{"id":10752,"date":"2018-08-31T17:36:12","date_gmt":"2018-08-31T15:36:12","guid":{"rendered":"https:\/\/www.hollandbio.nl\/?p=10752"},"modified":"2018-09-04T17:39:49","modified_gmt":"2018-09-04T15:39:49","slug":"crispr-therapeutics-and-vertex-start-first-company-backed-human-crispr-trial","status":"publish","type":"post","link":"https:\/\/www.hollandbio.nl\/en\/nieuws\/crispr-therapeutics-and-vertex-start-first-company-backed-human-crispr-trial\/","title":{"rendered":"CRISPR Therapeutics and Vertex start first company-backed human CRISPR trial"},"content":{"rendered":"<p>While research groups in China have already started testing CRISPR in humans, CRISPR Therapeutics and Vertex Pharmaceuticals are the first companies to sponsor a human trial of the gene editing technology. The trial will take place at a single site in Germany and will test a gene therapy in patients with beta thalassemia.<\/p>\n<div>\n<p xml:lang=\"EN-US\">Vertex\u00a0<a href=\"https:\/\/www.fiercebiotech.com\/biotech\/vertex-licenses-blood-disorder-gene-therapy-from-crispr-therapeutics\" target=\"_blank\" rel=\"noopener noreferrer\">licensed<\/a>\u00a0CTX001, an autologous gene-edited hematopoietic stem cell therapy, from CRISPR in December. It was the first CRISPR-based treatment to come out of a four-year, $105 million deal the pair struck in 2015. At the time, Vertex\u00a0<a href=\"https:\/\/www.fiercebiotech.com\/biotech\/updated-vertex-pays-105m-to-kick-off-gene-editing-pact-crispr-therapeutics\" target=\"_blank\" rel=\"noopener noreferrer\">paid up<\/a>\u00a0$75 million in cash and took a $30 million stake in CRISPR Therapeutics in exchange for the right to license up to six gene-editing programs. CTX001 is being developed for the blood disorders sickle cell disease and beta thalassemia.<\/p>\n<\/div>\n<p>Both disorders are caused by mutations in the beta-globin gene, which codes for a part of hemoglobin, the oxygen-carrying component of red blood cells. This results in missing or defective hemoglobin. CTX001 was developed on the knowledge that fetal hemoglobin\u2014found in newborn babies but later replaced by adult hemoglobin\u2014can be protective in adults who have blood disorders.<\/p>\n<p>CTX001 uses CRISPR gene-editing ex vivo\u2014that is, outside the body. A patient\u2019s cells are harvested and edited to increase fetal hemoglobin levels in the patient\u2019s blood cells. The edited cells are then infused back into the patient where they are expected to produce blood cells with fetal hemoglobin and compensate for defective adult hemoglobin.<\/p>\n<p>The new trial will test CTX001 in up to 12 adult patients who have transfusion-dependent beta thalassemia,\u00a0<a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03655678\" target=\"_blank\" rel=\"noopener noreferrer\">according<\/a>\u00a0to an announcement on Clinicaltrials.gov. It will take place at a single hospital in Regensburg, Germany.<\/p>\n<p>\u201cCRISPR Therapeutics and Vertex are advancing CTX001 as the first gene editing treatment for both sickle cell disease and beta thalassemia using the CRISPR\/Cas9 technology, and have now opened the beta thalassemia study for enrollment in a Phase 1\/2 trial in Europe,\u201d said a CRISPR Therapeutics spokesperson via email. \u201cThis is one important step of many toward bringing the promise of this new technology to patients with serious diseases like SCD and beta thalassemia, and we are thrilled to be at the forefront of what we believe may be a fundamental change in the treatment of disease.\u201d<\/p>\n<div>\n<p xml:lang=\"EN-US\">The partners have been planning a U.S. trial testing the treatment in sickle cell disease, but its IND\u00a0<a href=\"https:\/\/www.fiercebiotech.com\/biotech\/fda-puts-vertex-crispr-sickle-cell-trial-hold\" target=\"_blank\" rel=\"noopener noreferrer\">ran into<\/a>\u00a0an FDA clinical hold in May. Details were few and far between and shares in CRISPR slipped 15% at the time.<\/p>\n<\/div>\n<p>For a time, it looked like Editas Medicine would be the first to launch a human trial, with plans to start testing a CRISPR treatment for a rare form of blindness in 2017. Manufacturing issues delayed the timeline to 2018, with CEO Katrine Bosley saying in the company\u2019s second-quarter update that the IND filing is slated for October.<\/p>\n<p>Source: <a href=\"https:\/\/www.fiercebiotech.com\/biotech\/crispr-therapeutics-vertex-start-first-company-backed-human-crispr-trial\"><span style=\"text-decoration: underline;\"><strong>FierceBiotech<\/strong><\/span><\/a><\/p>\n","protected":false},"excerpt":{"rendered":"<p>While research groups in China have already started testing CRISPR in humans, CRISPR Therapeutics and Vertex Pharmaceuticals are the first companies to sponsor a human trial of the gene editing technology. The trial will take place at a single site in Germany and will test a gene therapy in patients with beta thalassemia. Vertex\u00a0licensed\u00a0CTX001, an [&hellip;]<\/p>\n","protected":false},"author":6,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[39],"tags":[],"class_list":["post-10752","post","type-post","status-publish","format-standard","hentry","category-gezondheid"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.0 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>CRISPR Therapeutics and Vertex start first company-backed human CRISPR trial - hollandbio<\/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.hollandbio.nl\/en\/nieuws\/crispr-therapeutics-and-vertex-start-first-company-backed-human-crispr-trial\/\" \/>\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 start first company-backed human CRISPR trial - hollandbio\" \/>\n<meta property=\"og:description\" content=\"While research groups in China have already started testing CRISPR in humans, CRISPR Therapeutics and Vertex Pharmaceuticals are the first companies to sponsor a human trial of the gene editing technology. The trial will take place at a single site in Germany and will test a gene therapy in patients with beta thalassemia. 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