Ultragenyx Says Gene Therapy Shows Lasting Benefits, Slows Brain Decline In Fatal Childhood Disorder

Benzinga_recent_news02-04 01:58

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) on Tuesday announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec). It is an investigational AAV9 gene therapy for...

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(NASDAQ:RARE) on Tuesday announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec).\nIt is an investigational AAV9 gene therapy for...</p>\n\n<a href=\"https://www.benzinga.com/news/health-care/26/02/50345196/ultragenyx-says-gene-therapy-shows-lasting-benefits-slows-brain-decline-in-fatal-childhood-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 Says Gene Therapy Shows Lasting Benefits, Slows Brain Decline In Fatal Childhood 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 Says Gene Therapy Shows Lasting Benefits, Slows Brain Decline In Fatal Childhood Disorder\n</h2>\n\n<h4 class=\"meta\">\n\n\n2026-02-04 01:58 GMT+8&nbsp;&nbsp;&nbsp;<a href=https://www.benzinga.com/news/health-care/26/02/50345196/ultragenyx-says-gene-therapy-shows-lasting-benefits-slows-brain-decline-in-fatal-childhood-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) on Tuesday announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec).\nIt is an investigational AAV9 gene therapy for...</p>\n\n<a href=\"https://www.benzinga.com/news/health-care/26/02/50345196/ultragenyx-says-gene-therapy-shows-lasting-benefits-slows-brain-decline-in-fatal-childhood-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":"BK4139","symbol_name":"生物科技","start_time":0,"source_url":"https://www.benzinga.com/news/health-care/26/02/50345196/ultragenyx-says-gene-therapy-shows-lasting-benefits-slows-brain-decline-in-fatal-childhood-disorder","article_id":"2608480971","we_media_id":null,"thumbnails":["https://cdn.benzinga.com/files/images/story/2026/02/03/Gene-therapy.jpeg?optimize=medium&dpr=2&auto=webp&width=240"],"rights":{"source":"benzinga_recent_news","url":"https://www.benzinga.com/news/health-care/26/02/50345196/ultragenyx-says-gene-therapy-shows-lasting-benefits-slows-brain-decline-in-fatal-childhood-disorder","rn_cache_url":null,"directOrigin":true},"url":"https://stock-news.laohu8.com/highlight/detail?id=2608480971","pubTimestamp":1770141480,"columns":[],"sourceInfo":{"source_id":"benzinga_recent_news","name":"Benzinga"},"weMediaInfo":null,"summary":"Ultragenyx reported long-term study data showing lasting brain and skill improvements with UX111 for Sanfilippo syndrome, supporting its resubmitted FDA application ahead of a 2026 decision.","collect":0,"end_time":0,"defaultTopTitle":"benzinga.com","property":[],"viewcount":null,"language":"en","relate_stocks":{"BK4139":"生物科技","RARE":"Ultragenyx Pharmaceutical Inc"},"translate_title":"Ultragenyx表示基因疗法显示出持久的益处，减缓致命儿童疾病的大脑衰退","themeId":null,"isJumpTheme":false,"ttsUrl":null,"symbols_score_info":{"RARE":1},"content_text":"Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) on Tuesday announced new long‑term data from clinical studies evaluating UX111 (rebisufligene etisparvovec).\nIt is an investigational AAV9 gene therapy for Sanfilippo syndrome Type A (MPS IIIA), a fatal neurodegenerative lysosomal storage disorder.\nMPS IIIA is caused by mutations in the SGSH gene, leading to a deficiency of the enzyme heparan N-sulfatase, causing toxic accumulation of heparan sulfate in the brain and body.\nIt causes rapid cognitive decline, severe behavioral issues, and premature death.\nThe results demonstrate substantial and durable biomarker improvements and meaningful functional benefits compared with natural history.\nCognitive function, expressive and receptive communication, and fine and gross motor skills were measured using Bayley-III and compared to natural history data from untreated patients with reported rapid progressor phenotypes.\nChildren under two years of age or with earlier stage disease at the time of treatment (n=17) demonstrated a +23.2 point (p<0.0001) treatment effect in the mean Bayley-III cognitive raw score compared to natural history data during 24-60 months of age.\nIn addition to cognitive function, clinical improvements were also observed.\nOn a separate caregiver-reported outcome utilizing Vineland 3, there were comparable improvements in the communication, motor, and personal subdomains.\nEight children reached a 36‑month cognitive developmental age, enabling higher‑level testing—none of the natural‑history patients reached this milestone, the company said in a press release on Tuesday.\nPatients with older age or having more advanced disease at the time of treatment (n=10) showed retention of functional abilities in at least one of three areas at the time of last assessment that exceeded typical decline patterns in untreated children with Sanfilippo syndrome Type A.\nAs of the September 2025 cutoff date, the median reduction in cerebrospinal fluid heparan sulfate (CSF-HS) exposure was 63.98% (p<0.001).\nCSF-HS is a biomarker for monitoring neurodegenerative lysosomal storage disorders.\nThe majority of children treated (88.2% of younger patients and 81.5% of the overall efficacy set) achieved a 50% or greater reduction.\nUX111 was well-tolerated, and the safety profile remains favorable.\nThese longer-term data were included in the resubmitted Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for UX111.\nThe company anticipates up to a 6-month review period from the date of resubmission, with a PDUFA date expected in the third quarter of 2026.\nPrice Action: RARE stock is up 0.08% at $24.78 at the last check on Tuesday, according to Benzinga Pro data.\nPhoto via Shutterstock","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/2608480971","isCrawlerRequest":true}