Tenaya Therapeutics Receives $8 Million Clinical Grant from California Institute for Regenerative Medicine
Tenaya Therapeutics (NASDAQ: TNYA) has received an $8 million CLIN2 grant from the California Institute for Regenerative Medicine (CIRM) to support its ongoing RIDGE-1 Phase 1b clinical trial of TN-401 gene therapy. The therapy is being developed to treat PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), a severe progressive disease affecting approximately 70,000 people in the U.S.
TN-401 is designed as a one-time intravenous gene replacement therapy using AAV9 capsid to deliver functional PKP2 genes into heart muscle cells, addressing the underlying cause of the disease. The RIDGE-1 trial is currently enrolling symptomatic adults with PKP2-associated ARVC, with initial data from the low-dose cohort expected in the second half of 2025.
Tenaya Therapeutics (NASDAQ: TNYA) ha ricevuto un finanziamento CLIN2 di 8 milioni di dollari dall'Istituto della California per la Medicina Rigenerativa (CIRM) per supportare il suo ongoing trial clinico RIDGE-1 di fase 1b della terapia genica TN-401. Questa terapia è in fase di sviluppo per trattare la cardiomiopatia aritmogena ventricolare destra associata a PKP2 (ARVC), una grave malattia progressiva che colpisce circa 70.000 persone negli Stati Uniti.
TN-401 è progettata come una terapia di sostituzione genica intraveneosa da somministrare una sola volta, utilizzando un capsid AAV9 per consegnare geni PKP2 funzionanti nelle cellule muscolari del cuore, affrontando così la causa sottostante della malattia. Il trial RIDGE-1 sta attualmente arruolando adulti sintomatici con ARVC associato a PKP2, con dati iniziali dal gruppo a bassa dose previsti nella seconda metà del 2025.
Tenaya Therapeutics (NASDAQ: TNYA) ha recibido una subvención CLIN2 de 8 millones de dólares del Instituto de Medicina Regenerativa de California (CIRM) para apoyar su ensayo clínico RIDGE-1 de fase 1b de la terapia genética TN-401. Esta terapia se está desarrollando para tratar la miocardiopatía arritmogénica ventricular derecha asociada a PKP2 (ARVC), una enfermedad progresiva grave que afecta aproximadamente a 70,000 personas en los EE. UU.
TN-401 está diseñada como una terapia de reemplazo genético intravenosa única, utilizando un capsid AAV9 para entregar genes PKP2 funcionales a las células musculares del corazón, abordando así la causa subyacente de la enfermedad. El ensayo RIDGE-1 está actualmente reclutando adultos sintomáticos con ARVC asociado a PKP2, con datos iniciales del grupo de baja dosis esperados en la segunda mitad de 2025.
Tenaya Therapeutics (NASDAQ: TNYA)는 캘리포니아 재생 의학 연구소(CIRM)로부터 TN-401 유전자 요법의 RIDGE-1 1b 단계 임상 시험을 지원하기 위해 800만 달러의 CLIN2 보조금을 받았습니다. 이 요법은 PKP2 관련 심장성 우심실 비대증(ARVC)을 치료하기 위해 개발되고 있으며, 이 질병은 미국에서 약 70,000명에게 영향을 미치는 심각한 진행성 질환입니다.
TN-401은 AAV9 캡시드를 사용하여 기능성 PKP2 유전자를 심장 근육 세포에 전달하는 일회성 정맥 투여 유전자 대체 요법으로 설계되었습니다. RIDGE-1 임상 시험은 현재 PKP2 관련 ARVC 증상을 가진 성인을 모집하고 있으며, 저용량 군의 초기 데이터는 2025년 하반기에 예상됩니다.
Tenaya Therapeutics (NASDAQ: TNYA) a reçu une subvention CLIN2 de 8 millions de dollars de l'Institut californien de médecine régénérative (CIRM) pour soutenir son essai clinique RIDGE-1 de phase 1b pour la thérapie génique TN-401. Cette thérapie est en cours de développement pour traiter la cardiomyopathie arythmogène ventriculaire droite associée à PKP2 (ARVC), une maladie progressive grave touchant environ 70 000 personnes aux États-Unis.
TN-401 est conçue comme une thérapie de remplacement génique intraveineux à usage unique utilisant un capsid AAV9 pour délivrer des gènes PKP2 fonctionnels dans les cellules musculaires cardiaques, s'attaquant ainsi à la cause sous-jacente de la maladie. L'essai RIDGE-1 recrute actuellement des adultes symptomatiques atteints d'ARVC associée à PKP2, avec des données préliminaires du groupe à faible dose attendues au cours de la deuxième moitié de 2025.
Tenaya Therapeutics (NASDAQ: TNYA) hat einen CLIN2-Zuschuss in Höhe von 8 Millionen US-Dollar vom kalifornischen Institut für regenerative Medizin (CIRM) erhalten, um die laufende RIDGE-1-Phase-1b-Studie zur TN-401-Gentherapie zu unterstützen. Die Therapie wird entwickelt, um die mit PKP2 assoziierte arrhythmogene rechtsventrikuläre Kardiomyopathie (ARVC) zu behandeln, eine schwere fortschreitende Erkrankung, die in den USA etwa 70.000 Menschen betrifft.
TN-401 ist als einmalige intravenöse Gentherapie konzipiert, die das AAV9-Capsid nutzt, um funktionale PKP2-Gene in die Herzmuskelzellen zu transportieren und somit die zugrunde liegende Ursache der Krankheit zu bekämpfen. Die RIDGE-1-Studie rekrutiert derzeit symptomatische Erwachsene mit PKP2-assoziierter ARVC, wobei erste Daten aus der Niedrigdosisgruppe in der zweiten Hälfte des Jahres 2025 erwartet werden.
- Secured $8 million CLIN2 grant funding for clinical trial costs
- Initial clinical trial data expected in 2H 2025
- Addressing a market of approximately 70,000 patients in the U.S.
- Early-stage Phase 1b trial with no efficacy data yet
- Results not expected until second half of 2025
Insights
The
The addressable market is substantial, with an estimated 70,000 patients in the U.S. affected by PKP2-associated ARVC, a severe progressive heart condition currently lacking effective treatments. Using a conservative annual pricing estimate typical for gene therapies (
The AAV9 viral vector approach for cardiac gene delivery has shown promise in other cardiovascular applications and TN-401's one-time treatment paradigm could offer a transformative solution for ARVC patients. The RIDGE-1 trial's design, focusing on symptomatic adults, represents a well-defined initial patient population that could support an efficient path to market.
From a financial perspective, this non-dilutive funding is particularly valuable for a microcap biotech, potentially extending the company's operational runway. The upcoming data readout in 2H25 represents a important catalyst that could validate their platform technology and potentially drive strategic partnership discussions.
The parallel natural history study (NCT06311708) demonstrates strategic foresight, as such data often proves important for regulatory submissions and commercial planning. This comprehensive approach to clinical development suggests a well-thought-out strategy to address both FDA requirements and future market access considerations.
This CIRM grant is strategically significant for Tenaya, particularly given their current market capitalization of just
The development path for TN-401 appears well-structured, with the Phase 1b RIDGE-1 trial positioned to generate initial safety and efficacy signals in 2H25. This data readout represents a critical value-inflection point that could reshape market perception. The current market cap suggests significant investor skepticism, creating potential upside if the data proves promising.
The ongoing natural history study demonstrates a sophisticated clinical development strategy, as such data typically strengthens regulatory submissions and supports pricing discussions with payers. This approach, combined with the well-defined patient population and genetic basis of the disease, could support an efficient path to market if the therapy proves effective.
However, investors should note that gene therapy development carries substantial technical and regulatory risks. The company's relatively small market cap, despite targeting a significant addressable market, reflects these uncertainties and the competitive nature of the gene therapy landscape.
Funding will Support Ongoing RIDGE™-1 Clinical Trial of TN-401 Gene Therapy for the Potential Treatment of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy
Initial Data from RIDGE-1 Phase 1b Clinical Trial Expected in 2H25
SOUTH SAN FRANCISCO, Calif., Feb. 03, 2025 (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 the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, and gene therapy research, has awarded Tenaya Therapeutics an
TN-401 is being developed for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC), (also known as arrhythmogenic cardiomyopathy or ACM), caused by mutations in the plakophilin-2 (PKP2) gene. PKP2 gene mutations result in insufficient levels of critical proteins needed to maintain the structural integrity and cell-to-cell signaling of heart muscle cells. TN-401 gene replacement therapy is designed to address the underlying cause of disease by delivering a functional PKP2 gene into heart muscle cells using an adeno associated virus serotype 9 (AAV9) capsid. The RIDGE-1 clinical trial will assess the safety, tolerability and preliminary clinical efficacy of a one-time intravenous infusion of TN-401. The open-label, dose-escalation study is currently enrolling symptomatic adults who have been diagnosed with PKP2-associated ARVC.
"We are honored to be awarded this substantial grant and gratified to continue our collaboration with CIRM, whose efforts to accelerate world-class science and deliver transformative regenerative medicine treatments are making a profound impact on the advancement of novel treatments for serious conditions," said Faraz Ali, Chief Executive Officer of Tenaya Therapeutics. "Funds from this grant will support our ongoing RIDGE-1 clinical trial of TN-401, a potential best-in-class gene therapy for the treatment of PKP2-associated ARVC, a severe, progressive disease affecting an estimated 70,000 people in the U.S. RIDGE-1 is actively enrolling patients at leading centers, and we look forward to sharing initial data from the low-dose cohort in the second half of this year."
To learn more about gene therapy for ARVC and participation in the RIDGE-1 study, please visit ARVCstudies.com or ClinicalTrials.gov (NCT06228924). Tenaya is also conducting a global natural history and seroprevalence study of adults with PKP2-assocated ARVC (NCT06311708).
About PKP2-Associated ARVC
Plakophilin-2 (PKP2) mutations are the most common genetic cause of arrhythmogenic right ventricular cardiomyopathy (ARVC), estimated to represent approximately 40 percent of the overall ARVC population. The prevalence of PKP2-associated ARVC is estimated at more than 70,000 people in the U.S. alone.
In PKP2-associated ARVC, mutations of the PKP2 gene results in insufficient expression of a protein needed for the proper functioning of the desmosomal complex that maintains physical connections and electrical signaling between heart muscle cells. As the desmosome structure degrades, cardiac muscle cells are replaced by fibrofatty tissue and electrical pulses in the heart become unstable, resulting in irregular heart rhythms. ARVC symptoms include arrhythmias, palpitations, lightheadedness, dizziness and fainting. It is typically diagnosed before age 40, and sudden cardiac arrest due to life-threatening ventricular arrhythmias is frequently the first manifestation of disease. Current treatments include anti-arrhythmic medications, implantable cardioverter-defibrillators (ICDs) and ablation procedures, which do not address the underlying genetic cause of disease.
About TN-401 Gene Therapy
TN-401 is an investigational AAV9-based gene therapy being developed for the treatment of ARVC due to mutations in the PKP2 gene. AAV9 was selected as the vector for delivery of Tenaya’s PKP2 gene therapy based on its extensive clinical and commercial safety record and demonstrated ability to target heart muscle cells. In preclinical studies, Tenaya has shown that a single dose of TN-401 restored healthy levels of PKP2 protein, normalized heart rhythms, improved right and left ventricular size and function and extended survival.
Tenaya is conducting the RIDGE-1 Phase 1b clinical trial of TN-401 in patients with PKP2-associated ARVC. To support TN-401’s clinical development, the company is currently enrolling the RIDGE global non-interventional study to collect natural history and AAV9 antibody (seroprevalence) data among ARVC patients carrying PKP2 gene mutations. TN-401 has received Orphan Drug and Fast Track Designations from the FDA.
About the California Institute for Regenerative Medicine (CIRM)
CIRM is a funding agency established by Californians to accelerate regenerative medicine research to deliver treatments for patients with unmet medical needs. Established in 2004 through the passage of Proposition 71, CIRM was initially funded with
CIRM has provided billions in funding to support stem cell, genetic research, and development programs in its portfolio. Through the Agency’s research, infrastructure, and education programs, CIRM aims to transform the field of regenerative medicine, stimulate economic growth, and improve the lives of diverse communities throughout the state. For more information go to cirm.ca.gov.
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. Tenaya employs a suite of integrated internal capabilities, including modality agnostic target validation, capsid engineering and manufacturing, to generate a portfolio of genetic medicines aimed at the treatment of both rare genetic disorders and more prevalent heart conditions. Tenaya’s pipeline includes TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), TN-401, a gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), TN-301, a small molecule HDAC6 inhibitor intended for heart failure with preserved ejection fraction (HFpEF), and multiple early-stage programs in preclinical development. For more information, visit www.tenayatherapeutics.com.
Forward Looking Statements
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 “expected,” “will,” “potential,” “estimated,” “look forward” and similar expressions are intended to identify forward-looking statements. Such forward-looking statements include, among other things, the planned timing to report initial data from RIDGE-1, the clinical, therapeutic and commercial potential of, and expectations regarding TN-401; clinical development plans for TN-401; targeted populations for PKP2-associated ARVC clinical trials and treatments; and statements made by Tenaya’s Chief Executive Officer. 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 timing and progress of RIDGE-1; the potential failure of TN-401 to demonstrate safety and/or efficacy in clinical testing; availability of RIDGE-1 data at the referenced time; the potential for any RIDGE-1 clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; our estimates of the number of patients who suffer from PKP2-associated ARVC; 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 continuing compliance with applicable legal and regulatory requirements; Tenaya’s ability to raise any additional funding it will need to continue to pursue the development of TN-401; 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 TN-401; 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.
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FAQ
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