{"id":98090,"date":"2025-05-13T13:00:10","date_gmt":"2025-05-13T13:00:10","guid":{"rendered":"https:\/\/www.europesays.com\/uk\/98090\/"},"modified":"2025-05-13T13:00:10","modified_gmt":"2025-05-13T13:00:10","slug":"fda-clears-ensomas-application-for-rare-genetic-disorder-treatment","status":"publish","type":"post","link":"https:\/\/www.europesays.com\/uk\/98090\/","title":{"rendered":"FDA clears Ensoma&#8217;s application for rare genetic disorder treatment"},"content":{"rendered":"<p>                                    <img decoding=\"async\" src=\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/05\/shutterstock_2361780231-430x241.jpg\" alt=\"\"\/><\/p>\n<p>                                    CGD patients often suffer from severe, recurrent bacterial and fungal infections. Credit: sergey kolesnikov \/ Shutterstock.<\/p>\n<p class=\"drop-cap\">The US Food and Drug Administration (FDA) has cleared the investigational new drug application (IND) for Ensoma\u2019s lead programme, EN-374, targeting X-linked chronic granulomatous disease (X-CGD), a rare genetic disorder.<\/p>\n<p>According to Ensoma, this in vivo haematopoietic stem cell (HSC)-directed therapy utilises virus-like particles (VLPs) for the delivery of genetic payloads to engineer HSCs, aiming for sustained expression of a cytochrome b-245 beta chain (CYBB) transgene in neutrophils.<\/p>\n<p>This process is intended to restore the function of the nicotinamide adenine dinucleotide phosphate hydrogen\u00a0(NADPH) oxidase enzyme complex, which is essential for immune defence.<\/p>\n<p>A Phase I\/II trial is set to assess the therapy\u2019s potential efficacy and safety and determine an appropriate dose for further clinical development in patients with X-CGD.<\/p>\n<p>Adult X-CGD subjects will initially be enrolled in the trial\u2019s dose-escalation part, followed by paediatric subjects\u2019 enrolment in a dose-expansion cohort.<\/p>\n<p>EN-374 received FDA\u2019s orphan drug and rare paediatric disease designations earlier this year.<\/p>\n<p>In preclinical studies, the therapy claims to have shown promise in restoring CYBB protein expression and NADPH oxidase activity in circulating neutrophils.<\/p>\n<p>Ensoma noted that CGD is said to impact approximately one in 100,000 to 200,000 live births.<\/p>\n<p>The most prevalent CGD form, X-CGD, is said to account for 60%-70% of cases and is characterised by mutations in the CYBB gene that hinder neutrophils from combating infections.<\/p>\n<p>CGD patients often suffer from severe, recurrent bacterial and fungal infections, resulting in chronic inflammation and life-threatening complications.<\/p>\n<p>Ensoma CEO Jim Burns said: \u201cThe FDA\u2019s clearance of our EN-374 IND is a pivotal moment for Ensoma that further establishes our unique\u00a0in vivo\u00a0HSC engineering platform and brings us one step closer to meaningfully improving outcomes for people living with X-CGD and other chronic diseases.<\/p>\n<p>\u201cWe have completed all manufacturing activities for EN-374, through which we have successfully demonstrated reproducibility and scalability, and anticipate initiating our Phase I\/II clinical trial in Q4 2025.\u201d<\/p>\n<p>                    <img decoding=\"async\" src=\"https:\/\/www.europesays.com\/uk\/wp-content\/uploads\/2025\/05\/1746120078_246_newsletter-new.svg.svg+xml\" alt=\"Email newsletter icon\"\/><\/p>\n<p>\n                    Sign up for our daily news round-up!<br \/>\n                    Give your business an edge with our leading industry insights.\n                <\/p>\n","protected":false},"excerpt":{"rendered":"CGD patients often suffer from severe, recurrent bacterial and fungal infections. Credit: sergey kolesnikov \/ Shutterstock. The US&hellip;\n","protected":false},"author":2,"featured_media":98091,"comment_status":"","ping_status":"","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[3846],"tags":[45629,5885,267,70,16,15],"class_list":{"0":"post-98090","1":"post","2":"type-post","3":"status-publish","4":"format-standard","5":"has-post-thumbnail","7":"category-genetics","8":"tag-approvals","9":"tag-drug-development","10":"tag-genetics","11":"tag-science","12":"tag-uk","13":"tag-united-kingdom"},"share_on_mastodon":{"url":"https:\/\/pubeurope.com\/@uk\/114500653940330106","error":""},"_links":{"self":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/98090","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/comments?post=98090"}],"version-history":[{"count":0,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/posts\/98090\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media\/98091"}],"wp:attachment":[{"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/media?parent=98090"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/categories?post=98090"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.europesays.com\/uk\/wp-json\/wp\/v2\/tags?post=98090"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}