{"id":20069,"date":"2023-11-21T17:02:46","date_gmt":"2023-11-21T22:02:46","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=20069"},"modified":"2023-12-20T05:15:32","modified_gmt":"2023-12-20T10:15:32","slug":"emilys-entourage-to-host-scientific-symposium-on-the-promise-of-gene-based-therapies-for-cystic-fibrosis","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/sv\/emilys-entourage-to-host-scientific-symposium-on-the-promise-of-gene-based-therapies-for-cystic-fibrosis\/","title":{"rendered":"Emily's Entourage v\u00e4rd f\u00f6r vetenskapligt symposium om l\u00f6ftet om genbaserade behandlingar f\u00f6r cystisk fibros"},"content":{"rendered":"

Philadelphia, PA - 21 november<\/strong>, 2023<\/strong> - Emily's Entourage (EE), en innovativ 501(c)3 som p\u00e5skyndar forskning f\u00f6r nya behandlingar och ett botemedel f\u00f6r de sista 10% hos personer med cystisk fibros (CF), inklusive de med nonsensmutationer, tillk\u00e4nnagav idag sitt vetenskapliga symposium 2023, <\/span>L\u00f6ftet om genbaserade terapier f\u00f6r cystisk fibros<\/span><\/i>som kommer att h\u00e5llas den 4-5 december 2023 p\u00e5 Quorum i University City Science Center.\u00a0<\/span><\/p>\n

Temat f\u00f6r symposiet \u00e4r en bekr\u00e4ftelse p\u00e5 de senaste framstegen i utvecklingen av genbaserade behandlingar vid CF som har potential att n\u00e5 de sista 10% i CF-samfundet som inte har nytta av dagens tillg\u00e4ngliga behandlingar, och som kan leda till en botande behandling f\u00f6r alla med CF. Trots betydande framsteg kvarst\u00e5r kritiska utmaningar n\u00e4r det g\u00e4ller att utveckla och leverera nya genbaserade behandlingar f\u00f6r CF. Symposiet samlar ledande personer inom - och utanf\u00f6r - forskarv\u00e4rlden, industrin och FDA f\u00f6r att \u00f6ppet dela med sig, samarbeta, st\u00e4lla sv\u00e5ra fr\u00e5gor och i slut\u00e4ndan skapa l\u00f6sningar och strategiska, snabbare v\u00e4gar fram\u00e5t.<\/span><\/p>\n

Symposiet, som leds av Dr Patrick Harrison, PhD, Maria Limberis, PhD, och Batsheva Kerem, PhD, \u00e4r endast tillg\u00e4ngligt f\u00f6r inbjudna och samlar 34 talare och 86 deltagare fr\u00e5n 18 l\u00e4nder. Bland de inbjudna talarna finns Dr. Drew Weissman, BS, MS, PhD, MD, mottagare av Nobelpriset i fysiologi eller medicin 2023.\u00a0<\/span><\/p>\n

Symposiet omfattar sju tankev\u00e4ckande sessioner som p\u00e5g\u00e5r under tv\u00e5 dagar. Varje session kommer att erbjuda presentationer och rikliga m\u00f6jligheter att delta i djupg\u00e5ende diskussioner. Som den stora finalen kommer de tv\u00e5 avslutande sessionerna att ta formen av dynamiska rundabordsdiskussioner, modererade av experter och berikade av insikter fr\u00e5n flera paneldeltagare.<\/span><\/p>\n

I. <\/b>CF-terapier - f\u00f6rflutet, nutid och framtid<\/b><\/p>\n

Paul Negulescu, PhD | Vertex Pharmaceuticals
\n<\/span>Jennifer Taylor-Cousar, MD, MSCS, ATSF | National Jewish Health
\n<\/span>Kara Foshay, PhD | Cystic Fibrosis Foundation<\/span><\/p>\n

II. RNA-baserade metoder<\/b><\/p>\n

Drew Weissman, BS, MS, PhD, MD | University of Pennsylvania
\n<\/span>Fred Van Goor, PhD | Vertex Pharmaceuticals
\n<\/span>John Lueck, PhD | University of Rochester, School of Medicine and Dentistry
\n<\/span>Zoya Ignatova, PhD | Universitetet i Hamburg<\/span><\/p>\n

III. <\/b>Utnyttja nya leveransplattformar f\u00f6r mRNA-, tRNA- och oligonukleotidbaserade metoder<\/b><\/p>\n

Rana Ghosh, PhD | University of Texas at Austin
\n<\/span>Alexandra Piotrowski-Daspit, PhD | University of Michigan
\n<\/span>Marc Abrams, PhD | Carbon Biosciences<\/span><\/p>\n

IV. CFTR-genterapi och genredigering vid CF<\/b><\/p>\n

Marianne Carlon, PhD | KU Leuven
\n<\/span>Anna Cereseto, PhD | Universitetet i Trento
\n<\/span>Smriti Pandey | Harvard University
\n<\/span>Greg Newby, PhD | John Hopkins University
\n<\/span>Ahad Rahim, PhD | University College London<\/span><\/p>\n

V. Aktuellt l\u00e4ge f\u00f6r genbaserade terapier<\/b><\/p>\n

Matt Porteus, MD, PhD | Stanford University
\n<\/span>Kiran Musunuru, MD, PhD, MPH, ML | University of Pennsylvania
\n<\/span>Marcus Noyes, PhD | NYU Grossman School of Medicine<\/span><\/p>\n

VI. Perspektiv p\u00e5 s\u00e4llsynta genterapier f\u00f6r att p\u00e5skynda utvecklingen av terapier f\u00f6r CF<\/b>\u00a0<\/b><\/p>\n

Maria Limberis, PhD (Moderator)
\n<\/span>David Geller, MD | Arcturus Therapeutics
\n<\/span>Patrick Harrison, PhD | University College Cork
\n<\/span>Gili Hart, PhD | Splisense
\n<\/span>Craig Hodges, PhD | Case Western Reserve University
\n<\/span>Prateek Shukla, MD | FDA
\n<\/span>Richard Horgan, MBA | Cure Rare Disease<\/span><\/p>\n

VII. Utmaningar i kliniska pr\u00f6vningar av genterapi i ett CF-perspektiv<\/b><\/p>\n

Jennifer Taylor-Cousar, MD, MSCS, ATSF | National Jewish Health (moderator)
\n<\/span>Marty Burke, PhD, MD | University of Illinois
\n<\/span>Ilan Ganot, MBA | Solid Biosciences
\n<\/span>Jessica Lee | Spirovant
\n<\/span>Joel Schneider, PhD | Carbon Biosciences
\n<\/span>Wolfgang Seibold, MD | Boehringer Ingelheim
\n<\/span>Alan Cohen, l\u00e4kare | 4DMT<\/span><\/p>\n

\"Det har gjorts enorma framsteg i utvecklingen av genbaserade terapier under de senaste \u00e5ren. Vi \u00e4r uppmuntrade av framstegen eftersom de utg\u00f6r en ljusglimt av hopp f\u00f6r alla i CF-samh\u00e4llet och s\u00e4rskilt f\u00f6r de sista 10% som inte gynnas av befintliga mutationsinriktade terapier och som fortfarande desperat v\u00e4ntar p\u00e5 behandlingsalternativ\", s\u00e4ger Chandra Ghose, Chief Scientific Officer p\u00e5 Emily's Entourage. \"Vi \u00e4r mycket glada \u00f6ver att kunna samla ledare i v\u00e4rldsklass till v\u00e5rt symposium f\u00f6r att tillsammans kartl\u00e4gga en snabb v\u00e4g till en framtid d\u00e4r 100% av CF-gruppen har tillg\u00e5ng till livr\u00e4ddande behandlingar som g\u00f6r att de kan leva ett l\u00e5ngt och h\u00e4lsosamt liv, oavsett deras genetiska mutation.\"<\/span><\/p>\n

EE tackar sponsorerna f\u00f6r 2023 \u00e5rs symposium: Presenting, Dream Team och Violet-Level, <\/span>Viatris,<\/b> 4D Molekyl\u00e4r terapi<\/b>, <\/span>Vertex Pharmaceuticals, Inc, ReCode Therapeutics, Boomer Esiason Foundation,<\/b> och mer<\/span> vars st\u00f6d g\u00f6r denna viktiga h\u00e4ndelse och de framsteg som den leder till m\u00f6jliga.\u00a0\u00a0<\/span><\/p>\n

##<\/span><\/p>\n

Om Emily's Entourage<\/b><\/p>\n

Emily's Entourage \u00e4r en innovativ 501(c)3 som p\u00e5skyndar forskning f\u00f6r nya behandlingar och ett botemedel f\u00f6r individer i den sista 10% av cystisk fibros (CF)-populationen som inte gynnas av f\u00f6r n\u00e4rvarande tillg\u00e4ngliga mutationsinriktade terapier. Sedan 2011 har Emily's Entourage beviljat miljontals dollar i forskningsanslag, lanserat ett nu f\u00f6rv\u00e4rvat CF-genterapibolag, utvecklat ett patientregister och ett matchningsprogram f\u00f6r kliniska pr\u00f6vningar f\u00f6r att p\u00e5skynda rekryteringen till kliniska pr\u00f6vningar samt lett v\u00e4rldsomsp\u00e4nnande insatser f\u00f6r att driva forskning och l\u00e4kemedelsutveckling med stor genomslagskraft. Organisationen har omn\u00e4mnts i nationella medier, bland annat New York Times, STAT, CNN, People med flera. L\u00e4s mer p\u00e5 <\/span>emilysentourage.org<\/span><\/a>.\u00a0<\/span><\/p>\n

 <\/p>\n

Kontakt med media<\/span><\/p>\n

Emily's Entourage<\/span><\/p>\n

admin@emilysentourage.org<\/span><\/a><\/p>","protected":false},"excerpt":{"rendered":"

Philadelphia, PA - 21 november 2023 - Emily's Entourage (EE), en innovativ 501(c)3 som p\u00e5skyndar forskning f\u00f6r nya behandlingar och ett botemedel f\u00f6r de sista 10% av personer med cystisk fibros (CF), inklusive de med nonsensmutationer, tillk\u00e4nnagav idag sitt vetenskapliga symposium 2023, The Promise of Gene-Based Therapies for Cystic Fibrosis, som kommer att h\u00e5llas den... <\/p>\n

<\/div>\n

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