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Click here for more information about advertising.</a><center><hr style="margin-top: 10px;"/> <div style="font-size: 10pt;">You can also make a donation by PayPal and support the Gene Therapy Net website.</div> <form action="https://www.paypal.com/cgi-bin/webscr" method="post" target="_top"> <input type="hidden" name="cmd" value="_s-xclick"> <input type="hidden" name="hosted_button_id" value="YYU4T4HSRK4P8"> <input type="image" src="https://www.paypalobjects.com/en_US/i/btn/btn_donateCC_LG_global.gif" border="0" name="submit" alt="PayPal – The safer, easier way to pay online!"> <img alt="" border="0" src="https://www.paypalobjects.com/en_US/i/scr/pixel.gif" width="1" height="1"> </form> </center></div> </div> </div> <div> <div class="uk-panel _menu menulist tm-child-list" id="module-67"> <h3> Most Popular Items </h3> <ul class="mostread"> <li><a href="/breaking-gene-therapy-news.html">Breaking Gene Therapy News</a></li> <li><a href="/diseases-treated-by-gene-therapy.html">Diseases Treated by Gene Therapy </a></li> <li><a href="/types-of-gene-therapy.html">Types of Gene Therapy</a></li> <li><a href="/viral-vector/adeno-associated-viruses.html">Adeno-Associated Viral Vectors</a></li> <li><a href="/glossary-of-gene-therapy-terms.html">Glossary of Gene Therapy Terms</a></li> </ul> </div> </div> <div> <div class="uk-panel" id="module-66"> <h3> Who's Online </h3> <div class="mod-whosonline"> <p>We have 255 guests and 2 members online</p> </div> </div> </div> <div> <div class="uk-panel" id="module-29"> <h3> Statistics </h3> <ul class="mod-stats list-group"> <li class="list-group-item"> Users <span class="badge bg-secondary float-end rounded-pill">5531</span> </li> <li class="list-group-item"> Articles <span class="badge bg-secondary float-end rounded-pill">742</span> </li> <li class="list-group-item"> Articles View Hits <span class="badge bg-secondary float-end rounded-pill">17328030</span> </li> </ul> </div> </div></div></div> </div> <div class="uk-width-1-2@m uk-width-3-5@l uk-flex-first"> <div class="afgeronde-artikelen uk-margin"> <div class="uk-grid uk-child-width-1-1 uk-grid-row-small uk-grid-match" uk-grid> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-link-reset uk-margin-top uk-margin-remove-bottom"> <a href="/user-login.html">Welcome to Gene Therapy Net</a> </h3> <div class="el-content uk-panel uk-margin-top"><strong>Gene Therapy Net</strong> is the web resource for <a href="/patient-information.html">patients</a> and professionals interested in <a href="/what-is-gene-therapy.html">gene therapy</a>. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international <a href="/regulatory-affairs.html">regulations and guidelines</a> associated with <a href="/clinicaltrialsgov.html">clinical gene therapy trials</a>. For resources pertaining to regenerative cell therapy and clinical treatment, check out <a href="https://r3stemcell.com" target="_blank">R3 Stem Cell</a>.<br /><center><br /> <div class="moduletable "> <div class="mod-banners bannergroup"> <div class="mod-banners__item banneritem"> <img src="https://www.genetherapynet.com/images/banners/9thCommercContiProcessingSummit-feb2025.png" alt="9thCommercContiProcessingSummit-feb2025"> </div> </div> </div> </center></div> <div class="uk-margin-top"><a href="/user-login.html" class="el-link uk-button uk-button-default">Log in or register to read more</a></div> </div></div> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-link-reset uk-margin-top uk-margin-remove-bottom"> <a href="/user-login.html">Hemophilia B Gene Therapy Achieves Sustained Benefit Over 4 Years</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 8 February 2025, source: HCP live</em><br />More than 90% of patients with hemophilia B eliminated factor IX prophylaxis 4 years after treatment with etranacogene dezaparvovec (HEMGENIX) gene therapy. Hemophilia B Gene Therapy Achieves Sustained Benefit Over 4 Years. A one-time infusion of etranacogene dezaparvovec-drlb (HEMGENIX) gene therapy continued to demonstrate long-term sustained efficacy and safety in adults with hemophilia B over 4 years in the pivotal Phase 3 HOPE-B study. Announced by CSL on February 7, 2025, these data, presented at the 18th Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD), showed the gene therapy delivered sustained factor IX activity levels, offered greater bleed protection than prophylactic treatment, and maintained favorable safety over time. <center><div class="fastsocialshare_container fastsocialshare-align-left"><div class="fastsocialshare-subcontainer"><script> var loadAsyncDeferredFacebook = function() { (function(d, s, id) { var js, fjs = d.getElementsByTagName(s)[0]; if (d.getElementById(id)) return; js = d.createElement(s); js.id = id; js.src = "//connect.facebook.net/en_GB/sdk.js#xfbml=1&version=v3.0&appId=201432789890280"; fjs.parentNode.insertBefore(js, fjs); }(document, 'script', 'facebook-jssdk')); } if (window.addEventListener) window.addEventListener("load", loadAsyncDeferredFacebook, false); else if (window.attachEvent) window.attachEvent("onload", loadAsyncDeferredFacebook); else window.onload = loadAsyncDeferredFacebook; 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} @media(min-width: 500px) { .aangepast { width: 468px; } ) @media(min-width: 800px) { .aangepast { width: 468px; } } </style> <script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js?client=ca-pub-1253043591735915" crossorigin="anonymous"></script> <!-- Gene Therapy Net Link 468x15 [voormalig linkadvertentieblok] --> <ins class="adsbygoogle" style="display:block" data-ad-client="ca-pub-1253043591735915" data-ad-slot="4912972607" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script> </center> </div> <div class="uk-margin-top"><a href="/user-login.html" class="el-link uk-button uk-button-default">Log in or register to read more</a></div> </div></div> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-link-reset uk-margin-top uk-margin-remove-bottom"> <a href="/user-login.html">NHS Approves Landmark Gene Therapy For Sickle Cell Disease</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 1 February 2025, source: evrimagaci.org</em><br />On February 1, 2025, the National Institute for Health and Care Excellence (NICE) announced the approval of exagamglogene autotemcel (exa-cel), marking what many are calling a significant breakthrough for the treatment of sickle cell disease. This groundbreaking gene therapy utilizes the CRISPR gene-editing technology, which has transformed medical approaches to genetic disorders, enabling some patients suffering from this debilitating condition to access potentially life-changing treatment. <center><div class="fastsocialshare_container fastsocialshare-align-left"><div class="fastsocialshare-subcontainer"><script> var loadAsyncDeferredFacebook = function() { (function(d, s, id) { var js, fjs = d.getElementsByTagName(s)[0]; if (d.getElementById(id)) return; js = d.createElement(s); js.id = id; js.src = "//connect.facebook.net/en_GB/sdk.js#xfbml=1&version=v3.0&appId=201432789890280"; fjs.parentNode.insertBefore(js, fjs); }(document, 'script', 'facebook-jssdk')); } if (window.addEventListener) window.addEventListener("load", loadAsyncDeferredFacebook, false); else if (window.attachEvent) window.attachEvent("onload", loadAsyncDeferredFacebook); else window.onload = loadAsyncDeferredFacebook; </script><div class="fastsocialshare-share-fbl fastsocialshare-standard"> <div class="fb-like" data-href="https://www.genetherapynet.com/gene-therapy-news/817-nhs-approves-landmark-gene-therapy-for-sickle-cell-disease.html" data-layout="standard" data-width="100" data-action="like" data-show-faces="true" data-share="false"> </div></div><div class="fastsocialshare-share-fbsh fb-shareme-core"> <div class="fb-share-button fb-shareme-core" data-href="https://www.genetherapynet.com/gene-therapy-news/817-nhs-approves-landmark-gene-therapy-for-sickle-cell-disease.html" data-layout="button_count" data-size="small"> </div></div><div class="fastsocialshare-share-tw"> <a href="https://twitter.com/intent/tweet" data-dnt="true" class="twitter-share-button" data-text="NHS Approves Landmark Gene Therapy For Sickle Cell Disease" data-count="horizontal" data-via="genetherapynet" data-url="https://www.genetherapynet.com/gene-therapy-news/817-nhs-approves-landmark-gene-therapy-for-sickle-cell-disease.html" data-lang="en"></a> </div> <script> var loadAsyncDeferredTwitter = function() { var d = document; 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The phase 3 STEER study has been evaluating intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with SMA type 2 who were able to sit but had never walked independently. 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</script> </div><div class="fastsocialshare-share-whatsapp"> <a style="text-decoration:none; border-radius: 2px; padding:1px 5px 2px; font-size:14px; background-color:#25d366; color:#ffffff !important;" onclick="window.open('https://api.whatsapp.com/send?text=https%3A%2F%2Fwww.genetherapynet.com%2Fgene-therapy-news%2F816-novartis%E2%80%99-investigational-spinal-muscular-atrophy-gene-therapy-shows-promise-in-late-stage-study.html - Novartis%E2%80%99%20investigational%20spinal%20muscular%20atrophy%20gene%20therapy%20shows%20promise%20in%20late-stage%20study','whatsappshare','width=640,height=480')" href="javascript:void(0)"><span class='fastsocialshare-share-whatsappicon' style='margin-right:4px'><svg style="vertical-align:text-bottom" fill="#fff" preserveAspectRatio="xMidYMid meet" height="1em" width="1em" viewBox="0 2 40 40"><g><path d="m25 21.7q0.3 0 2.2 1t2 1.2q0 0.1 0 0.3 0 0.8-0.4 1.7-0.3 0.9-1.6 1.5t-2.2 0.6q-1.3 0-4.3-1.4-2.2-1-3.8-2.6t-3.3-4.2q-1.6-2.3-1.6-4.3v-0.2q0.1-2 1.7-3.5 0.5-0.5 1.2-0.5 0.1 0 0.4 0t0.4 0.1q0.4 0 0.6 0.1t0.3 0.6q0.2 0.5 0.8 2t0.5 1.7q0 0.5-0.8 1.3t-0.7 1q0 0.2 0.1 0.3 0.7 1.7 2.3 3.1 1.2 1.2 3.3 2.2 0.3 0.2 0.5 0.2 0.4 0 1.2-1.1t1.2-1.1z m-4.5 11.9q2.8 0 5.4-1.1t4.5-3 3-4.5 1.1-5.4-1.1-5.5-3-4.5-4.5-2.9-5.4-1.2-5.5 1.2-4.5 2.9-2.9 4.5-1.2 5.5q0 4.5 2.7 8.2l-1.7 5.2 5.4-1.8q3.5 2.4 7.7 2.4z m0-30.9q3.4 0 6.5 1.4t5.4 3.6 3.5 5.3 1.4 6.6-1.4 6.5-3.5 5.3-5.4 3.6-6.5 1.4q-4.4 0-8.2-2.1l-9.3 3 3-9.1q-2.4-3.9-2.4-8.6 0-3.5 1.4-6.6t3.6-5.3 5.3-3.6 6.6-1.4z"></path></g></svg></span><span class='fastsocialshare-share-whatsapptext'>Whatsapp</span></a> </div></div></div></center></div> <div class="uk-margin-top"><a href="/user-login.html" class="el-link uk-button uk-button-default">Log in or register to read more</a></div> </div></div> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-link-reset uk-margin-top uk-margin-remove-bottom"> <a href="/user-login.html">India’s first gene therapy offers new hope for haemophilia patients</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 17 December 2024, source: Indian Express</em><br />In a significant medical breakthrough, India’s first human gene therapy with a lentiviral vector for severe haemophilia-A has been found to be successful with zero bleeding rate in patients. Haemophilia is a serious bleeding disorder caused by blood deficiency of clotting factors (factor VIII and factor IX proteins). It can lead to spontaneous bleeding episodes, joint damage, and a reduced quality of life for patients and their families. The new gene therapy has shown promising results during the first phase trial on five patients, aged between 22 and 41. The annualised bleeding rate has been observed to be zero in all five patients over a cumulative follow-up of 81 months. Supported by the Department of Biotechnology, this first-in-human gene therapy for haemophilia-A patients, has been developed by Centre for Stem Cell Research (CSCR) at CMC, Vellore. <center><div class="fastsocialshare_container fastsocialshare-align-left"><div class="fastsocialshare-subcontainer"><script> var loadAsyncDeferredFacebook = function() { (function(d, s, id) { var js, fjs = d.getElementsByTagName(s)[0]; if (d.getElementById(id)) return; js = d.createElement(s); js.id = id; js.src = "//connect.facebook.net/en_GB/sdk.js#xfbml=1&version=v3.0&appId=201432789890280"; fjs.parentNode.insertBefore(js, fjs); }(document, 'script', 'facebook-jssdk')); } if (window.addEventListener) window.addEventListener("load", loadAsyncDeferredFacebook, false); else if (window.attachEvent) window.attachEvent("onload", loadAsyncDeferredFacebook); else window.onload = loadAsyncDeferredFacebook; </script><div class="fastsocialshare-share-fbl fastsocialshare-standard"> <div class="fb-like" data-href="https://www.genetherapynet.com/gene-therapy-news/813-india’s-first-gene-therapy-offers-new-hope-for-haemophilia-patients.html" data-layout="standard" data-width="100" data-action="like" data-show-faces="true" data-share="false"> </div></div><div class="fastsocialshare-share-fbsh fb-shareme-core"> <div class="fb-share-button fb-shareme-core" data-href="https://www.genetherapynet.com/gene-therapy-news/813-india’s-first-gene-therapy-offers-new-hope-for-haemophilia-patients.html" data-layout="button_count" data-size="small"> </div></div><div class="fastsocialshare-share-tw"> <a href="https://twitter.com/intent/tweet" data-dnt="true" class="twitter-share-button" data-text="India’s first gene therapy offers new hope for haemophilia patients" data-count="horizontal" data-via="genetherapynet" data-url="https://www.genetherapynet.com/gene-therapy-news/813-india’s-first-gene-therapy-offers-new-hope-for-haemophilia-patients.html" data-lang="en"></a> </div> <script> var loadAsyncDeferredTwitter = function() { var d = document; 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But Neurogene is able to avoid a lengthy clinical trial pause largely because of its participation in an FDA pilot program intended to speed up the development of therapies for rare diseases. The clinical trial participant who was hospitalized for severe complications after receiving the high dose of Neurogene’s experimental Rett syndrome gene therapy has died. Neurogene disclosed the death in a regulatory filing after the market close Thursday. The company said the FDA is permitting the Phase 1/2 study to continue with the low dose of the therapy, code-named NGN-401. 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In this approach, a nanoparticle exposes cells to the instructions for building CRISPR reagents and the correct gene sequence to repair a gene defect. The method also aims to repair genetic problems at very early stages of development. While more research will be needed, the investigators are hopeful that this technique will eventually be used to treat human patients who are identified during prenatal testing. <center><div class="fastsocialshare_container fastsocialshare-align-left"><div class="fastsocialshare-subcontainer"><script> var loadAsyncDeferredFacebook = function() { (function(d, s, id) { var js, fjs = d.getElementsByTagName(s)[0]; if (d.getElementById(id)) return; js = d.createElement(s); js.id = id; js.src = "//connect.facebook.net/en_GB/sdk.js#xfbml=1&version=v3.0&appId=201432789890280"; fjs.parentNode.insertBefore(js, fjs); }(document, 'script', 'facebook-jssdk')); } if (window.addEventListener) window.addEventListener("load", loadAsyncDeferredFacebook, false); else if (window.attachEvent) window.attachEvent("onload", loadAsyncDeferredFacebook); else window.onload = loadAsyncDeferredFacebook; </script><div class="fastsocialshare-share-fbl fastsocialshare-standard"> <div class="fb-like" data-href="https://www.genetherapynet.com/gene-therapy-news/799-a-new-gene-therapy-technique-aims-to-deliver-treatment-before-birth.html" data-layout="standard" data-width="100" data-action="like" data-show-faces="true" data-share="false"> </div></div><div class="fastsocialshare-share-fbsh fb-shareme-core"> <div class="fb-share-button fb-shareme-core" data-href="https://www.genetherapynet.com/gene-therapy-news/799-a-new-gene-therapy-technique-aims-to-deliver-treatment-before-birth.html" data-layout="button_count" data-size="small"> </div></div><div class="fastsocialshare-share-tw"> <a href="https://twitter.com/intent/tweet" data-dnt="true" class="twitter-share-button" data-text="A New Gene Therapy Technique Aims to Deliver Treatment Before Birth" data-count="horizontal" data-via="genetherapynet" data-url="https://www.genetherapynet.com/gene-therapy-news/799-a-new-gene-therapy-technique-aims-to-deliver-treatment-before-birth.html" data-lang="en"></a> </div> <script> var loadAsyncDeferredTwitter = function() { var d = document; 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border-radius: 2px; padding:1px 5px 2px; font-size:14px; background-color:#25d366; color:#ffffff !important;" onclick="window.open('https://api.whatsapp.com/send?text=https%3A%2F%2Fwww.genetherapynet.com%2Fgene-therapy-news%2F799-a-new-gene-therapy-technique-aims-to-deliver-treatment-before-birth.html - A%20New%20Gene%20Therapy%20Technique%20Aims%20to%20Deliver%20Treatment%20Before%20Birth','whatsappshare','width=640,height=480')" href="javascript:void(0)"><span class='fastsocialshare-share-whatsappicon' style='margin-right:4px'><svg style="vertical-align:text-bottom" fill="#fff" preserveAspectRatio="xMidYMid meet" height="1em" width="1em" viewBox="0 2 40 40"><g><path d="m25 21.7q0.3 0 2.2 1t2 1.2q0 0.1 0 0.3 0 0.8-0.4 1.7-0.3 0.9-1.6 1.5t-2.2 0.6q-1.3 0-4.3-1.4-2.2-1-3.8-2.6t-3.3-4.2q-1.6-2.3-1.6-4.3v-0.2q0.1-2 1.7-3.5 0.5-0.5 1.2-0.5 0.1 0 0.4 0t0.4 0.1q0.4 0 0.6 0.1t0.3 0.6q0.2 0.5 0.8 2t0.5 1.7q0 0.5-0.8 1.3t-0.7 1q0 0.2 0.1 0.3 0.7 1.7 2.3 3.1 1.2 1.2 3.3 2.2 0.3 0.2 0.5 0.2 0.4 0 1.2-1.1t1.2-1.1z m-4.5 11.9q2.8 0 5.4-1.1t4.5-3 3-4.5 1.1-5.4-1.1-5.5-3-4.5-4.5-2.9-5.4-1.2-5.5 1.2-4.5 2.9-2.9 4.5-1.2 5.5q0 4.5 2.7 8.2l-1.7 5.2 5.4-1.8q3.5 2.4 7.7 2.4z m0-30.9q3.4 0 6.5 1.4t5.4 3.6 3.5 5.3 1.4 6.6-1.4 6.5-3.5 5.3-5.4 3.6-6.5 1.4q-4.4 0-8.2-2.1l-9.3 3 3-9.1q-2.4-3.9-2.4-8.6 0-3.5 1.4-6.6t3.6-5.3 5.3-3.6 6.6-1.4z"></path></g></svg></span><span class='fastsocialshare-share-whatsapptext'>Whatsapp</span></a> </div></div></div></center></div> <div class="uk-margin-top"><a href="/user-login.html" class="el-link uk-button uk-button-default">Log in or register to read more</a></div> </div></div> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-link-reset uk-margin-top uk-margin-remove-bottom"> <a href="/user-login.html">Bluebird bio’s Skysona led to seven cases of blood cancer in gene therapy trials</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 12 October 2024, source: pharmaceutical-technology.com</em><br />The side effects of bluebird bio’s gene therapy Skysona (elivaldogene autotemcel) have been thrust back into the spotlight after new data shows seven children who took part in its clinical studies went on to develop a type of blood cancer. The findings, from a study published in The New England Journal of Medicine (NEJM) yesterday (9 October), show that seven out of 67 patients under 18 years of age who took part in a Phase II and Phase III trial for Skysona developed haematologic cancers. 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