{"id":459890,"date":"2026-02-10T09:35:16","date_gmt":"2026-02-10T09:35:16","guid":{"rendered":"https:\/\/www.newsbeep.com\/us\/459890\/"},"modified":"2026-02-10T09:35:16","modified_gmt":"2026-02-10T09:35:16","slug":"fda-rejects-regenxbios-mps-ii-rare-disease-gene-therapy","status":"publish","type":"post","link":"https:\/\/www.newsbeep.com\/us\/459890\/","title":{"rendered":"FDA rejects Regenxbio\u2019s MPS II rare-disease gene therapy"},"content":{"rendered":"

Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud<\/a> and author of the newsletter Adam\u2019s Biotech Scorecard.<\/a> You can reach Adam on Signal at stataf.54.<\/p>\n

The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday.\u00a0<\/p>\n

The one-time treatment, called RGX-121, is designed to replace a malfunctioning gene that causes mucopolysaccharidosis type II, also known as Hunter syndrome, an ultra-rare disorder that causes physical and cognitive impairments.\u00a0<\/p>\n

Regenxbio had applied for accelerated approval, a type of conditional market clearance, based on RGX-121\u2019s ability to significantly reduce in the short term a specific biomarker in cerebrospinal fluid believed to correlate with longer-term cognitive improvements in patients with the severe form of Hunter syndrome.\u00a0<\/p>\n

STAT+ Exclusive Story<\/p>\n

Already have an account? Log in<\/a><\/p>\n

\t\t\t\"STAT+\"\/<\/p>\n

\t\t\t\t\t
\n\t\t\t\t\t\t\"STAT+\"\/
\n\t\t\t\t\t<\/a><\/p>\n

\t\t\t\tThis article is exclusive to STAT+ subscribers
\n\t\t\t\tUnlock this article \u2014 plus daily coverage and analysis of the biotech sector \u2014 by subscribing to STAT+.<\/p>\n

Already have an account? Log in<\/a><\/p>\n

\t\t\t\t\t\tIndividual plans<\/p>\n

\t\t\t\t\t\tGroup plans<\/p>\n

\t\t\t\tView All Plans<\/a><\/p>\n

\n\t\t\t\t\t\tTo read the rest of this story subscribe to STAT+.\t\t\t\t\t<\/p>\n

\t\t\t\t\tSubscribe<\/a><\/p>\n","protected":false},"excerpt":{"rendered":"Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall…\n","protected":false},"author":2,"featured_media":152895,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[244,28,13056,9779,248],"class_list":{"0":"post-459890","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-business","8":"tag-biotechnology","9":"tag-business","10":"tag-gene-therapy","11":"tag-rare-diseases","12":"tag-stat"},"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/459890","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/comments?post=459890"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/459890\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/152895"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=459890"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=459890"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=459890"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}