Ultragenyx's Gene Therapy Study Shows Ammonia Reduction In Rare Metabolic Disorder

Benzinga_recent_news03-13

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are down on Thursday as the company reported data from its Phase 3 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency. ...

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(NASDAQ:RARE) shares are down on Thursday as the company reported data from its Phase 3 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency.\n...</p>\n\n<a href=\"https://www.benzinga.com/news/health-care/26/03/51221955/ultragenyxs-gene-therapy-study-shows-ammonia-reduction-in-rare-metabolic-disorder\">Source Link</a>\n\n</div>\n","source":"benzinga_recent_news","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>Ultragenyx's Gene Therapy Study Shows Ammonia Reduction In Rare Metabolic Disorder</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\">\nUltragenyx's Gene Therapy Study Shows Ammonia Reduction In Rare Metabolic Disorder\n</h2>\n\n<h4 class=\"meta\">\n\n\n2026-03-13 01:35 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://www.benzinga.com/news/health-care/26/03/51221955/ultragenyxs-gene-therapy-study-shows-ammonia-reduction-in-rare-metabolic-disorder><strong>Benzinga_recent_news</strong></a>\n\n\n</h4>\n\n</header>\n<article>\n<div>\n<p>Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are down on Thursday as the company reported data from its Phase 3 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency.\n...</p>\n\n<a href=\"https://www.benzinga.com/news/health-care/26/03/51221955/ultragenyxs-gene-therapy-study-shows-ammonia-reduction-in-rare-metabolic-disorder\">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":"SMA","symbol_name":"Smartstop Self Storage REIT Inc","start_time":0,"source_url":"https://www.benzinga.com/news/health-care/26/03/51221955/ultragenyxs-gene-therapy-study-shows-ammonia-reduction-in-rare-metabolic-disorder","article_id":"2619959155","we_media_id":null,"thumbnails":["https://cdn.benzinga.com/files/images/story/2026/03/12/Gene-therapy.jpeg?optimize=medium&dpr=2&auto=webp&width=240"],"rights":{"source":"benzinga_recent_news","url":"https://www.benzinga.com/news/health-care/26/03/51221955/ultragenyxs-gene-therapy-study-shows-ammonia-reduction-in-rare-metabolic-disorder","rn_cache_url":null,"directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2619959155","pubTimestamp":1773336900,"columns":[],"sourceInfo":{"source_id":"benzinga_recent_news","name":"Benzinga"},"weMediaInfo":null,"summary":"Ultragenyx Phase 3 gene therapy data shows reduced ammonia levels in rare disease patients, with further results expected in 2027.","collect":0,"end_time":0,"defaultTopTitle":"benzinga.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"SMA":"Smartstop Self Storage REIT Inc","BK4139":"生物科技","RARE":"Ultragenyx Pharmaceutical Inc","BK4217":"自存仓储房地产信托"},"translate_title":"Ultragenyx的基因治疗研究显示罕见代谢紊乱的氨减少","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"SMA":1,"RARE":1},"content_text":"Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) shares are down on Thursday as the company reported data from its Phase 3 study of DTX301, a gene therapy for ornithine transcarbamylase (OTC) deficiency.\n       • Ultragenyx Pharmaceutical shares are sliding. Why is RARE stock dropping?\nOTC deficiency is an inherited disorder that causes ammonia to accumulate in the blood. OTC is one of six enzymes that play a role in the breakdown and removal of nitrogen from the body, a process known as the urea cycle. The lack of the OTC enzyme results in excessive accumulation of nitrogen, in the form of ammonia (hyperammonemia), in the blood.\nThe 36-week data from the phase 3 study of DTX301 showed promising results, with a significant increase in urinary orotic acid levels.\nAt week 36, DTX301-treated patients (n=18) demonstrated a 18% reduction in 24-hour plasma ammonia compared to placebo (n=19) and maintained average ammonia AUC0-24 in the normal range through week 36.\nEight of nine patients with abnormal ammonia at baseline, despite optimal current drug treatment and diet restriction, reached normal ammonia levels rapidly, which were generally maintained during this treatment period.\nBaseline 24-hour ammonia AUC levels were normal in 50% of DTX301-treated patients and 68% of placebo patients, who were all on current care of scavenger medications and strict dietary control of protein intake.\nPatients treated with DTX301 (n=18) experienced a rapid reduction in 24-hour ammonia by week 6 and were decreased by 18% compared to placebo (n=19) at Week 36.\nAmmonia was generally maintained in normal range in treated patients despite their mean 27% reduction in ammonia scavenger medications at week 36 (n=18) and an approximately 13% increase in protein intake relative to no change in placebo (n=19).\nDTX301 was well tolerated with an acceptable safety profile, consistent with prior phase 1/2 safety data.\nThe most common treatment-emergent adverse events were mild to moderate transient hepatic reactions managed with steroids.\nOne serious adverse event (SAE) of acute hepatitis was assessed as treatment-related and resolved with steroids.\nThe study is continuing to its second primary endpoint, the reduction in treatment burden through 64 weeks of follow-up.\nThe company expects data in the first half of 2027.\nCurrently, Ultragenyx is trading 3.7% below its 20-day simple moving average (SMA) and 24.8% below its 100-day SMA, indicating bearish momentum in the short to medium term.\nOver the past 12 months, shares have decreased by 46.87%, and they are positioned closer to their 52-week lows.\nThe RSI is at 43.32, which is considered neutral territory, while the MACD shows a value of -0.5147, with the signal line at -0.6510, indicating bullish momentum as the MACD is above the signal line.\nThe combination of neutral RSI and bullish MACD suggests mixed momentum, reflecting uncertainty in the stock’s performance.\nAnalyst Consensus & Recent Actions: The stock carries a Buy Rating with an average price target of $71.45. Recent analyst moves include:\nTop ETF Exposure\nRARE Stock Price Activity: Ultragenyx Pharmaceutical shares were down 3.02% at $21.54 at the time of publication on Thursday, according to Benzinga Pro data.\nImage via Shutterstock\nTo add Benzinga News as your preferred source on Google, click here.","kind":"highlight","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"dataReport","news_rank":0,"symbols":[],"gpt_button":0,"need_auth":false,"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,"tenantId":"TBGLOBAL","deviceId":"web-server-community-laohu8-v3","version":"4.36.2","shortVersion":"4.36.2","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/2619959155","isCrawlerRequest":true}