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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"> <a href="/component/banners/click/164.html" target="_blank" rel="noopener noreferrer" title="4thCellTherapyPotencyAssaySummit-mrt2025"> <img src="https://www.genetherapynet.com/images/banners/4thCellTherapyPotencyAssaySummit-mrt2025.png#joomlaImage://local-images/banners/4thCellTherapyPotencyAssaySummit-mrt2025.png?width=468&height=60" alt="4thCellTherapyPotencyAssaySummit-mrt2025" > </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-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. 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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. 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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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