Gene Therapy Special News Items

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style="display:block" data-ad-client="ca-pub-1253043591735915" data-ad-slot="7012885353" data-ad-format="auto" data-full-width-responsive="true"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script> </center></div> </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-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-margin-top uk-margin-remove-bottom"> <a href="/user-login.html" class="uk-link-reset">Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 25 November 2024, source: medcitynews.com</em><br />A patient death in a clinical trial usually prompts the FDA to impose a clinical hold. 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. Neurogene added that it will incorporate this dose in planning the design of the clinical trial expected to support a regulatory submission seeking FDA approval of the gene therapy. <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/800-patient-dies-in-gene-therapy-trial,-but-fda-permits-neurogene-to-proceed-with-low-dose.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/800-patient-dies-in-gene-therapy-trial,-but-fda-permits-neurogene-to-proceed-with-low-dose.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="Patient Dies in Gene Therapy Trial, But FDA Permits Neurogene to Proceed With Low Dose" data-count="horizontal" data-via="genetherapynet" data-url="https://www.genetherapynet.com/gene-therapy-news/800-patient-dies-in-gene-therapy-trial,-but-fda-permits-neurogene-to-proceed-with-low-dose.html" data-lang="en"></a> </div> <script> var loadAsyncDeferredTwitter = function() { var d = document; 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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"></script> <ins class="adsbygoogle aangepast" style="display:block; text-align:center;" data-ad-layout="in-article" data-ad-format="fluid" data-ad-client="ca-pub-1253043591735915" data-ad-slot="5059787231"></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-margin-top uk-margin-remove-bottom"> <a href="/user-login.html" class="uk-link-reset">A New Gene Therapy Technique Aims to Deliver Treatment Before Birth</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 30 October 2024, source: Labroots</em><br />Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where they perform their gene editing function. 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. 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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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The clinical trial, an open-label study (NCT06270316) focused on safety and early efficacy, is still recruiting about 12 adult male patients at a site in Fairfax, Virginia. Eligible men will have had a suboptimal response to enzyme replacement therapy (ERT). 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The protein Tau is essential to the function of cells in the brain and central nervous system, but when over-produced under certain conditions, it forms tangles that clog the cells’ internal structures. These tangles have also been found in Alzheimer’s disease patients. 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It’s an all-or-nothing proposition. But what if it wasn’t? What if gene therapy could be dialed up or down, on a daily basis, with a simple pill? The possibility has inspired MeiraGTx to develop a riboswitch technology that is designed to allow for the precise, dose-responsive control of gene expression by oral small molecules. The riboswitch technology is just part of MeiraGTx’s work in gene therapy. The company has technologies for the optimization of adeno-associated virus (AAV) vectors and for the design of promoter sequences. Also, the company has an internally developed manufacturing platform process and several production facilities. 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The family of the toddler taking part in a medical trial has called the change in their daughter "mind-blowing." Opal Sandy, now 18 months old, was born with total deafness due to a fault in the OTOF gene, which makes a protein called Otoferlin. Otoferlin enables communication between cells of the inner ear, or cochlea, and the brain. As part of a trial run by Cambridge University, Opal received an infusion of a working copy of the OTOF gene in her right ear. 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On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. 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This is the first FDA approval for a gene therapy addressing pre-symptomatic late infantile, early juvenile or early symptomatic early juvenile MLD. Lenmeldy is a single-use, personalised infusion, utilising the patient’s genetically modified hematopoietic stem cells (HSCs) to halt disease progression. The gene therapy involves collecting HSCs from the patient and modifying them to include functional copies of the ARSA gene. 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A group of patients with a hereditary disorder have had their lives transformed by a single treatment of a breakthrough gene-editing therapy, according to the lead researcher. The patients from New Zealand, the Netherlands and the UK have hereditary angioedema, a genetic disorder characterised by severe, painful and unpredictable swelling attacks. 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CRISPR Therapeutics and Vertex Pharmaceuticals’ gene editing cell therapy, branded as Casgevy, has officially been approved to treat TDT — a rare inherited blood disorder that requires regular blood transfusions — in patients 12 and over. 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Peter Marks, the Food and Drug Administration’s (FDA) top regulator of gene therapies, has indicated the full approval of Sarepta’s Duchenne gene therapy, Elevidys, is imminent. 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The children were born deaf because they inherited two defective copies of the gene for a protein called otoferlin that helps the inner ear’s hair cells transmit sound to the brain. In an attempt to restore this function, researchers injected harmless viruses carrying DNA for a working copy of the otoferlin gene into the children’s ears. 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The unusual approach could be a way to more reliably deliver gene therapies directly to the epithelial cells of the lungs. The company said preclinical research suggests its technology — dubbed InGenuiTy — could work with high efficiency and long-lasting effects. AlveoGene aims to bring its first candidate, a treatment for patients with alpha-1 antitrypsin deficiency or AATD, into clinical testing over the next two to three years. 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The technology works by targeting the NaV1.7 sodium ion channel present on neurons, which is an important component of the pain response. The researchers encoded a version of a peptide that allows a modulatory protein, called CRMP2, to bind to NaV1.7 sodium ion channels and modulate their activity. Treating neurons so that they now express this peptide interfered with the ability of CRMP2 to affect the sodium channel, reducing the transmission of pain. 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Similar treatments could help millions</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 26 July 2023, source: Albuquerque Journal</em><br />Dr. Alfonso Sabater pulled up two photos of Antonio Vento Carvajal’s eyes. One showed cloudy scars covering both eyeballs. The other, taken after months of gene therapy given through eyedrops, revealed no scarring on either eye. Antonio, who's been legally blind for much of his 14 years, can see again. The teen was born with dystrophic epidermolysis bullosa, a rare genetic condition that causes blisters all over his body and in his eyes. But his skin improved when he joined a clinical trial to test the world’s first topical gene therapy. That gave Sabater an idea: What if it could be adapted for Antonio's eyes? <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/767-gene-therapy-eyedrops-restored-a-boy-s-sight-similar-treatments-could-help-millions.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/767-gene-therapy-eyedrops-restored-a-boy-s-sight-similar-treatments-could-help-millions.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="Gene therapy eyedrops restored a boy's sight. 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Connor Stoll from Chicago was the first patient in the nation to receive the infusion as part of a clinical trial at Nationwide Children’s Hospital in January 2018. <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/763-fda-needs-more-cell,-gene-therapy-mid-managers-to-speed-reviews-2.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/763-fda-needs-more-cell,-gene-therapy-mid-managers-to-speed-reviews-2.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="Gene therapy approved by FDA for certain boys with rare genetic condition" data-count="horizontal" data-via="genetherapynet" data-url="https://www.genetherapynet.com/gene-therapy-news/763-fda-needs-more-cell,-gene-therapy-mid-managers-to-speed-reviews-2.html" data-lang="en"></a> </div> <script> var loadAsyncDeferredTwitter = function() { var d = document; 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Fixed and congenital dilated pupils, patent ductus arteriosus/aorto pulmonary window and other complications related to the bladder, lungs and guts, are the common manifestations of this mutation with only 60 diagnosed people in the world, most of them children. <a href="https://docs.google.com/forms/d/e/1FAIpQLSf1YkrzNbjwucxXgjfmZs6R6G8FtXX9r_2tbqZwptyAx8QUoA/viewform">Join the 2023 MSMDS Conference</a> taking place May 5-7 in Boston and online. <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); 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Inside the gut of a caterpillar lives a worm, and inside the worm lurks a bioluminescent bacterium named <i>Photorhabdus asymbiotica</i>, which makes the caterpillar glow in the dark. But this nesting-doll-like setup has another, more harmful effect: the bacteria secrete a deadly molecular syringe, 100 nanometers long, that latches onto the insect’s cells. Once attached to a cell, the syringe pushes a molecular spear through the cell’s membrane that releases a toxic payload. 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Hemgenix, originally developed by uniQure, is approved for adults with severe and moderately severe hemophilia B without a history of inhibitors. 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This was one of the learnings imparted by officials during a 7 February virtual town hall meeting to answer stakeholder questions on the clinical development of gene therapies for rare diseases outside the hematology and oncology space. 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A rare genetic disorder kept her from even lifting her head. Her parents took turns holding her upright at night just so she could breathe comfortably and sleep. Then, months later. doctors delivered gene therapy directly to her brain. 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The novel adenovirus vector-based gene therapy is specifically indicated for patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumours and is to be administered into the patient's bladder once every three months. 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The adeno-associated virus vector-based therapy is the first-of-its-kind for the treatment of Hemophilia B. “Gene therapy for hemophilia has been on the horizon for more than two decades,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in a press release. “Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life.” <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); 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