Business
Tenaya Therapeutics to Present Preclinical Data on Gene Therapy Programs and Platform Capabilities at the American Society of Gene and Cell Therapy 25th Annual Meeting
ASGCT Presentations Include Preclinical Data for TN-401 PKP2 and DWORF Gene Therapy Programs and AAV Capsid Engineering Innovations SOUTH SAN FRANCISCO,
About this update from Tenaya Therapeutics, Inc.
[{"type":"text","content":"\n ASGCT Presentations Include Preclinical Data for TN-401 PKP2 and DWORF Gene Therapy Programs and AAV Capsid Engineering Innovations\n\n SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--\nTenaya 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.\n\nTN-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.\n\nThe 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.\n\nTenaya 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 enhan...