{"id":10350,"date":"2026-02-09T23:02:57","date_gmt":"2026-02-09T23:02:57","guid":{"rendered":"https:\/\/mailitics.com\/index.php\/2026\/02\/09\/fda-rejects-regenxbio-rare-disease-gene-therapy\/"},"modified":"2026-02-09T23:02:57","modified_gmt":"2026-02-09T23:02:57","slug":"fda-rejects-regenxbio-rare-disease-gene-therapy","status":"publish","type":"post","link":"https:\/\/mailitics.com\/index.php\/2026\/02\/09\/fda-rejects-regenxbio-rare-disease-gene-therapy\/","title":{"rendered":"STAT+: FDA rejects Regenxbio\u2019s rare-disease gene therapy"},"content":{"rendered":"<p>    STAT+: FDA rejects Regenxbio\u2019s rare-disease gene therapy<br \/>\n \t<BR><br \/>\n<BR><\/BR><br \/>\n    <!-- no image --><br \/>\n \t<BR><br \/>\n<BR><\/BR><\/p>\n<div>\n<p>The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday.\u00a0<\/p>\n<p>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<p>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<p><a href=\"https:\/\/www.statnews.com\/2026\/02\/09\/fda-rejects-regenxbio-rare-disease-gene-therapy\/?utm_campaign=rss\">Continue to STAT+ to read the full story\u2026<\/a><\/p>\n<\/div>\n<p> \t<BR><br \/>\n <BR><\/BR><br \/>\n    Adam Feuerstein<br \/>\n \t<BR><br \/>\n<BR><\/BR><br \/>\n<a href=\"https:\/\/www.statnews.com\/2026\/02\/09\/fda-rejects-regenxbio-rare-disease-gene-therapy\/?utm_campaign=rss\">Go to statnews<\/a><br \/>\n \t<BR><br \/>\n <BR><\/BR><\/p>\n","protected":false},"excerpt":{"rendered":"<p>STAT+: FDA rejects Regenxbio\u2019s rare-disease gene therapy The Food and Drug Administration has rejected a rare-disease gene therapy from Regenxbio, the company said Monday.\u00a0 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 [&hellip;]<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[2194,2154,2659,2678,2152],"tags":[2150],"class_list":["post-10350","post","type-post","status-publish","format-standard","hentry","category-biotech","category-biotechnology","category-gene-therapy","category-rare-diseases","category-stat","tag-statnews"],"_links":{"self":[{"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/posts\/10350"}],"collection":[{"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/comments?post=10350"}],"version-history":[{"count":0,"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/posts\/10350\/revisions"}],"wp:attachment":[{"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/media?parent=10350"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/categories?post=10350"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/mailitics.com\/index.php\/wp-json\/wp\/v2\/tags?post=10350"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}