{"id":2115,"date":"2019-01-04T17:40:23","date_gmt":"2019-01-04T17:40:23","guid":{"rendered":"https:\/\/scda.dojiggyengage.com\/?p=2115"},"modified":"2019-01-04T17:40:23","modified_gmt":"2019-01-04T17:40:23","slug":"crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease","status":"publish","type":"post","link":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/","title":{"rendered":"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease"},"content":{"rendered":"<p><span class=\"article-body\">CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies.<\/span><\/p>\n<p align=\"justify\">The FDA\u2019s Fast Track program is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.\u00a0 A drug granted Fast Track Designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if relevant criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA).<\/p>\n<p align=\"justify\">In October 2018, CRISPR and Vertex announced the FDA acceptance of the Investigational New Drug application (IND) for CTX001 for the treatment of SCD, and enrollment in a Phase 1\/2 trial in SCD is currently underway in the U.S. The companies are also evaluating CTX001 for the treatment of \u03b2-thalassemia, and enrollment in a Phase 1\/2 trial in \u03b2-thalassemia is currently open at multiple clinical trial sites in\u00a0Europe.<\/p>\n<p>Click\u00a0<a href=\"http:\/\/www.globenewswire.com\/news-release\/2019\/01\/04\/1680551\/0\/en\/CRISPR-Therapeutics-and-Vertex-Announce-FDA-Fast-Track-Designation-for-CTX001-for-the-Treatment-of-Sickle-Cell-Disease.html\"><strong>here<\/strong><\/a>\u00a0to read the press release issued today.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. The FDA\u2019s Fast Track program is designed to [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":1376,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-2115","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-uncategorized"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v24.0 - https:\/\/yoast.com\/wordpress\/plugins\/seo\/ -->\n<title>CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease - Sickle Cell Disease Association of America Inc.<\/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:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease - Sickle Cell Disease Association of America Inc.\" \/>\n<meta property=\"og:description\" content=\"CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. The FDA\u2019s Fast Track program is designed to [&hellip;]\" \/>\n<meta property=\"og:url\" content=\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/\" \/>\n<meta property=\"og:site_name\" content=\"Sickle Cell Disease Association of America Inc.\" \/>\n<meta property=\"article:published_time\" content=\"2019-01-04T17:40:23+00:00\" \/>\n<meta property=\"og:image\" content=\"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg\" \/>\n\t<meta property=\"og:image:width\" content=\"800\" \/>\n\t<meta property=\"og:image:height\" content=\"250\" \/>\n\t<meta property=\"og:image:type\" content=\"image\/jpeg\" \/>\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=\"1 minute\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\/\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/\",\"url\":\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/\",\"name\":\"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease - Sickle Cell Disease Association of America Inc.\",\"isPartOf\":{\"@id\":\"https:\/\/www.sicklecelldisease.org\/#website\"},\"primaryImageOfPage\":{\"@id\":\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#primaryimage\"},\"image\":{\"@id\":\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#primaryimage\"},\"thumbnailUrl\":\"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg\",\"datePublished\":\"2019-01-04T17:40:23+00:00\",\"dateModified\":\"2019-01-04T17:40:23+00:00\",\"author\":{\"@id\":\"https:\/\/www.sicklecelldisease.org\/#\/schema\/person\/efc0c6b0f986827becc4515dd736f366\"},\"breadcrumb\":{\"@id\":\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/\"]}]},{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#primaryimage\",\"url\":\"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg\",\"contentUrl\":\"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg\",\"width\":800,\"height\":250},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\/\/www.sicklecelldisease.org\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\/\/www.sicklecelldisease.org\/#website\",\"url\":\"https:\/\/www.sicklecelldisease.org\/\",\"name\":\"Sickle Cell Disease Association of America Inc.\",\"description\":\"\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\/\/www.sicklecelldisease.org\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"},{\"@type\":\"Person\",\"@id\":\"https:\/\/www.sicklecelldisease.org\/#\/schema\/person\/efc0c6b0f986827becc4515dd736f366\",\"name\":\"admin\",\"image\":{\"@type\":\"ImageObject\",\"inLanguage\":\"en-US\",\"@id\":\"https:\/\/www.sicklecelldisease.org\/#\/schema\/person\/image\/\",\"url\":\"https:\/\/secure.gravatar.com\/avatar\/2b80c3a97c5df05f644e48a1b6c6a1f0?s=96&d=mm&r=g\",\"contentUrl\":\"https:\/\/secure.gravatar.com\/avatar\/2b80c3a97c5df05f644e48a1b6c6a1f0?s=96&d=mm&r=g\",\"caption\":\"admin\"},\"url\":\"https:\/\/www.sicklecelldisease.org\/author\/admin\/\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease - Sickle Cell Disease Association of America Inc.","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:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/","og_locale":"en_US","og_type":"article","og_title":"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease - Sickle Cell Disease Association of America Inc.","og_description":"CRISPR Therapeutics and Vertex Pharmaceuticals Incorporated today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for CTX001 for the treatment of sickle cell disease (SCD). CTX001 is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. The FDA\u2019s Fast Track program is designed to [&hellip;]","og_url":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/","og_site_name":"Sickle Cell Disease Association of America Inc.","article_published_time":"2019-01-04T17:40:23+00:00","og_image":[{"width":800,"height":250,"url":"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg","type":"image\/jpeg"}],"author":"admin","twitter_card":"summary_large_image","twitter_misc":{"Written by":"admin","Est. reading time":"1 minute"},"schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/","url":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/","name":"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease - Sickle Cell Disease Association of America Inc.","isPartOf":{"@id":"https:\/\/www.sicklecelldisease.org\/#website"},"primaryImageOfPage":{"@id":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#primaryimage"},"image":{"@id":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#primaryimage"},"thumbnailUrl":"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg","datePublished":"2019-01-04T17:40:23+00:00","dateModified":"2019-01-04T17:40:23+00:00","author":{"@id":"https:\/\/www.sicklecelldisease.org\/#\/schema\/person\/efc0c6b0f986827becc4515dd736f366"},"breadcrumb":{"@id":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/"]}]},{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#primaryimage","url":"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg","contentUrl":"https:\/\/www.sicklecelldisease.org\/wp-content\/uploads\/2019\/01\/crispervertex.jpg","width":800,"height":250},{"@type":"BreadcrumbList","@id":"https:\/\/www.sicklecelldisease.org\/2019\/01\/04\/crispr-therapeutics-and-vertex-announce-fda-fast-track-designation-for-ctx001-for-the-treatment-of-sickle-cell-disease\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/www.sicklecelldisease.org\/"},{"@type":"ListItem","position":2,"name":"CRISPR Therapeutics and Vertex Announce FDA Fast Track Designation for CTX001 for the Treatment of Sickle Cell Disease"}]},{"@type":"WebSite","@id":"https:\/\/www.sicklecelldisease.org\/#website","url":"https:\/\/www.sicklecelldisease.org\/","name":"Sickle Cell Disease Association of America Inc.","description":"","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/www.sicklecelldisease.org\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"},{"@type":"Person","@id":"https:\/\/www.sicklecelldisease.org\/#\/schema\/person\/efc0c6b0f986827becc4515dd736f366","name":"admin","image":{"@type":"ImageObject","inLanguage":"en-US","@id":"https:\/\/www.sicklecelldisease.org\/#\/schema\/person\/image\/","url":"https:\/\/secure.gravatar.com\/avatar\/2b80c3a97c5df05f644e48a1b6c6a1f0?s=96&d=mm&r=g","contentUrl":"https:\/\/secure.gravatar.com\/avatar\/2b80c3a97c5df05f644e48a1b6c6a1f0?s=96&d=mm&r=g","caption":"admin"},"url":"https:\/\/www.sicklecelldisease.org\/author\/admin\/"}]}},"_links":{"self":[{"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/posts\/2115","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/comments?post=2115"}],"version-history":[{"count":0,"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/posts\/2115\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/media\/1376"}],"wp:attachment":[{"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/media?parent=2115"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/categories?post=2115"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.sicklecelldisease.org\/wp-json\/wp\/v2\/tags?post=2115"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}