more information, visit www.tenayatherapeutics.com. Forward-Looking StatementsThis 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,” “may,” “promise,” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, the presentation of data covering Tenaya’s capabilities for discovering and advancing genetic medicines; and the therapeutic and commercial potential of Tenaya’s capsid engineering, gene editing and manufacturing process optimizations efforts. 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: availability of data at the referenced times; risks associated with the process of discovering, developing and commercializing therapies that are safe and effective for use as human therapeutics; Tenaya’s ability to develop, initiate or complete preclinical studies and clinical trials, and obtain approvals, for any of its product candidates; Tenaya’s continuing compliance with applicable legal and regulatory requirements; Tenaya’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; Tenaya’s reliance on third parties; 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 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.Contact Michelle CorralVP, Corporate Communications and Investor RelationsTenaya TherapeuticsIR@TenayaThera.com InvestorsAnneMarie FieldsStern IR AnneMarie.Fields@SternIR.com MediaWendy RyanTen Bridge Communicationswendy@tenbridgecommunications.com

Next Generation Cardiovascular Biotech to Present non-clinical Data on Two of its Novel, Potent, and Highly Selective CaMKII Inhibitors First Company to Study a CaMKII Inhibitor in a Clinical Trial BURLINGTON, Mass.--(BUSINESS WIRE)-- Cardurion Pharmaceuticals, Inc. (“Cardurion” or “the Company”), a clinical-stage biotechnology company focused on the discovery and development of novel, next-generation therapeutics for the treatment of cardiovascular diseases, today announced that the Company will present preclinical data on its Calcium/Calmodulin-dependent Protein Kinase II (CaMKII) inhibitor program at the American Heart Association’s (AHA) Scientific Sessions 2023. Cardurion is the first company to evaluate a CaMKII inhibitor in clinical testing. The Company will present data on two of its highly selective and potent CaMKII inhibitors, CRD-2015 and CRD-2959, in a preclinical model of heart failure. Among the key data points: CRD-2015 and CRD-2959 both inhibit CaMKII-mediated phosphorylation of phospholamban and the ryanodine receptor, two wellknown myocardial CaMKII targets, in cells and in rats in vivo. Both CaMKII inhibitors prolonged survival in the calsequestrin transgenic heart failure model, which is characterized by a severe dilated cardiomyopathy and early mortality. The study was conducted by Cardurion’s cardiovascular research group in Shonan, Japan, led by Shota Ikeda, PhD. “These strong results demonstrate the successful creation of specific CaMKII inhibitors with preclinical efficacy and provide further support for the clinical development of our lead CaMKII inhibitors for the treatment of heart failure and other cardiovascular diseases,” said Howard K. Surks, M.D., Chief Medical and Scientific Officer of Cardurion. “These preclinical data underscore the enthusiasm that we have for the significant potential of this novel mechanism, and we look forward to making continued progress with human testing and studying select patient populations in the near term.” CaMKII has been a target of longstanding interest in cardiovascular drug development, based on the critical role of hyper-activated CaMKII in the pathogenesis of heart failure and several arrhythmic diseases, as demonstrated in multiple preclinical studies. Cardurion is currently conducting a Phase 1 clinical trial of its lead CaMKII inhibitor and is initiating a Phase 2 clinical trial for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare genetic arrhythmic disease. The Company next plans to expand this research into more common cardiovascular diseases, such as heart failure and atrial fibrillation. Details of the science session are as follows: Title: Novel, Potent, and Highly Selective Calcium/calmodulin-Dependent Protein Kinase II (CaMKII) Inhibitors Reduce Substrate Phosphorylation in Rat Hearts and Prolong Survival in a Mouse Model of Severe Heart Failure Session: Future of Lipid Lowering Therapy – Novel Mechanisms and Approaches Date and Time: Sunday, November 12, 2023, between 4:00-4:10 PM ET Location: Pennsylvania convention center room 120B About Cardurion Pharmaceuticals Cardurion is a clinical-stage biotechnology company focused on the discovery and development of novel, next-generation therapeutics for the treatment of cardiovascular diseases. Cardurion was founded by physician-scientists with world-class expertise in cardiovascular signaling pathways, and a shared passion to find and develop a pipeline of novel treatment options to improve the lives of patients. Cardurion has two groundbreaking clinical programs in development, a PDE9 program targeting heart failure and the first ever CaMKII inhibitor in clinical development targeting multiple CV indications. Cardurion Pharmaceuticals has facilities in Burlington, Massachusetts and Shonan, Japan. For more information, please visit the company’s website at .

SOUTH SAN FRANCISCO, Calif., May 03, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, announced that several abstracts have been accepted for presentation at the American Society of Gene and Cell Therapy (ASGCT) 26th Annual Meeting detailing the company’s expanded capabilities for manufacturing and discovering genetic medicines for heart disease. “Tenaya was founded with a singular focus on developing novel medicines for rare and prevalent forms of heart disease. To be a leader in this space requires a sustained commitment to differentiated platform capabilities that enable us to optimize the product profile for our therapeutic candidates and to address new conditions where there is high unmet need,” said Faraz Ali, Chief Executive Officer of Tenaya. “The presentations at this year’s ASGCT highlight innovations in manufacturing, capsid engineering and gene editing, that support the advancement of our current pipeline and create new opportunities for future pipeline candidates. We look forward to sharing work on additional innovative enabling technologies and genetic medicine programs at future meetings.” The ASGCT 26th Annual Meeting is being held in Los Angeles, California May 16-20, 2023. Details of the company’s upcoming presentations at ASGCT are available below: Manufacturing and Process Development TN-201 is an adeno-associated virus (AAV) gene therapy being advanced into the clinic for the treatment of MYBPC3-associated hypertrophic cardiomyopathy (HCM). Clinical supply of TN-201 was manufactured under current Good Manufacturing Practice regulations at Tenaya’s Genetic Medicines Manufacturing Center using the company’s proprietary Sf9 recombinant baculovirus (Sf9/rBV) production process at the 1000L scale. At ASGCT, Tenaya researchers will present data detailing the scale up, formulation and purification techniques deployed in the successful initial manufacture of TN-201. In addition to the Sf9/rBV manufacturing platform, Tenaya has also internalized the HEK293 manufacturing platform up to the 200L scale. Tenaya researchers will present data on a novel small molecule additive that increases the productivity of HEK293-based AAV manufacturing processes, which may have implications for improved scalability, yields and costs. Wednesday Poster Session, May 17, 2023, at 12 pm PT Development of Cost-Effective and Scalable Recombinant Baculovirus Production Process for the Manufacturing of AAV (Abstract #409) Lead author: Charles Feathers, Process Development Manager, Tenaya Therapeutics Development of a Comprehensive and Risk-Based Viral Safety Assurance Strategy for the Manufacturing of AAV Gene Therapy (Abstract #408) Lead author: Samantha Jones, Senior Manager, Process Development, Tenaya Therapeutics Titer Boosting of HEK293-based AAV Manufacturing Process using Proprietary Small Molecule Additive and Successful Scale up to 200L (Abstract #407) Lead author: Jackson Leong, Associate Scientist, Process Development, Tenaya Therapeutics Thursday Poster Session, May 18, 2023, 12 pm PT Development of Rational Formulation for the Delivery of AAV Viral Vector for Treatment of Heart Disease (Abstract #1010) Lead author: Joseph Woods, Process Development Associate, Tenaya Therapeutics Capsid Engineering AAV9 is the current capsid of choice for cardiovascular indications and the capsid being used in Tenaya’s TN-201 and TN-401 gene therapy product candidates. Through rigorous testing across multiple species, Tenaya has identified several novel AAV capsids with greater transduction efficiency for the heart and/or de-targeting of the liver compared to AAV9, that may be used in second generation approaches and for new product candidates. At ASGCT, Tenaya scientists will present new data from non-human primate studies for previously identified novel capsids. Wednesday Poster Session, May 17, 2023, at 12 pm PT Engineering Novel AAV Capsids for Cardiac Gene Delivery (Abstract #463) Lead author: Ze Cheng, Ph.D., Senior Scientist, Tenaya Therapeutics Gene Editing Phospholamban (PLN) plays a key role in the functioning of the heart, and carriers of the R14del pathogenic variant of the PLN gene have a high risk of developing a form of dilated cardiomyopathy that may result in severe arrhythmia, heart failure, and sudden cardiac death. At ASGCT, Tenaya scientists will show data for the first time in which genetic editing using a single AAV vector restores heart function and survival in a severe murine model of PLN cardiomyopathy caused by the R14del mutation. This foundational research will inform future drug development efforts for PLN cardiomyopathies, as well as for additional cardiomyopathies in which gene editing may be an advantageous approach to correcting specific pathogenetic drivers underlying disease. Thursday Poster Session, May 18, 2023, at 12 pm PT Gene Editing of R14del Mutation in PLN Rescues PLN-R14del-Associated Cardiomyopathy (Abstract #1113) Lead author: Huanyu Zhou, Ph.D., Senior Scientist, Tenaya Therapeutics To view full event programming, please visit the ASGCT 26th Annual Meeting website. Following the conference, Tenaya’s presentations will be available in the “Our Science” section of the company’s website. About Tenaya Therapeutics Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver potentially curative therapies that address the underlying drivers of heart disease. Leveraging its integrated and interrelated Gene Therapy, Cellular Regeneration and Precision Medicine platforms and proprietary core capabilities, the company is advancing a pipeline of novel therapies with diverse treatment modalities for rare genetic cardiovascular disorders and more prevalent heart conditions. Tenaya’s most advanced candidates include TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301, a small molecule HDAC6 inhibitor being initially developed for heart failure with preserved ejection fraction (HFpEF). Tenaya also has multiple early-stage programs progressing through preclinical development. For more information, visit www.tenayatherapeutics.com.