{"id":2348,"date":"2026-07-10T15:43:10","date_gmt":"2026-07-10T15:43:10","guid":{"rendered":"https:\/\/corporate-movers-usa.com\/?p=2348"},"modified":"2026-07-10T15:43:10","modified_gmt":"2026-07-10T15:43:10","slug":"feds-award-43-million-to-philly-gene-therapy-research-to-advance-treatments-for-rare-diseases","status":"publish","type":"post","link":"https:\/\/corporate-movers-usa.com\/?p=2348","title":{"rendered":"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases"},"content":{"rendered":"<div>\n<p>The federal government has awarded Children\u2019s Hospital of Philadelphia $39 million to develop a scalable platform for treating rare genetic, liver-related diseases in infants and children.<\/p>\n<p>Read more <a href=\"https:\/\/corporate-movers-usa.com\/?p=2346\">Here\u2019s how the World Cup provided a sense of \u2018community\u2019 at this University City coffee shop<\/a><\/p>\n<div><\/div>\n<p>The Advanced Research Projects Agency for Health (ARPA-H) also announced this week another $4 million for local gene therapy pioneer Jim Wilson to design gene therapies using AI at his University of Pennsylvania spinout, GEMMABio.<\/p>\n<div><\/div>\n<div><\/div>\n<p>The $43 million in funding earmarked for Philadelphia researchers comes through a new federal effort to advance gene therapies for rare diseases. <\/p>\n<div><\/div>\n<p>CHOP\u2019s team includes the duo behind last year\u2019s first-of-its-kind personalized gene-editing treatment involving a Philadelphia-area infant, known as Baby KJ. CHOP\u2019s Rebecca Ahrens-Nicklas and Penn Medicine\u2019s Kiran Musunuru together created a custom drug to correct the genetic mutation driving Baby KJ\u2019s rare metabolic disease \u2014 dramatically improving his liver function. <\/p>\n<div><\/div>\n<div><\/div>\n<p>The funding will expand their work and bring in another CHOP researcher, Lindsey George, who develops gene therapies for bleeding disorders. <\/p>\n<div><\/div>\n<div><\/div>\n<p>\u201cWe\u2019re trying to move beyond just one diagnosis or one gene,\u201d Ahrens-Nicklas said.<\/p>\n<div><\/div>\n<p>Philly-based scientists comprise two of seven research teams nationwide to receive funding through ARPA-H\u2019s new $160 million effort.<\/p>\n<div><\/div>\n<p>Called THRIVE, the program aims to help the roughly one in 10 people \u2014 most being newborns, infants, and children \u2014 with chronic genetic diseases. Ninety-five percent of rare diseases currently have no approved treatments.<\/p>\n<div><\/div>\n<p>Leaders want to develop \u201cprecision genetic medicines through platforms that can test multiple treatments for multiple diseases in a single clinical trial,\u201d ARPA-H director Alicia Jackson said in a Thursday news release.<\/p>\n<div><\/div>\n<div><\/div>\n<div><\/div>\n<div>\n<h3>CHOP\u2019s expanding focus<\/h3>\n<\/div>\n<div><\/div>\n<p>The CHOP team will focus on building a scalable gene-editing platform that can be used to treat a variety of infants and children.<\/p>\n<div><\/div>\n<p>Their initial focus is on liver-related genetic diseases, ranging from urea cycle disorders to blood clotting diseases.<\/p>\n<p>Read more <a href=\"https:\/\/corporate-movers-usa.com\/?p=2344\">El Ni\u00f1o is rapidly intensifying, could become the strongest on record, and last through winter<\/a><\/p>\n<div><\/div>\n<div><\/div>\n<p>\u201cEssentially it\u2019s the same drug, whether or not you\u2019re targeting a genetic variant that causes a rare metabolic disease or a genetic variant that causes a rare coagulation disorder,\u201d Ahrens-Nicklas said.<\/p>\n<div><\/div>\n<p>Their five-year plan includes launching preclinical and clinical trials testing the safety and efficacy of their individualized treatments.<\/p>\n<div><\/div>\n<p>They will also pursue regulatory approvals, work with payors, and implement their therapies at community sites and remote hubs to expand access.<\/p>\n<div><\/div>\n<p>\u201cWe are the three musketeers that already text 25,000 times a day, and will continue to do so,\u201d Ahrens-Nicklas said. <\/p>\n<div><\/div>\n<div>\n<h3>Wilson experimenting with AI<\/h3>\n<\/div>\n<div><\/div>\n<p>Wilson founded academia\u2019s first gene therapy program back in 1993 as a professor at Penn.<\/p>\n<div><\/div>\n<p>He left in 2024 to spin out biotech startups dedicated to tackling rare diseases with genetic medicines.<\/p>\n<div><\/div>\n<div><\/div>\n<div><\/div>\n<p>\u201cThe question is, can we find ways to scale this bespoke personalized medicine strategy, so that it is affordable and cost-effective?\u201d he said.<\/p>\n<div><\/div>\n<p>In partnership with a biotech called ProFluent, GEMMABio will use AI to design its base editors \u2014 the machinery that goes in and make edits to DNA. <\/p>\n<div><\/div>\n<p>The tool could be rapidly adapted to different patients and diseases, theoretically making the process more affordable, scalable, and efficient, Wilson said.<\/p>\n<div><\/div>\n<p>His $4 million award will fund preclinical studies of their technology. <\/p>\n<div><\/div>\n<p>GEMMABio\u2019s initial focus is on two rare liver diseases \u201cfor which there is significant unmet need,\u201d he said, including Maple Syrup Urine Disease and homozygous familial hypercholesterolemia (HoFH).<\/p>\n<p>Read more <a href=\"https:\/\/corporate-movers-usa.com\/?p=2342\">Artist bringing a new mural to South Philly ahead of MLB\u2019s All-Star Game: \u2018It\u2019s a love letter to Philadelphia\u2019<\/a><\/p>\n<div><\/div>\n<\/div>\n","protected":false},"excerpt":{"rendered":"<p>The federal agency ARPA-H earmarked $43 million for Philadelphia researchers to advance gene therapies for rare diseases. Among the recipients: gene therapy pioneer Jim Wilson and the Baby KJ team.<\/p>\n","protected":false},"author":1,"featured_media":2347,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[2],"tags":[],"class_list":["post-2348","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-news"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.7 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases - Corporate Movers USA<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/corporate-movers-usa.com\/?p=2348\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases - Corporate Movers USA\" \/>\n<meta property=\"og:description\" content=\"The federal agency ARPA-H earmarked $43 million for Philadelphia researchers to advance gene therapies for rare diseases. Among the recipients: gene therapy pioneer Jim Wilson and the Baby KJ team.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/corporate-movers-usa.com\/?p=2348\" \/>\n<meta property=\"og:site_name\" content=\"Corporate Movers USA\" \/>\n<meta property=\"article:published_time\" content=\"2026-07-10T15:43:10+00:00\" \/>\n<meta name=\"author\" content=\"admin\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<meta name=\"twitter:label1\" content=\"Written by\" \/>\n\t<meta name=\"twitter:data1\" content=\"admin\" \/>\n\t<meta name=\"twitter:label2\" content=\"Est. reading time\" \/>\n\t<meta name=\"twitter:data2\" content=\"3 minutes\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"Article\",\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#article\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348\"},\"author\":{\"name\":\"admin\",\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/#\\\/schema\\\/person\\\/e17225d0157cf1fa5c6e5e795cc26626\"},\"headline\":\"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases\",\"datePublished\":\"2026-07-10T15:43:10+00:00\",\"mainEntityOfPage\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348\"},\"wordCount\":607,\"commentCount\":0,\"image\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/corporate-movers-usa.com\\\/wp-content\\\/uploads\\\/2026\\\/07\\\/7a323157d84a7b81d9f01ef312949367.avif\",\"articleSection\":[\"News\"],\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"CommentAction\",\"name\":\"Comment\",\"target\":[\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#respond\"]}]},{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348\",\"url\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348\",\"name\":\"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases - Corporate Movers USA\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#primaryimage\"},\"image\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#primaryimage\"},\"thumbnailUrl\":\"https:\\\/\\\/corporate-movers-usa.com\\\/wp-content\\\/uploads\\\/2026\\\/07\\\/7a323157d84a7b81d9f01ef312949367.avif\",\"datePublished\":\"2026-07-10T15:43:10+00:00\",\"author\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/#\\\/schema\\\/person\\\/e17225d0157cf1fa5c6e5e795cc26626\"},\"breadcrumb\":{\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#primaryimage\",\"url\":\"https:\\\/\\\/corporate-movers-usa.com\\\/wp-content\\\/uploads\\\/2026\\\/07\\\/7a323157d84a7b81d9f01ef312949367.avif\",\"contentUrl\":\"https:\\\/\\\/corporate-movers-usa.com\\\/wp-content\\\/uploads\\\/2026\\\/07\\\/7a323157d84a7b81d9f01ef312949367.avif\",\"width\":760,\"height\":507},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?p=2348#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/corporate-movers-usa.com\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/#website\",\"url\":\"https:\\\/\\\/corporate-movers-usa.com\\\/\",\"name\":\"Corporate Movers USA\",\"description\":\"\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"},{\"@type\":\"Person\",\"@id\":\"https:\\\/\\\/corporate-movers-usa.com\\\/#\\\/schema\\\/person\\\/e17225d0157cf1fa5c6e5e795cc26626\",\"name\":\"admin\",\"image\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/50b1ad2e498f523425ee0a8cc5180a210646db1622662a3d56cc405d3e0c346a?s=96&d=mm&r=g\",\"url\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/50b1ad2e498f523425ee0a8cc5180a210646db1622662a3d56cc405d3e0c346a?s=96&d=mm&r=g\",\"contentUrl\":\"https:\\\/\\\/secure.gravatar.com\\\/avatar\\\/50b1ad2e498f523425ee0a8cc5180a210646db1622662a3d56cc405d3e0c346a?s=96&d=mm&r=g\",\"caption\":\"admin\"},\"sameAs\":[\"http:\\\/\\\/corporate-movers-usa.com\"],\"url\":\"https:\\\/\\\/corporate-movers-usa.com\\\/?author=1\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases - Corporate Movers USA","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/corporate-movers-usa.com\/?p=2348","og_locale":"en_US","og_type":"article","og_title":"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases - Corporate Movers USA","og_description":"The federal agency ARPA-H earmarked $43 million for Philadelphia researchers to advance gene therapies for rare diseases. Among the recipients: gene therapy pioneer Jim Wilson and the Baby KJ team.","og_url":"https:\/\/corporate-movers-usa.com\/?p=2348","og_site_name":"Corporate Movers USA","article_published_time":"2026-07-10T15:43:10+00:00","author":"admin","twitter_card":"summary_large_image","twitter_misc":{"Written by":"admin","Est. reading time":"3 minutes"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"Article","@id":"https:\/\/corporate-movers-usa.com\/?p=2348#article","isPartOf":{"@id":"https:\/\/corporate-movers-usa.com\/?p=2348"},"author":{"name":"admin","@id":"https:\/\/corporate-movers-usa.com\/#\/schema\/person\/e17225d0157cf1fa5c6e5e795cc26626"},"headline":"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases","datePublished":"2026-07-10T15:43:10+00:00","mainEntityOfPage":{"@id":"https:\/\/corporate-movers-usa.com\/?p=2348"},"wordCount":607,"commentCount":0,"image":{"@id":"https:\/\/corporate-movers-usa.com\/?p=2348#primaryimage"},"thumbnailUrl":"https:\/\/corporate-movers-usa.com\/wp-content\/uploads\/2026\/07\/7a323157d84a7b81d9f01ef312949367.avif","articleSection":["News"],"inLanguage":"en-US","potentialAction":[{"@type":"CommentAction","name":"Comment","target":["https:\/\/corporate-movers-usa.com\/?p=2348#respond"]}]},{"@type":"WebPage","@id":"https:\/\/corporate-movers-usa.com\/?p=2348","url":"https:\/\/corporate-movers-usa.com\/?p=2348","name":"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases - Corporate Movers USA","isPartOf":{"@id":"https:\/\/corporate-movers-usa.com\/#website"},"primaryImageOfPage":{"@id":"https:\/\/corporate-movers-usa.com\/?p=2348#primaryimage"},"image":{"@id":"https:\/\/corporate-movers-usa.com\/?p=2348#primaryimage"},"thumbnailUrl":"https:\/\/corporate-movers-usa.com\/wp-content\/uploads\/2026\/07\/7a323157d84a7b81d9f01ef312949367.avif","datePublished":"2026-07-10T15:43:10+00:00","author":{"@id":"https:\/\/corporate-movers-usa.com\/#\/schema\/person\/e17225d0157cf1fa5c6e5e795cc26626"},"breadcrumb":{"@id":"https:\/\/corporate-movers-usa.com\/?p=2348#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/corporate-movers-usa.com\/?p=2348"]}]},{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/corporate-movers-usa.com\/?p=2348#primaryimage","url":"https:\/\/corporate-movers-usa.com\/wp-content\/uploads\/2026\/07\/7a323157d84a7b81d9f01ef312949367.avif","contentUrl":"https:\/\/corporate-movers-usa.com\/wp-content\/uploads\/2026\/07\/7a323157d84a7b81d9f01ef312949367.avif","width":760,"height":507},{"@type":"BreadcrumbList","@id":"https:\/\/corporate-movers-usa.com\/?p=2348#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/corporate-movers-usa.com\/"},{"@type":"ListItem","position":2,"name":"Feds award $43 million to Philly gene therapy research to advance treatments for rare diseases"}]},{"@type":"WebSite","@id":"https:\/\/corporate-movers-usa.com\/#website","url":"https:\/\/corporate-movers-usa.com\/","name":"Corporate Movers USA","description":"","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/corporate-movers-usa.com\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"},{"@type":"Person","@id":"https:\/\/corporate-movers-usa.com\/#\/schema\/person\/e17225d0157cf1fa5c6e5e795cc26626","name":"admin","image":{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/secure.gravatar.com\/avatar\/50b1ad2e498f523425ee0a8cc5180a210646db1622662a3d56cc405d3e0c346a?s=96&d=mm&r=g","url":"https:\/\/secure.gravatar.com\/avatar\/50b1ad2e498f523425ee0a8cc5180a210646db1622662a3d56cc405d3e0c346a?s=96&d=mm&r=g","contentUrl":"https:\/\/secure.gravatar.com\/avatar\/50b1ad2e498f523425ee0a8cc5180a210646db1622662a3d56cc405d3e0c346a?s=96&d=mm&r=g","caption":"admin"},"sameAs":["http:\/\/corporate-movers-usa.com"],"url":"https:\/\/corporate-movers-usa.com\/?author=1"}]}},"_links":{"self":[{"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=\/wp\/v2\/posts\/2348","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=2348"}],"version-history":[{"count":0,"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=\/wp\/v2\/posts\/2348\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=\/wp\/v2\/media\/2347"}],"wp:attachment":[{"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=2348"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=2348"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/corporate-movers-usa.com\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=2348"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}