{"id":176141,"date":"2023-11-16T11:23:04","date_gmt":"2023-11-16T17:23:04","guid":{"rendered":"https:\/\/lifeboat.com\/blog\/2023\/11\/u-k-greenlights-worlds-first-crispr-gene-editing-therapy"},"modified":"2023-11-16T11:23:04","modified_gmt":"2023-11-16T17:23:04","slug":"u-k-greenlights-worlds-first-crispr-gene-editing-therapy","status":"publish","type":"post","link":"https:\/\/lifeboat.com\/blog\/2023\/11\/u-k-greenlights-worlds-first-crispr-gene-editing-therapy","title":{"rendered":"U.K. Greenlights World\u2019s First Crispr Gene Editing Therapy"},"content":{"rendered":"<p><a class=\"aligncenter blog-photo\" href=\"https:\/\/lifeboat.com\/blog.images\/u-k-greenlights-worlds-first-crispr-gene-editing-therapy.jpg\"><\/a><\/p>\n<p>The one-time treatment helped relieve symptoms of disease and could free patients from the need for bone marrow transplants or regular blood transfusions, Beach said, adding that the drug hopefully offers a permanent fix for the condition.<\/p>\n<p>The MHRA said it identified no significant safety concerns during the trials and will continue to closely monitor Casgevy\u2019s safety after approval.<\/p>\n<p>Vertex CEO and President Reshma Kewalramanit <a href=\"https:\/\/investors.vrtx.com\/news-releases\/news-release-details\/vertex-and-crispr-therapeutics-announce-authorization-first\" target=\"_blank\" class=\"\" title=\"https:\/\/investors.vrtx.com\/news-releases\/news-release-details\/vertex-and-crispr-therapeutics-announce-authorization-first\" rel=\"nofollow noopener noreferrer\" aria-label=\"celebrated\">celebrated<\/a> Casgevy\u2019s approval as \u201ca historic day in science and medicine\u201d and Samarth Kulkarni, CEO and Chairman of Crispr Therapeutics, said it will hopefully mark \u201cthe first of many applications of this Nobel Prize winning technology to benefit eligible patients with serious diseases.\u201d The two companies are hoping for similarly positive decisions from the MHRA\u2019s counterparts in the Europe Union and the U.S., which are in the process of evaluating Casgevy, also known as exa-cel. The Food and Drug Administration is expected to make a decision in early December and has a <a href=\"https:\/\/www.npr.org\/sections\/health-shots\/2023\/10\/31\/1208041252\/a-landmark-gene-editing-treatment-for-sickle-cell-disease-moves-closer-to-realit\" target=\"_blank\" class=\"\" title=\"https:\/\/www.npr.org\/sections\/health-shots\/2023\/10\/31\/1208041252\/a-landmark-gene-editing-treatment-for-sickle-cell-disease-moves-closer-to-realit\" rel=\"nofollow noopener noreferrer\" aria-label=\"deadline\">deadline<\/a> of December 8. The agency appears poised to follow the MHRA and approve the treatment, with its advisors confident of the drug\u2019s efficacy and benefit but <a href=\"https:\/\/www.reuters.com\/business\/healthcare-pharmaceuticals\/fda-panel-says-vertexcrispr-assess-safety-risks-gene-therapy-follow-up-study-2023-10-31\/\" target=\"_blank\" class=\"\" title=\"https:\/\/www.reuters.com\/business\/healthcare-pharmaceuticals\/fda-panel-says-vertexcrispr-assess-safety-risks-gene-therapy-follow-up-study-2023-10-31\/\" rel=\"nofollow noopener noreferrer\" aria-label=\"wary\">wary<\/a> of theoretical unintended consequences of genetic modifications.<\/p>\n","protected":false},"excerpt":{"rendered":"<p>The one-time treatment helped relieve symptoms of disease and could free patients from the need for bone marrow transplants or regular blood transfusions, Beach said, adding that the drug hopefully offers a permanent fix for the condition. The MHRA said it identified no significant safety concerns during the trials and will continue to closely monitor [\u2026]<\/p>\n","protected":false},"author":511,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1902,11,412],"tags":[],"class_list":["post-176141","post","type-post","status-publish","format-standard","hentry","category-bioengineering","category-biotech-medical","category-genetics"],"_links":{"self":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/176141","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/users\/511"}],"replies":[{"embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/comments?post=176141"}],"version-history":[{"count":0,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/posts\/176141\/revisions"}],"wp:attachment":[{"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/media?parent=176141"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/categories?post=176141"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/lifeboat.com\/blog\/wp-json\/wp\/v2\/tags?post=176141"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}