Editor\u2019s Take: The partnership may provide a new option for a disorder that is a leading cause of intellectual disability.<\/p>\n
Enthorin Therapeutics<\/a> announced an option-to-license agreement it entered with Rumi Therapeutics<\/a>, for a candidate for Fragile X syndrome treatment.<\/p>\n ENT-7340, an Investigational New Drug (IND)-enabled, highly potent, selective small-molecule inhibitor, will be advanced by Rumi as RTX-7340.<\/p>\n In the deal, Enthorin receives an upfront payment\u2014exact terms undisclosed\u2014milestones, and an option exercise fee. Rumi assumes responsibility for RTX-7340\u2019s development. In the initial stages of that development, Enthorin will assist Rumi with scientific and technical expertise.<\/p>\n What is Fragile X?<\/p>\n Fragile X syndrome is a genetic neurodevelopmental disorder caused by changes in the FMR1 gene. The condition reduces or eliminates production of functional FMRP, a protein that has relevance to brain development.<\/p>\n As such, Fragile X is a leading inherited cause of intellectual disability, affecting learning, language, attention, behavior, and social function. Fragile X syndrome affects both males and females, and has substantial variation in its severity.<\/p>\n To treat this, ENT-7340 selectively inhibits PDE2A, an enzyme regulating both cAMP and cGMP (cyclic AMP and GMP, respectively) pathways. These each have a part to play in neuronal function and synaptic plasticity.<\/p>\n The program builds on Enthorin\u2019s previous work implicating disrupted cyclic nucleotide signaling and its role in Fragile X syndrome.<\/p>\n Enthorin Founder Talks Rumi Deal<\/p>\n Enthorin Co-Founder and Chief Scientific Officer Marco Peters, PhD, points to ENT-7340 as representative of years of discovery and development.<\/p>\n \u201cRumi brings distinctive capabilities for evaluating therapeutics in human cellular and organoid models,\u201d Peters said. \u201c[It\u2019s] an opportunity to further validate the biology of RTX-7340 in human systems and explore its mechanism of action \u2026 We are excited about this agreement and look forward to supporting Rumi during the initial development phase.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"Editor\u2019s Take: The partnership may provide a new option for a disorder that is a leading cause of…\n","protected":false},"author":2,"featured_media":897892,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[2],"tags":[4,450,451,3,452,453],"class_list":["post-897891","post","type-post","status-publish","format-standard","has-post-thumbnail","category-breaking-news","tag-breaking-news","tag-breakingnews","tag-headlines","tag-news","tag-top-stories","tag-topstories"],"_links":{"self":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/897891","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=897891"}],"version-history":[{"count":0,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/posts\/897891\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media\/897892"}],"wp:attachment":[{"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/media?parent=897891"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/categories?post=897891"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.newsbeep.com\/us\/wp-json\/wp\/v2\/tags?post=897891"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}