A sickle cell cure exists. But patients need more than just gene therapy.

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But patients need more than just gene therapy." /> <meta name="medium" content="news" /> <meta name="date" content="2024-11-01" /> <meta name="twitter:card" content="summary_large_image"> <meta name="twitter:domain" content="PharmaVoice"> <meta name="twitter:site" content="@PharmaVoice"> <meta property="og:title" name="og:title" content="A sickle cell cure exists. But patients need more than just gene therapy." /> <meta property="og:site_name" name="og:site_name" content="PharmaVoice" /> <meta property="og:type" name="og:type" content="article" /> <meta name="article_date_original" content="Friday, November 01, 2024, 07:57 AM" /> <meta property="og:url" name="og:url" content="https://www.pharmavoice.com/news/gene-therapy-sickle-cell-crispr-cellarity-vertex-pfizer/731542/" /> <meta property="og:image" name="og:image" content="https://imgproxy.divecdn.com/f5Z3w1_9fxOo5MT5bPSVKaNjx5xLAVZixjWdGxIYibU/g:ce/rs:fit:770:435/Z3M6Ly9kaXZlc2l0ZS1zdG9yYWdlL2RpdmVpbWFnZS9HZXR0eUltYWdlcy0xMjIyOTc2MjI3LmpwZw==.webp" /> <meta property="og:description" name="og:description" content="Almost a year after two historic approvals, Pfizer is pulling a sickle cell disease treatment from the market, and the outlook for patients feels shakier." /> <meta name="sailthru.date" content="2024-11-01T07:57:56" /> <meta name="sailthru.expire_date" content="2034-10-20T07:57:56" /> <meta name="sailthru.author" content="Alexandra Pecci" /> <meta name="sailthru.image.full" content="https://imgproxy.divecdn.com/f5Z3w1_9fxOo5MT5bPSVKaNjx5xLAVZixjWdGxIYibU/g:ce/rs:fit:770:435/Z3M6Ly9kaXZlc2l0ZS1zdG9yYWdlL2RpdmVpbWFnZS9HZXR0eUltYWdlcy0xMjIyOTc2MjI3LmpwZw==.webp" />  <meta name="sailthru.image.thumb" content="https://imgproxy.divecdn.com/cJShq4KpdWKiiljAsDIfTZVDj9hdtcfZSqzaCN_f6qs/g:ce/rs:fit:106:60/Z3M6Ly9kaXZlc2l0ZS1zdG9yYWdlL2RpdmVpbWFnZS9HZXR0eUltYWdlcy0xMjIyOTc2MjI3LmpwZw==.webp" />  <meta name="sailthru.description" content="Almost a year after two historic approvals, Pfizer is pulling a sickle cell disease treatment from the market, and the outlook for patients feels shakier." /> <link rel="stylesheet" href="/static/css/pharmavoice_app.css?510416050225"> <script> // Initialize dataLayer empty array for GTM events window.dataLayer = window.dataLayer || []; window.dataLayer.push({ 'site': 'PharmaVoice', }); // wrapper function to push GTM events to dataLayer pushGTMEvent = (eventObject) => { // if window.datalayer is not defined, then set to an empty array window.dataLayer.push(eventObject); }; </script>  <script>(function(w,d,s,l,i){w[l]=w[l]||[];w[l].push({'gtm.start': new Date().getTime(),event:'gtm.js'});var f=d.getElementsByTagName(s)[0], j=d.createElement(s),dl=l!='dataLayer'?'&l='+l:'';j.async=true;j.src= 'https://www.googletagmanager.com/gtm.js?id='+i+dl;f.parentNode.insertBefore(j,f); })(window,document,'script','dataLayer','GTM-55G8WRR');</script>  <script> let eventParams = {}; eventParams.author = "Meagan Parrish"; eventParams.date_published_readable = "2024-11-01"; eventParams.post_type = "Article"; eventParams.tags = "Blood Disorders, Gene Therapy"; eventParams.topics = "Biotech, Patient, Pharma, Research & Development"; eventParams.clone = "False"; const gtmEventObject = { event: 'page.content', 'page.content': { v:11, // seer version ...eventParams } }; window.pushGTMEvent(gtmEventObject); </script> <script type="module" src="/static/js/dist/lyticsHelper.bundle.js?290716050225" ></script> <script type="text/javascript" src="/static/js/ad_blocker_recovery.js?160016291122"></script>  <script async src="https://fundingchoicesmessages.google.com/i/pub-8553262192892817?ers=1" nonce="RORABhIXgMhQdZXbks8fZQ"></script> <script nonce="RORABhIXgMhQdZXbks8fZQ">(function() {function signalGooglefcPresent() {if (!window.frames['googlefcPresent']) {if (document.body) {const iframe = document.createElement('iframe'); iframe.style = 'width: 0; height: 0; border: none; z-index: -1000; left: -1000px; top: -1000px;'; iframe.style.display = 'none'; iframe.name = 'googlefcPresent'; document.body.appendChild(iframe);} else {setTimeout(signalGooglefcPresent, 0);}}}signalGooglefcPresent();})();</script>  <script>(function(){/* Copyright The Closure Library Authors. 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Any arguments passed to .trigger() will also be passed along // to the associated function function waitForTriggerHandler() { var function_queue = []; var trigger_arguments = []; this._triggered = false; this.push = function(fn) { if (this._triggered) { // just run it immediately if handler has already been triggered fn.apply(null, trigger_arguments); } else { // otherwise add to the queue to run later function_queue.push(fn); } }; this.trigger = function() { // any arguments passed to trigger(foo, bar, baz) will also be passed along to functions in the queue // first convert any arguments to an array trigger_arguments = Array.prototype.slice.call(arguments); while (function_queue.length){ // for each function in the queue, pop it off and then execute it while passing in any // args that may have been passed to trigger(). function_queue.pop().apply(null, trigger_arguments); // The "null" above is for the value of "this". // See https://developer.mozilla.org/en-US/docs/Web/JavaScript/Reference/Global_Objects/Function/apply#Parameters } this._triggered = true; } } // set up waitToLoadAds handler window.waitToLoadAds = new waitForTriggerHandler(); // We set up the triggers for this below, they depend on whether we're loading lytics on the page </script> <script type="text/javascript"> // This token is here for test_lytics_snippet.py test: lytics-is-enabled window.googletag = window.googletag || {}; googletag.cmd = googletag.cmd || []; // initialize callback (source: https://learn.lytics.com/product-docs/lytics-javascript-tag/receiving-data-from-lytics) !function(){"use strict";var o=window.jstag||(window.jstag={}),r=[];function n(e){o[e]=function(){for(var n=arguments.length,t=new Array(n),i=0;i<n;i++)t[i]=arguments[i];r.push([e,t])}}n("send"),n("mock"),n("identify"),n("pageView"),n("unblock"),n("getid"),n("setid"),n("loadEntity"),n("getEntity"),n("on"),n("once"),n("call"),o.loadScript=function(n,t,i){var e=document.createElement("script");e.async=!0,e.src=n,e.onload=t,e.onerror=i;var o=document.getElementsByTagName("script")[0],r=o&&o.parentNode||document.head||document.body,c=o||r.lastChild;return null!=c?r.insertBefore(e,c):r.appendChild(e),this},o.init=function n(t){return this.config=t,this.loadScript(t.src,function(){if(o.init===n)throw new Error("Load error!");o.init(o.config),function(){for(var n=0;n<r.length;n++){var t=r[n][0],i=r[n][1];o[t].apply(o,i)}r=void 0}()}),this}}(); // custom init + lytics default install tag from https://activate.getlytics.com/connect?aid=2751 // see https://learn.lytics.com/understanding/product-docs/lytics-javascript-tag/configuration#configuration-options var lyticsCid = '44fda7674f94228540d8fba6786f9770'; console.log('You are using the Lytics Prod account'); jstag.init({ src: `https://c.lytics.io/api/tag/${lyticsCid}/latest.min.js`, pageAnalysis: { dataLayerPull: { disabled: true } } }); jstag.pageView(); (function(){ var sailthru_cookie, sailthru_user_id; function read_cookie(cookieName) { var re = new RegExp('[; ]'+cookieName+'=([^\\s;]*)'); var sMatch = (' '+document.cookie).match(re); if (cookieName && sMatch) return unescape(sMatch[1]); return ''; } sailthru_cookie = read_cookie('sailthru_hid'); if (sailthru_cookie.length > 56) { sailthru_user_id = sailthru_cookie.slice(32,56); jstag.send({ st_profile_id: sailthru_user_id }); } })(); jstag.call('entityReady', waitToLoadAds.trigger); setTimeout(waitToLoadAds.trigger, 1000); // also trigger lytics if it's taking too long waitToLoadAds.push(function (lytics_data) { // lytics_data won't be defined if lytics failed to load correctly. In that case // we don't need to set any segments. if (lytics_data && lytics_data.segments) { googletag.cmd.push(function() { googletag.pubads().setTargeting("LyticsSegments", lytics_data.segments); }); } }); function getUID(id) { window.dataLayer.push({'user_id': id}); }; jstag.getid(getUID); </script> <script type="text/javascript" src="/static/js/mailcheck.min.js?192616240919"></script> <script type="text/javascript" src="/static/js/dive_mailcheck.js?192616240919"></script> <script src="/static/js/app.js?262317060225"></script> <script type="text/javascript"> var divecounter_settings = {}; divecounter_settings.showSignupAd = true; </script> <script type="text/javascript" src="/static/js/divecounter.js?261616040423"></script> <script type="text/javascript"> // Special unbounce signup thing for google traffic. Test with ?test_unbounce in the URL // Note: divecounter must already be loaded. Only enabled on pages that would normally have signup ads // Note: showSignupAd is always off if prestitial is enabled! if ((typeof divecounter !== "undefined") && ($(window).width() > 640)) { var test_unbounce = (window.location.href.indexOf("?test_unbounce")>=0); var force_unbounce = (window.location.href.indexOf("signupbounce=1")>=0) || (window.location.href.indexOf("utm_campaign=Linked-HR")>=0) || (window.location.href.indexOf("utm_source=outbrain")>=0); if (test_unbounce || force_unbounce || (document.referrer.match(/^https?:\/\/(www\.google\.|news\.google\.|t\.co|twitter\.com|www\.facebook\.com)/i) && divecounter.getOption('showSignupAd') && divecounter.canShow(1,1)) ) { // dyanmically load ouibounce.js only on pages where its needed $.getScript("/static/js/ouibounce.min.js?102717100918", function(){ // Once ouibounce.js loads, call the script // We're reusing divecounter's modal instead of OuiBounce's modal code (that's why we pass it 'false') var _ouibounce = ouibounce(false, { aggressive: test_unbounce, // ignore cookie in test mode cookieName: '_viewedOuiBounce', // underscored cookies should be ignored by Varnish callback: function() { divecounter_open_ad(); } }); }); } // end should show unbounce } // end divecounter defined </script> <script type="module" src="/static/js/dist/googleAnalyticsHelper.bundle.js?290716050225" ></script> <script type="text/javascript"> function read_cookie(cookieName) { var re = new RegExp('[; ]'+cookieName+'=([^\\s;]*)'); var sMatch = (' '+document.cookie).match(re); if (cookieName && sMatch) return unescape(sMatch[1]); return ''; } var script = document.createElement('script'); script.type = 'text/javascript'; script.async = true; script.addEventListener('load', function(e) { // we only pass the template this context variable on newsposts let informaData = false; informaData = {"canonicalUrl": "https://www.pharmavoice.com/news/gene-therapy-sickle-cell-crispr-cellarity-vertex-pfizer/731542/", "pageIsSponsored": false, "pageSponsor": "", "contentAuthor": "Alexandra Pecci", "contentPubDate": "2024-11-01 07:57:56", "pageID": "newspost 731542", "pageTitle": "A sickle cell cure exists. But patients need more than just gene therapy.", "pageType": "article", "primaryTerm": "Biotech, Patient, Pharma, Research & Development", "secondaryTerm": "Blood Disorders, Gene Therapy"}; if (window.IIRISTracker) { var sailthru_id = read_cookie('sailthru_hid'); if (sailthru_id.length > 56) { sailthru_id = sailthru_id.slice(32,56); } var notProduction = 'False' === 'True'; var domain = 'www.pharmavoice.com'.split('.')[1]; // on non-prod environment, there is specific collector domain to send the data to. var cDomain = notProduction ? 'com-informa-prod1.mini.snplow.net' : 'c.' + domain + '.com' // removed .com previous line var cfg = { config: { td: {}, snowplow: { context: {} } } }; cfg.config.idsource = 'sailthruid'; cfg.config.snowplow.appId = domain; cfg.config.snowplow.cDomain = cDomain; cfg.config.td.lob = 'it'; cfg.config.td.domain = domain; cfg.config.snowplow.contentTagEnabled = !!informaData cfg.config.snowplow.context = informaData window.IIRISTracker.initConfig(cfg); window.IIRISTracker.setTrackingUID(sailthru_id); var irisEvt = new Event('trackerLoaded'); window.dispatchEvent(irisEvt); } else { window.addEventListener('trackerLoaded', function(evt) { if(window.IIRISTracker) { window.IIRISTracker.setTrackingUID(sailthru_id); } }); } }); script.src = 'https://static.iris.informa.com/widgets/v3.0/iris-t.js'; document.getElementsByTagName('head')[0].appendChild(script); </script> <script type="text/javascript"> jQuery.browser = {}; (function () { jQuery.browser.msie = false; jQuery.browser.version = 0; if (navigator.userAgent.match(/MSIE ([0-9]+)\./)) { jQuery.browser.msie = true; jQuery.browser.version = RegExp.$1; } })(); </script>  <script type="text/javascript" src="/static/js/jquery.simplemodal.divefork.min.js?093716120922"></script> <style> /* dynamic style overrides based on DB go here*/ </style> <meta name="sailthru.title" content="A sickle cell cure exists. But patients need more than just gene therapy." /> <meta name="sailthru.post_type" content="Article" /> <meta name="sailthru.dive_awards" content="False" /> <meta name="sailthru.post_updated" content="False" /> <meta name="lytics:topics" content="Pharma, Research & Development, Patient, Biotech, Blood Disorders, Gene Therapy" /> <meta name="robots" content="max-image-preview:large"> <meta name="news_keywords" content="Biotech,biotech,Patient,patient,Pharma,pharma,Research & Development,research-development"/> <script type="text/javascript"> /** Stolen from http://stackoverflow.com/questions/24816/escaping-html-strings-with-jquery **/ $(document).ready(function () { //"Tweet this" pullquote sections // This is used in the processing of 'tweet this' pull quotes function escapeHtml(string) { var entityMap = { "&": "&", "<": "<", ">": ">", '"': '"', "'": ''', "/": '/' }; return String(string).replace(/[&<>"'\/]/g, function (s) { return entityMap[s]; }); } // Process the twitter pullquote sections: // * create the link // * create the text ('tweet this quote') for the link $(".pquote").each(function (index) { var j_words; if ($(this).data('twitter-text') != 'undefined') { j_words = $(this).children('.tweetwords').first(); } else { j_words = $(this).data('twitter-text'); } var j_replace = $(this).children('.tweetquote').first(); var clean_text = escapeHtml(j_words.text()); var link_attributes = 'href="https://twitter.com/intent/tweet?text=' + clean_text + '%20https%3A//www.pharmavoice.com/news/gene-therapy-sickle-cell-crispr-cellarity-vertex-pfizer/731542/' + '" class="popup button twitter" data-sharebutton="tweet_quote" data-shareusing="twitter"'; var img = '<img src="/static/img/social_icons/twitter-follow-icon.png?102717100918" alt="Share on Twitter" loading="lazy">'; var link = '<a ' + link_attributes + '>Tweet this quote' + img + '</a>'; j_replace.html(link); }); }); </script> <script type="module" src="/static/js/dist/signupReferrer.bundle.js?290716050225" ></script> <script data-cfasync="false" type="text/javascript" src="/static/js/prestitial.js?270116210524"></script> <script async src="https://securepubads.g.doubleclick.net/tag/js/gpt.js"></script> <script src="/static/js/dist/signupDataGam.bundle.js?290716050225"></script> <script type="text/javascript" src="/static/js/dist/adTemplates.bundle.js?290716050225"></script> <script> var eventMethod = window.addEventListener ? "addEventListener" : "attachEvent"; var eventer = window[eventMethod]; var messageEvent = eventMethod == "attachEvent" ? "onmessage" : "message"; // Build the site_logos object to be later referenced in house ads const site_logos = {}; site_logos['diveapi'] = { domain: 'diveapi', site_name: "API Site", alt_text: "API Site logo", is_current_site: false, white: { png: "None", svg: "None", }, black: { png: "None", svg: "None", } }; site_logos['agriculturedive'] = { domain: 'agriculturedive', site_name: "Agriculture Dive", alt_text: "Agriculture Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/agriculture_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/agriculture_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/agriculture_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/agriculture_black.svg", } }; site_logos['automotivedive'] = { domain: 'automotivedive', site_name: "Automotive Dive", alt_text: "Automotive Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/automotive_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/automotive_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/automotive_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/automotive_black.svg", } }; site_logos['bankingdive'] = { domain: 'bankingdive', site_name: "Banking Dive", alt_text: "Banking Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/banking_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/banking_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/banking_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/banking_black.svg", } }; site_logos['biopharmadive'] = { domain: 'biopharmadive', site_name: "BioPharma Dive", alt_text: "BioPharma Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/biopharma_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/biopharma_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/biopharma_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/biopharma_black.svg", } }; site_logos['cfo'] = { domain: 'cfo', site_name: "CFO.com", alt_text: "CFO.com logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfodotcom_white.svg", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfodotcom_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfodotcom_black.svg", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfodotcom_black.svg", } }; site_logos['cfodive'] = { domain: 'cfodive', site_name: "CFO Dive", alt_text: "CFO Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfo_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfo_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfo_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cfo_black.svg", } }; site_logos['ciodive'] = { domain: 'ciodive', site_name: "CIO Dive", alt_text: "CIO Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cio_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cio_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cio_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cio_black.svg", } }; site_logos['constructiondive'] = { domain: 'constructiondive', site_name: "Construction Dive", alt_text: "Construction Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/construction_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/construction_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/construction_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/construction_black.svg", } }; site_logos['cstoredive'] = { domain: 'cstoredive', site_name: "C-Store Dive", alt_text: "C-Store Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cstore_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cstore_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cstore_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cstore_black.svg", } }; site_logos['customerexperiencedive'] = { domain: 'customerexperiencedive', site_name: "CX Dive", alt_text: "CX Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cx_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cx_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cx_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cx_black.svg", } }; site_logos['cybersecuritydive'] = { domain: 'cybersecuritydive', site_name: "Cybersecurity Dive", alt_text: "Cybersecurity Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cybersecurity_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cybersecurity_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cybersecurity_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/cybersecurity_black.svg", } }; site_logos['educationdive'] = { domain: 'educationdive', site_name: "Education Dive", alt_text: "Education Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/education_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/education_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/education_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/education_black.svg", } }; site_logos['esgdive'] = { domain: 'esgdive', site_name: "ESG Dive", alt_text: "ESG Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/esg_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/esg_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/esg_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/esg_black.svg", } }; site_logos['facilitiesdive'] = { domain: 'facilitiesdive', site_name: "Facilities Dive", alt_text: "Facilities Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/facilities_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/facilities_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/facilities_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/facilities_black.svg", } }; site_logos['fashiondive'] = { domain: 'fashiondive', site_name: "Fashion Dive", alt_text: "Fashion Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/fashion_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/fashion_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/fashion_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/fashion_black.svg", } }; site_logos['fooddive'] = { domain: 'fooddive', site_name: "Food Dive", alt_text: "Food Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/food_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/food_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/food_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/food_black.svg", } }; site_logos['grocerydive'] = { domain: 'grocerydive', site_name: "Grocery Dive", alt_text: "Grocery Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/grocery_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/grocery_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/grocery_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/grocery_black.svg", } }; site_logos['healthcaredive'] = { domain: 'healthcaredive', site_name: "Healthcare Dive", alt_text: "Healthcare Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/healthcare_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/healthcare_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/healthcare_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/healthcare_black.svg", } }; site_logos['highereddive'] = { domain: 'highereddive', site_name: "Higher Ed Dive", alt_text: "Higher Ed Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/highered_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/highered_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/highered_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/highered_black.svg", } }; site_logos['hoteldive'] = { domain: 'hoteldive', site_name: "Hotel Dive", alt_text: "Hotel Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hotel_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hotel_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hotel_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hotel_black.svg", } }; site_logos['hrdive'] = { domain: 'hrdive', site_name: "HR Dive", alt_text: "HR Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hr_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hr_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hr_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/hr_black.svg", } }; site_logos['k12dive'] = { domain: 'k12dive', site_name: "K-12 Dive", alt_text: "K-12 Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/k12_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/k12_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/k12_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/k12_black.svg", } }; site_logos['legaldive'] = { domain: 'legaldive', site_name: "Legal Dive", alt_text: "Legal Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/legal_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/legal_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/legal_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/legal_black.svg", } }; site_logos['manufacturingdive'] = { domain: 'manufacturingdive', site_name: "Manufacturing Dive", alt_text: "Manufacturing Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/manufacturing_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/manufacturing_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/manufacturing_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/manufacturing_black.svg", } }; site_logos['marketingdive'] = { domain: 'marketingdive', site_name: "Marketing Dive", alt_text: "Marketing Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/marketing_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/marketing_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/marketing_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/marketing_black.svg", } }; site_logos['medtechdive'] = { domain: 'medtechdive', site_name: "MedTech Dive", alt_text: "MedTech Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/medtech_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/medtech_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/medtech_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/medtech_black.svg", } }; site_logos['mobilemarketer'] = { domain: 'mobilemarketer', site_name: "Mobile Marketer", alt_text: "Mobile Marketer logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/mobilemarketer_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/mobilemarketer_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/mobilemarketer_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/mobilemarketer_black.svg", } }; site_logos['multifamilydive'] = { domain: 'multifamilydive', site_name: "Multifamily Dive", alt_text: "Multifamily Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/multifamily_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/multifamily_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/multifamily_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/multifamily_black.svg", } }; site_logos['packagingdive'] = { domain: 'packagingdive', site_name: "Packaging Dive", alt_text: "Packaging Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/packaging_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/packaging_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/packaging_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/packaging_black.svg", } }; site_logos['paymentsdive'] = { domain: 'paymentsdive', site_name: "Payments Dive", alt_text: "Payments Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/payments_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/payments_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/payments_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/payments_black.svg", } }; site_logos['pharmavoice'] = { domain: 'pharmavoice', site_name: "PharmaVoice", alt_text: "PharmaVoice logo", is_current_site: true, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/pharmavoice_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/pharmavoice_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/pharmavoice_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/pharmavoice_black.svg", } }; site_logos['proformative'] = { domain: 'proformative', site_name: "Proformative", alt_text: "Proformative logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/proformative_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/proformative_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/proformative_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/proformative_black.svg", } }; site_logos['restaurantdive'] = { domain: 'restaurantdive', site_name: "Restaurant Dive", alt_text: "Restaurant Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/restaurant_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/restaurant_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/restaurant_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/restaurant_black.svg", } }; site_logos['retaildive'] = { domain: 'retaildive', site_name: "Retail Dive", alt_text: "Retail Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/retail_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/retail_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/retail_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/retail_black.svg", } }; site_logos['smartcitiesdive'] = { domain: 'smartcitiesdive', site_name: "Smart Cities Dive", alt_text: "Smart Cities Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smartcities_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smartcities_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smartcities_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smartcities_black.svg", } }; site_logos['socialmediatoday'] = { domain: 'socialmediatoday', site_name: "Social Media Today", alt_text: "Social Media Today logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smt_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smt_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smt_color.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/smt_color.svg", } }; site_logos['supplychaindive'] = { domain: 'supplychaindive', site_name: "Supply Chain Dive", alt_text: "Supply Chain Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/supplychain_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/supplychain_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/supplychain_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/supplychain_black.svg", } }; site_logos['truckingdive'] = { domain: 'truckingdive', site_name: "Trucking Dive", alt_text: "Trucking Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/trucking_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/trucking_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/trucking_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/trucking_black.svg", } }; site_logos['utilitydive'] = { domain: 'utilitydive', site_name: "Utility Dive", alt_text: "Utility Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/utility_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/utility_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/utility_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/utility_black.svg", } }; site_logos['wastedive'] = { domain: 'wastedive', site_name: "Waste Dive", alt_text: "Waste Dive logo", is_current_site: false, white: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/waste_white.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/waste_white.svg", }, black: { png: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/waste_black.png", svg: "https://d12v9rtnomnebu.cloudfront.net/logo/publications/waste_black.svg", } }; // Get the dive domain and return the dive logo object, or null if it doesn't exist in site_logos function getDivesiteLogoObject(link_url, alt_domain='') { let dive_domain; // Split link URL on 'adurl=' and take what comes after as the ad url const ad_url = decodeURIComponent(link_url.split('adurl=')[1]); // Split on . to get an array of hostname segments const dive_domain_array = new URL(ad_url).hostname.split('.'); // Retrieve the second to last element of the hostname array, which should always be the domain dive_domain = dive_domain_array[dive_domain_array.length - 2] if (alt_domain) { // If alt_domain has been specified, normalize it. i.e. "HR Dive", "http://hrdive.com", etc. => "hrdive" // This will override the link url's domain. dive_domain = alt_domain.replace(/\s+/g, '').replace(/^(https?:\/\/)?(www\.)?|\.com.*|\?.*|#.*/gi, '').toLowerCase(); } // Return the site logos object for this domain if it exists, or null if it does not if (site_logos[dive_domain]) { return site_logos[dive_domain]; } else { // Fallback logo const url_prefix = "https://d12v9rtnomnebu.cloudfront.net/divesite/responsive_ad_assets/small_logos/"; return { domain: 'industrydive', site_name: 'Industry Dive', alt_text: 'Industry Dive logo', white: { svg: url_prefix + 'industrydive-small-white.svg', png: url_prefix + 'industrydive-small-white.png', }, black: { svg: url_prefix + 'industrydive-small-black.svg', png: url_prefix + 'industrydive-small-black.png', } } } } eventer(messageEvent, function (e) { var key = e.message ? "message" : "data"; var data = e[key]; var eventName = data.message || 'ignore'; if (eventName == 'adContentAvailable') { var $ad_element; $ad_element = $('div').filter('[data-container-ad-unit-id="' + data.adUnit + '-' + window.dfp_visibility + '"]'); $ad_element.hide() var nativeAdEditorialSelector = '.sidebar .hybrid-ad-wrapper .native-ad-editorial'; var nativeAdEditorialExists = $(nativeAdEditorialSelector).length > 0; if ( nativeAdEditorialExists ) { $(nativeAdEditorialSelector).closest('.hybrid-ad-wrapper').addClass('native-ad-editorial-sidebar-border'); } // Inline homepage ad $('.feed-item-ad .hybrid-ad-wrapper a').each(function (index, element) { window.updateGamAdUrl(element, 'inline'); }); // Editorial native ad sidebar // NOTE: must go before inline native ad to ensure correct location added $('.sidebar .native-ad.editorial a').each(function (index, element) { window.updateGamAdUrl(element, 'sidebar'); }); // Editorial native ad inline $('.native-ad.editorial a').each(function (index, element) { window.updateGamAdUrl(element, 'inline'); }); // Sidebar ad $('.sidebar .hybrid-ad-wrapper a').each(function (index, element) { window.updateGamAdUrl(element, 'sidebar'); }); } }, false); var googletag = googletag || {}; googletag.cmd = googletag.cmd || []; googletag.cmd.push(function () { var desktopWidth = 1024; if (window.innerWidth >= desktopWidth) { window.dfp_visibility = 'desktop'; } else { window.dfp_visibility = 'mobile'; } var dfpDisplayConfiguration = { "prestitialEnabled": true, "leaderboardEnabled": true, "sidebarAdsEnabled": true, "visibility": window.dfp_visibility }; if (window.dfp_visibility == 'desktop') { if (typeof prestitialAds == 'object' && prestitialAds.pageIsPrestitialEligible()) { googletag.defineSlot('/21662595662/pharmavoice/pharmavoiceprestitial', [[500, 300], [640, 480]], 'paychekgoeshere').addService(googletag.pubads()); } if (dfpDisplayConfiguration.leaderboardEnabled) { googletag.defineSlot('/21662595662/pharmavoice/pharmavoiceleaderboard', [[970, 180], [728, 90], [970, 90]], 'dfp-leaderboard-desktop').addService(googletag.pubads()); googletag.pubads().addEventListener('slotRenderEnded', function (event) { if ((event.slot.getAdUnitPath() === '/21662595662/pharmavoice/pharmavoiceleaderboard' || event.slot.getAdUnitPath() === '/21662595662/pharmavoice/pharmavoiceleaderboard') && !event.isEmpty ) { $(window).blur(function () { if (document.activeElement === $('#dfp-leaderboard-desktop iframe')[0] || document.activeElement === $('#dfp-leaderboard-desktop iframe')[0]) { try { localStorage.setItem('leaderboard_signup_path', document.location.pathname); } catch (e) { // local storage could not be available due to privacy settings or something } } }); } }); } if (dfpDisplayConfiguration.sidebarAdsEnabled) { googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid1', [[300, 250], 'fluid', [160, 600]], 'dfp-hybrid1-desktop').addService(googletag.pubads()); } googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid2', [[300, 250], 'fluid'], 'dfp-hybrid2-desktop').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid3', [[300, 250], 'fluid'], 'dfp-hybrid3-desktop').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid4', [[300, 250], 'fluid'], 'dfp-hybrid4-desktop').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicethankyouhybrid1', ['fluid'], 'dfp-thankyouhybrid1-desktop').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicethankyouhybrid2', ['fluid'], 'dfp-thankyouhybrid2-desktop').addService(googletag.pubads()); } else { googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid1', [[300, 250], 'fluid'], 'dfp-hybrid1-mobile').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid2', [[300, 250], 'fluid'], 'dfp-hybrid2-mobile').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid3', [[300, 250], 'fluid'], 'dfp-hybrid3-mobile').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicehybrid4', [[300, 250], 'fluid'], 'dfp-hybrid4-mobile').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicethankyouhybrid1', ['fluid'], 'dfp-thankyouhybrid1-mobile').addService(googletag.pubads()); googletag.defineSlot('/21662595662/pharmavoice/pharmavoicethankyouhybrid2', ['fluid'], 'dfp-thankyouhybrid2-mobile').addService(googletag.pubads()); } googletag.pubads().setTargeting("topics", ["Biotech","biotech","Patient","patient","Pharma","pharma","Research & Development","research-development"]); googletag.pubads().setTargeting("tags", ["pharmavoice-tag-blood-disorders","pharmavoice-tag-gene-therapy"]); if ((typeof divecounter !== "undefined") && (divecounter.getData().is_subscriber)) { googletag.pubads().setTargeting("is_subscriber", "yes"); } var site = window.location.host; googletag.pubads().setTargeting("site", site); var first_path = window.location.pathname.split( '/' )[1]; googletag.pubads().setTargeting("first_path", first_path); googletag.pubads().setTargeting("newspost_type", "Article"); googletag.pubads().setTargeting("articleID", "731542"); googletag.pubads().setTargeting("slug", "gene therapy sickle cell crispr cellarity vertex pfizer"); googletag.pubads().setTargeting("is_sponsored", "false" ); // Don't show skyscraper ads on full-width articles googletag.pubads().setTargeting("show_skyscraper", "true"); // Set ad targeting on the trendline to show the ads designated for it var isTopicPage = window.location.pathname.indexOf('/topic/') !== -1; if(isTopicPage) { googletag.pubads().setTargeting('is_topics_page', 'true'); } googletag.pubads().setCentering(true); googletag.pubads().collapseEmptyDivs(false); // Whether to collapse the slots even before the ads are fetched. googletag.pubads().enableSingleRequest(); googletag.enableServices(); }); </script> <script>window.fallbackAds = '[{\u0022adType\u0022: \u0022editorial_native_ad\u0022, \u0022link\u0022: \u0022/trendline/rare\u002Ddiseases/351/?utm_source\u003DPV\u0026utm_medium\u003DNativeFB\u0026utm_campaign\u003DTrendlineFB\u0022, \u0022label\u0022: \u0022Trendline\u0022, \u0022image\u0022: \u0022https://imgproxy.divecdn.com/I_u8au9WqN8hyzOfMtCQJU6EPeM\u002D5EXb\u002D2_YUn_XcAs/g:ce/rs:fit:212:100/Z3M6Ly9kaXZlc2l0ZS1zdG9yYWdlL2RpdmVpbWFnZS9HZXR0eUltYWdlcy0xNDY3MjM4OTgxXzEuanBn.webp\u0022, \u0022attribution\u0022: \u0022\u0022, \u0022headline\u0022: \u0022Inside the growing treatment market for rare diseases\u0022, \u0022ctaText\u0022: \u0022Access now\u0022, \u0022description\u0022: \u0022As drug developers find promise in the rare diseases, deals and treatments are on the rise\u0022}, 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100vw" alt=""> <article class=""> <div class="first-page-pdf">  <div class="printed-branding"> <span class="promoted-branded-copy">An article from</span> <img src="https://d12v9rtnomnebu.cloudfront.net/logo/printer_friendly/pharmavoice.jpg" alt="site logo"> </div> <div class="article-title-wrapper"> <div class="post-label-wrapper"> </div> <h1 class="display-heading-04"> A sickle cell cure exists. But patients need more than just gene therapy. </h1> <p>Almost a year after two historic approvals, Pfizer is pulling a sickle cell disease treatment from the market, and the outlook for patients feels shakier.</p> </div> <div class="byline"> <div class="article-byline"> <div class="date date-bottom-border"> <span class="published-info"> Published Nov. 1, 2024 </span> </div> <div class="byline-row "> <div class="author "> <span>By</span> Alexandra Pecci </div> <div class="social-share"> <ul class="social-icon-list"> <li> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https%3A//www.pharmavoice.com/news/gene-therapy-sickle-cell-crispr-cellarity-vertex-pfizer/731542/&title=A%20sickle%20cell%20cure%20exists.%20But%20patients%20need%20more%20than%20just%20gene%20therapy." aria-label="Share article to Linkedin" class="social-icon analytics t-article-byline-social-linkedin"> <img src="/static/img/social_icons/article_page/linkedin.svg?092716141024" alt="Post to LinkedIn"> <span 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Please let us know if you have <a href="/contact/">feedback</a>. </div> </div> </div> <p>Excitement around the sickle cell treatment market has been palpable. Around the beginning of the year, two new breakthrough gene editing medications — including the <a href="https://www.pharmavoice.com/news/fda-crispr-vertex-casgevy-gene-editing/701933/">first FDA-approved</a> CRISPR-based drug — won approval for sickle cell indications, providing patients with potentially curative care. </p> <p>But multiple barriers to access have kept those therapies from reaching a wide swath of patients and the treatment landscape is getting slimmer. </p> <p>Last month, Pfizer announced plans to <a href="https://www.pfizer.com/news/press-release/press-release-detail/pfizer-voluntarily-withdraws-all-lots-sickle-cell-disease#:~:text=NEW%20YORK%2D%2D(BUSINESS%20WIRE,disease%20with%20limited%20treatment%20options.">yank</a> its sickle cell drug Oxbryta from the market over safety concerns, leaving patients with one less treatment and <a href="https://www.sicklecelldisease.org/2024/09/28/marac-statement-oxbryta/">unanswered questions</a> about how to safely stop the medication.</p> <p>Moreover, patient uptake for the two new gene therapies — Vertex Pharmaceuticals and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia — has been slow. </p> <div class="hybrid-ad-wrapper show-small hide-large"> <div class="hybrid-ad-inner-wrapper"> <div class="show-small hide-large"> <div data-container-ad-unit-id="/21662595662/pharmavoice/pharmavoicehybrid1-mobile"></div> <div id="dfp-hybrid1-mobile"> <script data-cfasync="false" src="/cdn-cgi/scripts/5c5dd728/cloudflare-static/email-decode.min.js"></script><script> waitToLoadAds.push(function() { googletag.cmd.push(function() { if (window.dfp_visibility == 'mobile' ) { googletag.display('dfp-hybrid1-mobile'); googletag.pubads().addEventListener('slotRenderEnded', function (event) { var adUnitPath = '/21662595662/pharmavoice/pharmavoicehybrid1'; var onProformative = false; if (onProformative && event.slot.getAdUnitPath() === adUnitPath && !event.isEmpty ) { var adUnitPathWithVisibility = adUnitPath + '-mobile'; var selector = '.pf-comments__ad-wrapper [data-container-ad-unit-id="' + adUnitPathWithVisibility + '"]'; if (!$(selector).closest('.pf-comments__ad-wrapper').hasClass('borders')) { $(selector).closest('.pf-comments__ad-wrapper').addClass('borders') } } }); } }); }); </script> </div> </div> </div> </div> <div class="hybrid-ad-wrapper hide-small show-large"> <div class="hybrid-ad-inner-wrapper"> <div class="hide-small show-large"> <div data-container-ad-unit-id="/21662595662/pharmavoice/pharmavoicehybrid2-desktop"></div> <div id="dfp-hybrid2-desktop"> <script> waitToLoadAds.push(function() { googletag.cmd.push(function() { if (window.dfp_visibility == 'desktop' ) { googletag.display('dfp-hybrid2-desktop'); googletag.pubads().addEventListener('slotRenderEnded', function (event) { var adUnitPath = '/21662595662/pharmavoice/pharmavoicehybrid2'; var onProformative = false; if (onProformative && event.slot.getAdUnitPath() === adUnitPath && !event.isEmpty ) { var adUnitPathWithVisibility = adUnitPath + '-desktop'; var selector = '.pf-comments__ad-wrapper [data-container-ad-unit-id="' + adUnitPathWithVisibility + '"]'; if (!$(selector).closest('.pf-comments__ad-wrapper').hasClass('borders')) { $(selector).closest('.pf-comments__ad-wrapper').addClass('borders') } } }); } }); }); </script> </div> </div> </div> </div> <p>In addition to their multimillion-dollar <a href="https://www.pharmavoice.com/news/worlds-priciest-drug-gene-therapy-sarepta-crispr-vertex/725723/">price tags</a>, patients cleared for Casgevy face a daunting, months-long ex-vivo <a href="https://www.massgeneralbrigham.org/en/about/newsroom/articles/gene-therapy-for-sickle-cell-disease#:~:text=Before%20returning%20the%20extracted%20cells,a%20portion%20of%20the%20process.">process</a> that includes extracting stem cells from bone marrow, sending them to the manufacturers for modification and chemotherapy to make room for the edited cells.</p> <p>“They’ve been considered a functional cure in the field. However, they're challenged by cost, complexity and toxicity of delivery,” said Dr. Cameron Trenor, head of translational medicine for Cellarity, which is developing a sickle cell drug of its own. “It seems hard to get around that safety barrier and the scalability challenges given the cost and complexity of manufacturing.”</p> <div class="pullquote"> <hr/> <div class="pq-hr-wrapper"> <p class="pq-quote">“The disease is not going away, and we need something that all or almost all patients can take.”</p> <div class="pq-headshot"><img alt="" class="pq-headshot-img-hidden" src="https://d12v9rtnomnebu.cloudfront.net/diveimages/corporate_site/teampage/square_profiles/placeholder-200.png"/></div> <div class="pq-speaker-details"> <p class="pq-speaker">Dr. Cameron Trenor</p> <p class="pq-speaker-title">Head, translational medicine, Cellarity</p> </div> </div> <hr/></div> <p>As of the end of September, just 10 patients <a href="https://investor.bluebirdbio.com/static-files/74c08f3b-85c2-4d3b-953d-8df9e50e6e5d">had started on Lyfgenia</a> this year, and bluebird <a href="https://www.pharmavoice.com/news/bluebird-bio-restructuring-layoffs-gene-therapy/727923/">recently announced</a> plans to lay off about a quarter of its workforce to cut costs.</p> <p>On top of that, a <a href="https://www.nejm.org/doi/full/10.1056/NEJMoa2405541">study published this month</a> revealed seven children developed blood cancer after receiving Skysona, bluebird’s gene therapy for a rare brain disease, recalling when bluebird temporarily <a href="https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-announces-temporary-suspension-phase-12-and-phase-3">suspended trials</a> for Lyfgenia (then called LentiGlobin) after <a href="https://www.yalemedicine.org/news/gene-therapies-sickle-cell-disease">two patients</a> developed cancer in 2021.</p> <p>The sickle cell pipeline has taken other hits too. Graphite Bio abandoned its lead asset, a sickle cell gene editing therapy, after a <a href="https://jamanetwork.com/journals/jama/article-abstract/2800764">severe adverse event</a> occurred in the first dosed patient. Now a newly formed company, Kamau Therapeutics, is trying to <a href="https://kamautx.com/wp-content/uploads/2023/12/ASH_poster_v3_1.pdf">revive that asset</a>, while Graphite Bio <a href="https://ir.lenz-tx.com/news-events/press-releases/detail/9/lenz-therapeutics-announces-completion-of-merger-with-graphite-bio-and-provides-update-on-recent-clinical-and-corporate-progress">is no more</a>.</p> <p>And despite the approval of one-and-done gene therapies, patients still face unmet needs, analysts say.</p> <div class="hybrid-ad-wrapper show-small hide-large"> <div class="hybrid-ad-inner-wrapper"> <div class="show-small hide-large"> <div data-container-ad-unit-id="/21662595662/pharmavoice/pharmavoicehybrid2-mobile"></div> <div id="dfp-hybrid2-mobile"> <script> waitToLoadAds.push(function() { googletag.cmd.push(function() { if (window.dfp_visibility == 'mobile' ) { googletag.display('dfp-hybrid2-mobile'); googletag.pubads().addEventListener('slotRenderEnded', function (event) { var adUnitPath = '/21662595662/pharmavoice/pharmavoicehybrid2'; var onProformative = false; if (onProformative && event.slot.getAdUnitPath() === adUnitPath && !event.isEmpty ) { var adUnitPathWithVisibility = adUnitPath + '-mobile'; var selector = '.pf-comments__ad-wrapper [data-container-ad-unit-id="' + adUnitPathWithVisibility + '"]'; if (!$(selector).closest('.pf-comments__ad-wrapper').hasClass('borders')) { $(selector).closest('.pf-comments__ad-wrapper').addClass('borders') } } }); } }); }); </script> </div> </div> </div> </div> <div class="hide-small show-large" id="desktop-inline-signup"></div> <p>“[Sickle cell] remains an attractive growth market with potential sales of $5 billion in 2030, despite Oxbryta’s withdrawal” because of the “clear unmet medical need,” according to a consensus forecast provided to PharmaVoice from Evaluate, a Norstella company.</p> <p>“This is a profoundly devastating disease. It's essentially a disease of hypoxia: not being able to get oxygen to your tissues well,” Trenor said. “That manifests in silent and painful ways.”</p> <p>However pain is just one of the effects.</p> <p>“Anywhere your blood goes is where sickle cell disease affects you,” Trenor said. “It causes stroke, it causes heart disease, it causes lung disease, it kills your spleen, it causes liver disease, it causes kidney disease.”</p> <p>Evaluate points to “several different gene editing biotechs seeking to launch [sickle cell] therapies over the coming years, in addition to some of the largest pharma companies.”</p> <p>According to a <a href="https://www.delveinsight.com/report-store/sickle-cell-disease-competitive-landscape?utm_source=cision&utm_medium=pressrelease&utm_campaign=spr">report from last year</a>, over 40 companies are developing more than 50 sickle cell treatments, including <a href="https://www.novonordisk.com/science-and-technology/r-d-pipeline.html">Novo Nordisk</a>, which has therapies in phase 2 and 3. Agios Pharmaceuticals <a href="https://investor.agios.com/news-releases/news-release-details/agios-completes-enrollment-phase-3-rise-study-mitapivat-sickle">just completed enrollment</a> for a phase 3 study of its oral, small molecule PK activator, while <a href="https://www.gsk.com/media/11102/q1-2024-pipeline-assets-and-clinical-trials-report.pdf">GSK</a> is in early development with its DNMT1 inhibitor.</p> <p>Additionally, sickle cell disease is “a proof-of-concept indication for new drug technologies,” the Evaluate forecast said, pointing to the first CRISPR drugs as an example.</p> <p>“The 2023 approvals for CRISPR/Vertex and bluebird bode well for the chances of some of these next-generation cell therapies, and this competition will help to improve patient access,” the forecast said.</p><div class="show-small hide-large" id="mobile-inline-signup"></div><section class="storylines-carousel-wrapper hide-small show-large" id="desktop-carousel"></section> <p>The Oxbryta withdrawal leaves the market with just a few non-gene therapy drugs including hydroxyurea, which can reduce sickling of red blood cells, and others aimed at stemming pain. Hydroxyurea works by increasing fetal hemoglobin, which reduces disease complications, but it also carries safety risks.</p> <p>“It causes immunosuppression, so you can't push the dose higher, and that limits how much fetal hemoglobin, and therefore how much benefit you get,” Trenor said.</p> <p>Cellarity’s <a href="https://cellarity.com/pipeline/">lead asset</a> is an oral drug developed to induce fetal hemoglobin “to a level that is really meaningful for patients,” Trenor said.</p> <p>“It is known from patients with mutations that have genetically high fetal hemoglobin, that if you can get over about 20% fetal hemoglobin you don't have many complications of sickle cell disease any longer,” he said. “We're hoping we'll be able to get patients up to or above that level with a small molecule, and that would be a paradigm shift in the field.”</p> <p>Cellarity is in the IND-enabling phase and plans to start clinical trials next year. The company hopes the oral treatment will help bridge the access divide.</p> <p>“The disease is not going away, and we need something that all or almost all patients can take,” Trenor said.</p> </div> <div class="reading-list recommended-reading"> <h3 class="sidebar-box__header reading-list__header">Recommended Reading</h3> <ul class="list-no-bullets"> <li class="reading-list__item"> <a href="https://www.pharmavoice.com/news/worlds-priciest-drug-gene-therapy-sarepta-crispr-vertex/725723/" target="_blank" class="reading-list__link analytics t-article-end-recommended"> Is anyone taking the world’s priciest drugs? </a> <span class="label label--soft"> <img src="/static/img/offsite_black.png?102717100918" class="off-site-link" alt="offsite link" width="12" height="12" /> <span>Meagan Parrish</span> </span> </li> <li class="reading-list__item"> <a href="https://www.pharmavoice.com/news/sickle-cell-crispr-gene-editing-vertex-exa-cel-barriers/699141/" target="_blank" class="reading-list__link analytics t-article-end-recommended"> What if a CRISPR cure isn’t such an easy choice? </a> <span class="label label--soft"> <img src="/static/img/offsite_black.png?102717100918" class="off-site-link" alt="offsite link" width="12" height="12" /> <span>Ned Pagliarulo</span> </span> </li> </ul> </div> <div class="post-article-wrapper"> <div class="share-buttons"> <ul class="social-button-list"> <li> <a href="http://www.linkedin.com/shareArticle?mini=true&url=https%3A//www.pharmavoice.com/news/gene-therapy-sickle-cell-crispr-cellarity-vertex-pfizer/731542/&title=A%20sickle%20cell%20cure%20exists.%20But%20patients%20need%20more%20than%20just%20gene%20therapy." class="social-button analytics t-article-end-social-linkedin" title="Post"> <img src="/static/img/social_icons/article_page/linkedin.svg?092716141024" alt="Post to LinkedIn"> </a> </li> <li> <a 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A sickle cell cure exists. But patients need more than just gene therapy. | PharmaVoice