Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst

Benzinga11-20

On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease. Danon disease is a rare X-...

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(NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.\nDanon disease is a rare X-...</p>\n\n<a href=\"https://finance.yahoo.com/news/rocket-pharmaceuticals-gene-therapy-progress-194724790.html\">Source Link</a>\n\n</div>\n","source":"yahoofinance","html":"<!DOCTYPE html>\n<html>\n<head>\n<meta http-equiv=\"Content-Type\" content=\"text/html; charset=utf-8\" />\n<meta name=\"viewport\" content=\"width=device-width,initial-scale=1.0,minimum-scale=1.0,maximum-scale=1.0,user-scalable=no\"/>\n<meta name=\"format-detection\" content=\"telephone=no,email=no,address=no\" />\n<title>Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst</title>\n<style type=\"text/css\">\na,abbr,acronym,address,applet,article,aside,audio,b,big,blockquote,body,canvas,caption,center,cite,code,dd,del,details,dfn,div,dl,dt,\nem,embed,fieldset,figcaption,figure,footer,form,h1,h2,h3,h4,h5,h6,header,hgroup,html,i,iframe,img,ins,kbd,label,legend,li,mark,menu,nav,\nobject,ol,output,p,pre,q,ruby,s,samp,section,small,span,strike,strong,sub,summary,sup,table,tbody,td,tfoot,th,thead,time,tr,tt,u,ul,var,video{ font:inherit;margin:0;padding:0;vertical-align:baseline;border:0 }\nbody{ font-size:16px; line-height:1.5; color:#999; background:transparent; }\n.wrapper{ overflow:hidden;word-break:break-all;padding:10px; }\nh1,h2{ font-weight:normal; line-height:1.35; margin-bottom:.6em; }\nh3,h4,h5,h6{ line-height:1.35; margin-bottom:1em; }\nh1{ font-size:24px; }\nh2{ font-size:20px; }\nh3{ font-size:18px; }\nh4{ font-size:16px; }\nh5{ font-size:14px; }\nh6{ font-size:12px; }\np,ul,ol,blockquote,dl,table{ margin:1.2em 0; }\nul,ol{ margin-left:2em; }\nul{ list-style:disc; }\nol{ list-style:decimal; }\nli,li p{ margin:10px 0;}\nimg{ max-width:100%;display:block;margin:0 auto 1em; }\nblockquote{ color:#B5B2B1; border-left:3px solid #aaa; padding:1em; }\nstrong,b{font-weight:bold;}\nem,i{font-style:italic;}\ntable{ width:100%;border-collapse:collapse;border-spacing:1px;margin:1em 0;font-size:.9em; }\nth,td{ padding:5px;text-align:left;border:1px solid #aaa; }\nth{ font-weight:bold;background:#5d5d5d; }\n.symbol-link{font-weight:bold;}\n/* header{ border-bottom:1px solid #494756; } */\n.title{ margin:0 0 8px;line-height:1.3;color:#ddd; }\n.meta {color:#5e5c6d;font-size:13px;margin:0 0 .5em; }\na{text-decoration:none; color:#2a4b87;}\n.meta .head { display: inline-block; overflow: hidden}\n.head .h-thumb { width: 30px; height: 30px; margin: 0; padding: 0; border-radius: 50%; float: left;}\n.head .h-content { margin: 0; padding: 0 0 0 9px; float: left;}\n.head .h-name {font-size: 13px; color: #eee; margin: 0;}\n.head .h-time {font-size: 11px; color: #7E829C; margin: 0;line-height: 11px;}\n.small {font-size: 12.5px; display: inline-block; transform: scale(0.9); -webkit-transform: scale(0.9); transform-origin: left; -webkit-transform-origin: left;}\n.smaller {font-size: 12.5px; display: inline-block; transform: scale(0.8); -webkit-transform: scale(0.8); transform-origin: left; -webkit-transform-origin: left;}\n.bt-text {font-size: 12px;margin: 1.5em 0 0 0}\n.bt-text p {margin: 0}\n</style>\n</head>\n<body>\n<div class=\"wrapper\">\n<header>\n<h2 class=\"title\">\nRocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst\n</h2>\n\n<h4 class=\"meta\">\n\n\n2024-11-20 03:47 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://finance.yahoo.com/news/rocket-pharmaceuticals-gene-therapy-progress-194724790.html><strong>Benzinga</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.\nDanon disease is a rare X-...</p>\n\n<a href=\"https://finance.yahoo.com/news/rocket-pharmaceuticals-gene-therapy-progress-194724790.html\">Source Link</a>\n\n</div>\n\n\n</article>\n</div>\n</body>\n</html>\n","isBrief":false,"type":0,"news_type":1,"symbol":"FA","symbol_name":"First Advantage Corp.","start_time":0,"source_url":"https://finance.yahoo.com/news/rocket-pharmaceuticals-gene-therapy-progress-194724790.html","article_id":"2484771989","we_media_id":null,"thumbnails":["https://s.yimg.com/uu/api/res/1.2/3vJwnV.fyXKsx.OAsR1l7A--~B/aD01NTM7dz05ODM7YXBwaWQ9eXRhY2h5b24-/https://media.zenfs.com/en/Benzinga/2d742347a4190f9eba3ad8b9cafa0828"],"rights":null,"url":"https://stock-news.laohu8.com/highlight/detail?id=2484771989","pubTimestamp":1732045644,"sourceInfo":{"source_id":"yahoofinance","name":"雅虎金融"},"weMediaInfo":null,"summary":"On Monday, Rocket Pharmaceuticals, Inc.  presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.Danon disease isa rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy , skeletal myopathy , and intellectual disability.Also Read: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides. Danon disease is caused by mutations in the LAMP2 gene, which contains instructions for the production of an enzyme called lysosomal-associated membrane protein-2 . The data showed that RP-A501 was generally well tolerated.All evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months  and reduction of left ventricular  mass index by ≥10% at 12 months  after treatment.These data were presented at the American Heart Association  Scientific Sessions 2024.Evidence of sustained clinically meaningful improvement was observed in pe","collect":0,"end_time":0,"defaultTopTitle":"yahoo.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"FA":"First Advantage Corp.","BK4130":"人力资源与就业服务","RCKTW":"ROCKET PHARMACEUTICALS INC C/WTS 02/09/2026 (TO PUR COM)","BK4535":"淡马锡持仓","BK4097":"系统软件","RCKT":"Rocket Pharmaceuticals, Inc.","PRGS":"Progress Software Corporation","BK4139":"生物科技","BK4539":"次新股","LU2065732104.USD":"ALLSPRING (LUX) WF GLOBAL SMALL CAP EQUITY \"I\" (USD) ACC"},"translate_title":"Rocket Pharmaceuticals的基因治疗进展增强了对未来发展的信心：分析师","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"FA":1,"PRGS":1,"RCKT":0.9,"RCKTW":0.9},"content_text":"On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study of RP-A501 in male patients with Danon disease.\nDanon disease is a rare X-linked dominant genetic disorder that manifests with the clinical triad of cardiomyopathy (stiff heart muscles), skeletal myopathy (weakness), and intellectual disability.\nAlso Read: FDA Rejects Rocket Pharmaceuticals' Gene Therapy For Rare Immune Disorder, Asks For More Data, Stock Slides\nDanon disease is caused by mutations in the LAMP2 gene, which contains instructions for the production of an enzyme called lysosomal-associated membrane protein-2 (LAMP-2). The data showed that RP-A501 was generally well tolerated.\nAll evaluable Danon disease patients demonstrated LAMP2 protein expression at 12 months (sustained up to 60 months) and reduction of left ventricular (LV) mass index by ≥10% at 12 months (sustained up to 54 months) after treatment.\nThese data were presented at the American Heart Association (AHA) Scientific Sessions 2024.\nEvidence of sustained clinically meaningful improvement was observed in pediatric patients followed up to 24 months and adult/adolescent patients followed up to 60 months.\nAll evaluable patients in the Phase 1 trial demonstrated:\n\nCardiac LAMP2 protein expression at 12 months and thereafter.\nReduction or stabilization of Left ventricular mass index (LV mass index)– the median reduction from baseline to the most recent visit of 24%.\nPreservation of normal LV ejection fraction (LVEF).\nReduction or stabilization of cardiac biomarkers (median cTnI and NTproBNP reductions of 84% and 57%, respectively).\nThe study also showed the absence of clinical signs of heart failure in patients treated with RP-A501.\n\nWilliam Blair views the long-term follow-up favorably and says it supports RP-A501's durable efficacy.\nOverall, the updated data from the Phase I study of RP-A501 de-risks the pivotal trial, as significant improvements in both co-primary endpoints were seen across all six treated patients.\nThe analyst looks forward to additional updates on the pivotal study and the company's prelaunch preparations in 2025.\n\"Although we continue to see RP-A501 as the main stock driver in the near term given its large total addressable market, we believe the approval of RP-L201 in leukocyte adhesion deficiency type I (LAD-I) and the completion of the rolling BLA for RP-L102 in Fanconi anemia (FA) could be incremental catalysts for Rocket in 2025,\" William Blair writes.\nPrice Action: RCKT stock is down 3.44% at $13.19 at the last check on Tuesday.\nRead Next:\n\nBiogen's Outlook Dims As Analyst Projects Flat Revenue Until 2026\n\nLatest Ratings for RCKT\n\n\n\nDate\nFirm\nAction\nFrom\nTo\n\n\n\n\nFeb 2022\nSVB Leerink\nMaintains\n\nOutperform\n\n\nDec 2021\nChardan Capital\nMaintains\n\nBuy\n\n\nAug 2021\nSVB Leerink\nMaintains\n\nOutperform\n\n\n\n View More Analyst Ratings for RCKT \n View the Latest Analyst Ratings \nUp Next: Transform your trading with Benzinga Edge's one-of-a-kind market trade ideas and tools. Click now to access unique insights that can set you ahead in today's competitive market.\nGet the latest stock analysis from Benzinga?\n\nThis article Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst originally appeared on Benzinga.com\n© 2024 Benzinga.com. Benzinga does not provide investment advice. 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