Tenaya Therapeutics publishes preclinical data on TN-201 in cardiac function

TipRanks03-25

Tenaya Therapeutics (TNYA) announced the publications of positive preclinical data for TN-201, the company’s gene therapy candidate for Myosin-Binding Protein C3-associated hypertrophic cardiomyopathy...

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}","selectors":"article","filters":"","directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2522765008","pubTimestamp":1742906750,"columns":[],"sourceInfo":{"source_id":"yahoofinance","name":"雅虎金融"},"weMediaInfo":null,"summary":"Tenaya Therapeutics (TNYA) announced the publications of positive preclinical data for TN-201, the company’s gene therapy candidate for Myosin-Binding Protein C3-associated hypertrophic cardiomyopathy","collect":0,"end_time":0,"defaultTopTitle":"yahoo.com","property":[],"viewcount":null,"language":"en","relate_stocks":{},"translate_title":"Tenaya Therapeutics公布TN-201在心脏功能方面的临床前数据","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"LENZ":1,"TNYA":1},"content_text":"Tenaya Therapeutics (TNYA) announced the publications of positive preclinical data for TN-201, the company’s gene therapy candidate for Myosin-Binding Protein C3-associated hypertrophic cardiomyopathy, in Nature Communications. Variants in the MYBPC3 gene resulting in insufficient levels of MyBP-C protein are the most common genetic cause of HCM. TN-201 is Tenaya’s adeno-associated virus serotype 9-based gene therapy designed to deliver a working MYBPC3 gene to heart muscle cells via a single intravenous infusion, increasing MyBP-C protein levels to address the underlying cause of MYBPC3-associated HCM with the aim of halting or even reversing disease. Preclinical results published in Nature Communications show that Tenaya’s MYBPC3 gene replacement therapy achieved dose-dependent increases in MyBP-C protein, improving multiple parameters of cardiac function at protein levels well below wild-type with doses as low as 1×1013 vg/kg. Of note, treatment with Tenaya’s MYBPC3 gene therapy reversed left ventricular hypertrophy, a hallmark of HCM, as evidenced by decreases in posterior wall thickness relative to vehicle and normalization of left ventricular mass relative to body weight. TN-201 is currently being evaluated at doses of 3×1013 vg/kg and 6×1013 vg/kg in Tenaya’s ongoing MyPEAK-1 Phase 1b/2 clinical trial for the treatment of MYBPC3-associated HCM. The article, titled, “AAV9-Mediated MYBPC3 Gene Therapy with Optimized Expression Cassette Enhances Cardiac Function and Survival in MYBPC3 Cardiomyopathy Models,” describes the results from in vitro and in vivo preclinical studies. Studies conducted in human induced pluripotent stem cell-derived cardiomyocytes compared various cassette configurations and informed the final design of TN-201, which incorporates a full-length MYBPC3 gene with a proprietary cardiac promoter that maintains high specificity for heart cells. To test transduction and expression strength, additional analyses in human iPSC-derived cardiomyocytes showed transduction equivalent to 1 vector genome per diploid genome resulted in near-wild type levels of MYBPC3 RNA and MyBP-C protein at 3×1013 vg/kg. Researchers observed proportional increases in transgene RNA at doses of 3×1013 and 1×1014 vg/kg, while MyBP-C protein levels did not exceed wild type levels, indicating that RNA overexpression does not result in overexpression of protein, suggesting an attractive safety feature of MYBPC3 gene therapy. To measure the efficacy of TN-201, a mouse surrogate of TN-201 was tested against vehicle in a homozygous Mybpc3-deficient murine model that mimics severe disease in humans. Treatment with mTN-201 in Mybpc3 knock-out mice at the time of disease onset or in a more challenging model of advanced disease resulted in: Sustained increases in Mybpc3 RNA and MYBPC3 protein expression; Decreased cardiac biomarkers associated with fibrosis and heart failure; Improved cardiac function, including improved ejection fraction and diastolic function; Heart remodeling; Extended survival; These results were dose dependent, with near-maximal efficacy achieved at doses of 3×1013 vg/kg, and durable, lasting out to 20 months post-treatment. Additional experiments in human engineered heart tissue models that replicate the hypercontractility associated with MYBPC3-associated HCM demonstrated:; Resolution of calcium handling abnormalities; Enhanced diastolic activity.\nLight Up your Portfolio with Spark:\n\nEasily identify stocks' risks and opportunities.\n Discover stocks' market position with detailed  competitor analyses.\n\n\nPublished first on TheFly – the ultimate source for real-time, market-moving breaking financial news. Try Now>>\n\n\nSee today’s best-performing stocks on TipRanks >>\n\n\nRead More on TNYA:Questions or Comments about the article? Write to editor@tipranks.com \n \nTenaya Therapeutics files $300M mixed securities shelf\nTenaya Therapeutics: Strong Financial Position and Promising Clinical Trial Results Justify Buy Rating\nBiotech Alert: Searches spiking for these stocks today\nPromising Clinical Data and Financial Stability Support Buy Rating for Tenaya Therapeutics Despite Price Target Reduction\nTenaya Therapeutics price target lowered to $6 from $18 at Canaccord","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"0","news_tag":"","news_rank":0,"symbols":[],"gpt_button":1,"code":"91000000","status":"200"},"commentList":[],"isCommentEnd":true,"newsSizeData":{"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"likeStatus":false,"favoriteStatus":false},"APP":{"userAgent":"Mozilla/5.0 AppleWebKit/537.36 (KHTML, like Gecko; compatible; ClaudeBot/1.0; +claudebot@anthropic.com)","isDev":false,"isTTM":true,"deviceId":"web-server-community-laohu8-v3","version":"4.32.4","shortVersion":"4.32.4","platform":"web","vendor":"web","appName":"ttm","isIOS":false,"isAndroid":false,"isTiger":false,"isTHS":false,"isWeiXin":false,"isWeiXinMini":false,"isWeiBo":false,"isQQ":false,"isBaiduSwan":false,"isBaiduBox":false,"isDingTalk":false,"isToutiao":false,"isOnePlus":false,"isHuaWei":false,"isXiaomi":false,"isXiaomiWebView":false,"isOppo":false,"isVivo":false,"isSamsung":false,"isMobile":false},"href":"/m/news/2522765008"}