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The rare and deadly genetic liver disorder, GSD type Ia, affects children from infancy through adulthood, causing dangerously low blood sugar levels and constant dependence on glucose consumption in the form of cornstarch every few hours for survival. If a cornstarch dose is missed, the disease can lead to seizures and even death. Weinstein, whose team first administered the investigational gene therapy at UConn John Dempsey Hospital in Farmington, Connecticut, on July 24, 2018, calls the results “remarkable.” </span><br /> <a style="font-weight: normal; font-size: 12px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: underline;" href="https://www.genetherapynet.com">Read more...</a></td> </tr> </tbody> </table> <hr /> <table style="margin-bottom: 10px;" width="100%" align="center"> <tbody> <tr> <td height="25"><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 16px; font-weight: bold;"> Conference: 3rd Complement-based Drug Development Summit</span></td> </tr> <tr> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">The <a href="https://ter.li/hsr3cz">Complement-based Drug Development Summit</a>, <b>13-15 November 2019</b> in <b>Boston</b>, is the only industry dedicated meeting that brings you the most up to date clinical and commercial developments utilizing complement inhibitors in rare and common disease indications. You will gain key insight on overcoming end to end challenges from preclinical modelling to streamlining process development. This event will enable you to understand the fundamental role of complement in specialized cells to manipulate the complement cascade to treat common and rare diseases. Join experts from InflaRx, Apellis, Novartis, Roche, Genentech and Achillion to discuss targeting the classical, lectin and alternative pathways and creating robust testing and assay measures for patient selection and response. With over 150 of the industry’s pioneers we will gather to advance to the clinic novel complement based drugs beyond C5. </span><br /> <a style="font-weight: normal; font-size: 12px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: underline;" href="https://ter.li/hsr3cz">Register now...</a></td> </tr> </tbody> </table> <hr /> <table style="margin-bottom: 10px;" width="100%" align="center"> <tbody> <tr> <td height="25"><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 16px; font-weight: bold;"> Conference: 1st RNA Editing Summit</span></td> </tr> <tr> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;"> As CRISPR genome editing struggles to overcome key specificity and delivery challenges in its pursuit to realize therapeutic applications, the <a href="https://ter.li/4tik26">1st RNA Editing Summit 2019</a>, <b>19-20 November 2019</b> in <b>Boston</b>, accelerates the translation and commercialization of RNA editing for an array of research and therapeutic applications. As the therapeutic potential of RNA editing explodes, discover the fundamental mechanisms of RNA editing, such as recruiting endogenous ADARs with encodable guideRNAs, and learn about the novel approaches being developed to precisely and efficiently deliver intended RNA edits. With the temporary nature of RNA editing providing a huge opportunity in many areas beyond genetic diseases, uncover how to recruit endogenous editing enzymes and ultimately deliver safe and effective transcriptome edits. Join RNA, functional genomic, cell biology and immuno-oncology experts from large pharma, biotech and academia at the 1st RNA Editing Summit 2019 in the pursuit of translating emerging RNA editing mechanisms into therapeutic reality. </span><br /> <a style="font-weight: normal; font-size: 12px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: underline;" href="https://ter.li/4tik26">Register now...</a></td> </tr> </tbody> </table> <hr /> <table style="margin-bottom: 10px;" width="100%" align="center"> <tbody> <tr> <td height="25"><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 16px; font-weight: bold;"> Conference: 2nd Annual RNA-Targeted Drug Discovery Summit</span></td> </tr> <tr> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">Finally drug the ‘undruggable’ RNA! Join 100+ of your colleagues from large pharma, innovative biotech and KOLs of academia at the <a href="https://rna-drugdiscovery.com/take-part/register/?utm_source=external-gene-therapy-net&utm_medium=newsletter&utm_campaign=13149-rna-tdd-gene-therapy-net-enewsletter">2nd RNA- Targeted Drug Discovery Summit</a>, <b>2-4 December 2019</b> in <b>Boston</b> to discuss unique discovery and development challenges that must be overcome for novel RNA targeted small molecule therapeutics to achieve their seemingly limitless potential. As a Gene Therapy Net subscriber, use your unique 10% discount code: GTN10. </span><br /> <a style="font-weight: normal; font-size: 12px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: underline;" href="https://rna-drugdiscovery.com/take-part/register/?utm_source=external-gene-therapy-net&utm_medium=newsletter&utm_campaign=13149-rna-tdd-gene-therapy-net-enewsletter">Register now...</a></td> </tr> </tbody> </table> <hr /> <table style="margin-bottom: 10px;" width="100%" align="center"> <tbody> <tr> <td height="25"><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 16px; font-weight: bold;"> Conference: Gene Therapy for Neurological Disorders</span></td> </tr> <tr> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">Discover Novel Gene Therapy Delivery Routes to Effectively Target the CNS, Optimizing Preclinical Models to Confidently Translate into the Clinic & Mitigate Immunogenicity to Deliver an Efficacious Dose. Built with the field’s thought-leaders from the likes of Pfizer, Biogen,Voyager and Novartis, GTxN is the industry’s first and only meeting dedicated to solving your translational drug development challenges enabling you to accelerate the development of your neurological gene therapy candidate. Join 100+ of your peers at this definitive conference (<a href="https://ter.li/wvta3x">Gene Therapy for Neurological Disorders Summit,GTxN</a>, <b>10-12 December</b> in <b>Boston</b>) over 3 days, 2 interactive workshops, 6+ hours of dedicated networking time and 18 expert industry speakers. </span><br /> <a style="font-weight: normal; font-size: 12px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: underline;" href="https://ter.li/wvta3x">Register now...</a></td> </tr> </tbody> </table> <hr /> <table style="margin-bottom: 10px;" width="100%" align="center"> <tbody> <tr> <td height="25"><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 16px; font-weight: bold;"> Company: Batavia Biosciences B.V.</span></td> </tr> <tr> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;"><a href="https://www.bataviabiosciences.com/">Batavia Biosciences</a> is a contract development and manufacturing organization, specialized in vaccines and gene therapies. To accelerate the journey to the clinic, we offer a full range of development and manufacturing services for all major classes of biopharmaceutical, and have developed a portfolio of technologies aimed to improve product yield, reduce process development time and increase product stability. These technologies have demonstrated to reduce risks, improve timelines and reduce costs in biopharmaceutical development to make sure the product receives the patient without any unnecessary delays. We are an active player in Global Health, where we collaborate with prominent academic institutions and public organizations to improve the availability and affordability of key biopharmaceuticals for all in need. </span><br /> <a style="font-weight: normal; font-size: 12px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: underline;" href="https://www.bataviabiosciences.com/">Read more...</a></td> </tr> </tbody> </table> <hr /> <table style="margin-bottom: 10px;" width="100%" align="center"> <tbody> <tr> <td height="25"><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 16px; font-weight: bold;"> Conferences </span></td> </tr> <tr> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;"> <table> <tbody> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">15 – 16 October 2019, 9th Annual Orphan Drugs and Rare Diseases conference, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">15 – 17 October 2019, Gene Therapy for Rare Disorders Europe 2019, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">21 – 22 October 2019, NORD's 2019 Rare Diseases & Orphan Products Breakthrough Summit, Washington, D.C.</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">21 – 25 October 2019, XXVII Congress of the European Society of Gene and Cell Therapy (ESGCT), Barcelona, Spain</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">22 – 24 October 2019, Macrophage-directed Therapies Summit, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">23 – 25 October 2019, Neuromuscular Drug Development Summit - Gene Therapies, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">24 – 25 October 2019, 5th Annual Cell & Gene Therapy Congress, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">29 – 31 October 2019, Cell and Gene Therapy Manufacturing Forum event, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">7 – 8 November 2019, 5th Annual Genome Editing Congress, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">12 – 14 November 2019, World Orphan Drug Congress, Barcelona, Spain</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">13 – 15 November 2019, 3rd Complement-based Drug Development Summit - Gene Therapy, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">19 – 20 November 2019, 1st RNA Editing Summit, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">19 – 20 November 2019, 3rd Annual Ophthalmic Drugs Conference - gene therapy for eye diseases, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">2 – 4 December 2019, 2nd RNA-Targeted Drug Discovery Summit, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">10 – 12 December 2019, Gene Therapy for Neurological Disorders, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">20 - 24 January 2020, PepTalk event on Cell and Gene Therapies, San Diego, CA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">29 - 30 January 2020, CAR-T Congress EU, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">29 - 30 January 2020, Vein-to-Vein Congress EU - Manufacturing, scale-up, regulatory and logistical bottlenecks to commercialise cell therapies, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">5 - 6 February 2020, Annual Cell and Gene Therapy Innovation Summit, Berlin, Germany</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">22 - 24 February 2020, CAR-TCR Summit Europe, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">2 - 3 March 2020, Tri-Conference 2020 - Renaissance of Gene Therapy and Genome Editing, San Francisco, CA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">23 - 25 March 2020, International Society for BioProcess Technology 10th Spring Meeting - Viral vectors and vaccines, Norfolk, Virginia</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">30 March - 2 April 2020, 4th Annual Gene Therapy for Rare Disorders, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">31 March - 2 April 2020, 15th Annual World Advanced Therapies Congress, London, UK</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">29 - 30 April 2020, CAR-T Congress USA, Boston, MA</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">12 - 15 May 2020, American Society of Gene and Cell Therapy (ASGCT) 23th Annual Meeting, Boston, MA </span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">15 - 19 June 2020, 9th International Symposium on Monolith - DNA/RNA, vaccines, gene therapy vectors, Portoroz, Slovenia</span></td> </tr> <tr> <td valign="top">-</td> <td><span style="font-family: Geneva, Arial, Helvetica, sans-serif; color: #000000; font-size: 12px; font-weight: normal;">20 – 23 October 2020, XXVIII Congress of the European Society of Gene and Cell Therapy (ESGCT), Edinburgh UK</span></td> </tr> </tbody> </table> </span></td> </tr> </tbody> </table> <br /> <a style="font-weight: normal; font-size: 12px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: underline;" href="/conferences.html">Read more...</a></td> </tr> <tr style="line-height: 0px;"></tr> </tbody> </table> <table style="margin-bottom: 20px; text-align: center;" width="100%" bgcolor="#e3e5e6" align="center"> <tbody> <tr> <td><center><hr /><a style="font-weight: normal; font-size: 11px; color: #000000; font-family: Geneva, Arial, Helvetica, sans-serif; text-decoration: none;" href="https://www.genetherapynet.com/"><strong>Gene Therapy Net</strong> is the information resource for basic and clinical research in gene therapy, and the site serves as a network in the exchange of gene therapy information and breaking news items. 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You are currently registered as: <strong>{subtag:name}</strong>, email address: {subtag:email}.</span></center></td> </tr> </tbody> </table> </td> </tr> </tbody> </table></div> <div class="uk-margin-top"><a href="/october-2019.html" class="el-link uk-button uk-button-default">Read more</a></div> </div></div> </div> </div> </div> <div class="uk-width-1-2 uk-width-1-4@m uk-width-1-5@l rechts"> <div> <div class="uk-grid uk-child-width-1-1 uk-grid-row-small" uk-grid> <div> <div class="uk-panel _menu menulist" id="module-123"> <h3> Newsletter Archive </h3> <ul class="uk-nav uk-nav-default"> <li class="item-1160"><a href="/december-2021.html"> December 2021</a></li> <li class="item-1159"><a href="/november-2021.html"> November 2021</a></li> <li class="item-1158"><a href="/october-2021.html"> October 2021</a></li> <li class="item-1157"><a href="/september-2021.html"> September 2021</a></li> <li class="item-1156"><a href="/august-2021.html"> August 2021</a></li> <li class="item-1155"><a 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