{"id":23831,"date":"2024-11-18T09:30:37","date_gmt":"2024-11-18T13:30:37","guid":{"rendered":"https:\/\/www.emilysentourage.org\/?p=23831"},"modified":"2024-11-18T11:45:19","modified_gmt":"2024-11-18T15:45:19","slug":"emilys-entourage-celebrates-landmark-achievement-first-patient-dosed-with-novel-gene-therapy-developed-through-ees-visionary-model","status":"publish","type":"post","link":"https:\/\/www.emilysentourage.org\/tr\/emilys-entourage-celebrates-landmark-achievement-first-patient-dosed-with-novel-gene-therapy-developed-through-ees-visionary-model\/","title":{"rendered":"Emily's Entourage D\u00f6n\u00fcm Noktas\u0131 Ba\u015far\u0131s\u0131n\u0131 Kutluyor: EE'nin Vizyoner Modeliyle Geli\u015ftirilen Yeni Gen Tedavisi \u0130lk Hastaya Uyguland\u0131"},"content":{"rendered":"

Philadelphia, Pennsylvania-18 Kas\u0131m 2024<\/b>-Kistik fibrozis (KF) hastalar\u0131n\u0131n son 10%'si i\u00e7in yeni tedaviler ve bir tedavi i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3 olan Emily's Entourage (EE), \u015fu anda mevcut mutasyon hedefli tedavilerden yararlanamayan KF hastalar\u0131 i\u00e7in at\u0131l\u0131mlar\u0131 h\u0131zland\u0131rma misyonunda \u00f6nemli bir kilometre ta\u015f\u0131n\u0131 gururla duyurur: EE'nin kurulmas\u0131na yard\u0131mc\u0131 oldu\u011fu klinik a\u015famadaki bir gen terapisi \u015firketi olan Spirovant Sciences, KF tedavisi i\u00e7in SP-101 + Augmenter'in SAAVe Faz 1\/2 klinik \u00e7al\u0131\u015fmas\u0131nda ilk hastay\u0131 dozlad\u0131.<\/p>\n

\"\"Bu ba\u015far\u0131 EE i\u00e7in tam bir d\u00f6ng\u00fc an\u0131n\u0131 temsil etmektedir. EE, 2015 y\u0131l\u0131nda o zamanki ad\u0131 Talee Bio olan Spirovant'\u0131n kurulmas\u0131na kataliz\u00f6rl\u00fck etmi\u015f, bilim camias\u0131n\u0131 ve bir y\u00f6netim \u015firketini bir araya getirmi\u015f ve Spirovant Sciences'\u0131 ba\u015flatmak i\u00e7in tohum finansman\u0131 sa\u011flam\u0131\u015ft\u0131. Yakla\u015f\u0131k on y\u0131l sonra, bu ilk yat\u0131r\u0131m, inhalasyon yoluyla verilen ve \u015fu anda klinik bir deneyde ilerleyen mutasyon tan\u0131l\u0131 bir gen terapisi olan SP-101'in geli\u015ftirilmesine yol a\u00e7t\u0131.<\/p>\n

Emily's Entourage'\u0131n kurucu orta\u011f\u0131 Emily Kramer-Golinkoff, MBE, \"Bu d\u00f6n\u00fcm noktas\u0131 ola\u011fan\u00fcst\u00fc bir \u015fey\" dedi. \"KF toplulu\u011funun son 10%'sinde olanlar i\u00e7in hayat kurtaran bilimi h\u0131zland\u0131rma hayali olarak ba\u015flayan \u015fey, bu d\u00f6n\u00fc\u015ft\u00fcr\u00fcc\u00fc ana d\u00f6n\u00fc\u015ft\u00fc: KF'li bir ki\u015fiye uygulanan yeni bir gen tedavisi. Birlikte, bir fikri klinik \u00f6ncesi ara\u015ft\u0131rmalardan insan deneylerine ta\u015f\u0131d\u0131k. Bu ilerleme, \u00e7al\u0131\u015fmalar\u0131m\u0131z\u0131n neyle ilgili oldu\u011funu \u00f6rneklemektedir.\"<\/p>\n

EE'nin vizyoneri 'Giri\u015fim Filantropisi'<\/a> KF i\u00e7in ara\u015ft\u0131rma ve terap\u00f6tik geli\u015ftirmeyi te\u015fvik etme yakla\u015f\u0131m\u0131 Spirovant'\u0131n kurulu\u015funu h\u0131zland\u0131rd\u0131 ve KF'nin temel nedenini hedef alarak CFTR genini iletmek i\u00e7in bir adeno-ili\u015fkili vir\u00fcs (AAV) vekt\u00f6r\u00fcnden yararlanan SP-101 \u00fczerindeki \u00e7al\u0131\u015fmalar\u0131n\u0131 destekledi. SP-101 mutasyon agnostiktir, yani etkili oldu\u011fu kan\u0131tlan\u0131rsa, \u015fu anda mevcut mutasyon hedefli tedavilerden yararlanmayanlar da dahil olmak \u00fczere t\u00fcm KF mutasyonlar\u0131 i\u00e7in i\u015fe yarayacakt\u0131r.<\/p>\n

Emily's Entourage'\u0131n Bilimsel Ba\u015f Sorumlusu Chandra Ghose, \"SP-101 ile dozlanan ilk hasta, KF toplulu\u011funda, \u00f6zellikle de son 10%'de kar\u015f\u0131lanmam\u0131\u015f ihtiya\u00e7lar\u0131 kar\u015f\u0131lamaya y\u00f6nelik kararl\u0131 misyonumuzda cesaret verici bir and\u0131r\" dedi. \"Bu d\u00f6n\u00fcm noktas\u0131, y\u0131llarca s\u00fcren \u00f6zveri, yenilik\u00e7ilik ve i\u015fbirli\u011fini yans\u0131t\u0131yor; stratejik yat\u0131r\u0131mlar ve kolektif \u00e7abayla bilimsel potansiyeli yeni se\u00e7enekler bekleyenler i\u00e7in anlaml\u0131 bir ilerlemeye d\u00f6n\u00fc\u015ft\u00fcrebilece\u011fimizin kan\u0131t\u0131.\"<\/p>\n

SAAVe Faz 1\/2 \u00e7al\u0131\u015fmas\u0131, mevcut tedavilerin yetersiz kald\u0131\u011f\u0131 veya kullan\u0131lamad\u0131\u011f\u0131 bireylerde g\u00fcvenlik, tolere edilebilirlik ve etkinli\u011fi de\u011ferlendirmek \u00fczere tasarlanm\u0131\u015ft\u0131r. Temel ikincil sonlan\u0131m noktalar\u0131 aras\u0131nda akci\u011fer fonksiyonu, ya\u015fam kalitesi ve terap\u00f6tik etkiyi g\u00f6steren biyobelirte\u00e7ler yer almaktad\u0131r.<\/p>\n

Kramer-Golinkoff, \"Bu ba\u015far\u0131, inan\u0131lmaz toplulu\u011fumuzun deste\u011fi olmadan m\u00fcmk\u00fcn olamazd\u0131,\" diye ekledi. \"Y\u0131llar boyunca ba\u011f\u0131\u015f yapan, kat\u0131lan veya misyonumuzu herhangi bir \u015fekilde payla\u015fan herkese: bu d\u00f6n\u00fcm noktas\u0131 sizin de d\u00f6n\u00fcm noktan\u0131z ve size t\u00fcm kalbimizle te\u015fekk\u00fcr ediyoruz.\"<\/p>\n

SP-101 klinik ara\u015ft\u0131rmas\u0131 hakk\u0131nda daha fazla bilgi i\u00e7in Spirovant'\u0131n \u015fu adresteki duyurusunun tamam\u0131n\u0131 ziyaret edin PRNewswire<\/a>.<\/p>\n

 <\/p>\n

Emily'nin \u00c7evresi Hakk\u0131nda<\/b><\/p>\n

Emily's Entourage, mevcut mutasyon hedefli tedavilerden yararlanamayan kistik fibrozis (KF) pop\u00fclasyonunun son 10%'sindeki bireyler i\u00e7in yeni tedaviler ve bir tedavi i\u00e7in ara\u015ft\u0131rmalar\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3't\u00fcr. Emily's Entourage 2011 y\u0131l\u0131ndan bu yana milyonlarca dolarl\u0131k ara\u015ft\u0131rma hibesi vermi\u015f, \u015fu anda sat\u0131n al\u0131nan bir KF gen terapisi \u015firketini kurmu\u015f, klinik deneylere kat\u0131l\u0131m\u0131 h\u0131zland\u0131rmak i\u00e7in bir hasta veri taban\u0131 ve klinik deney e\u015fle\u015ftirme program\u0131 geli\u015ftirmi\u015f ve d\u00fcnya \u00e7ap\u0131nda y\u00fcksek etkili ara\u015ft\u0131rma ve ila\u00e7 geli\u015ftirme \u00e7abalar\u0131na \u00f6nc\u00fcl\u00fck etmi\u015ftir. Kurulu\u015f, New York Times, STAT, CNN, People ve daha fazlas\u0131 dahil olmak \u00fczere ulusal medyada yer alm\u0131\u015ft\u0131r. Daha fazla bilgi i\u00e7in emilysentourage.org<\/a>.<\/p>\n

 <\/p>\n

Medya \u0130leti\u015fim<\/b><\/p>\n

Hannah Kleckner<\/p>\n

Pazarlama ve \u0130leti\u015fim Direkt\u00f6r\u00fc<\/p>\n

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

Philadelphia, Pennsylvania-18 Kas\u0131m 2024-Emily's Entourage (EE), kistik fibrozis (KF) hastalar\u0131n\u0131n son 10%'si i\u00e7in yeni tedaviler ve tedavi ara\u015ft\u0131rmalar\u0131n\u0131 h\u0131zland\u0131ran yenilik\u00e7i bir 501(c)3, \u015fu anda mevcut mutasyon hedefli tedavilerden yararlanamayan KF hastalar\u0131 i\u00e7in at\u0131l\u0131mlar\u0131 h\u0131zland\u0131rma misyonunda \u00f6nemli bir d\u00f6n\u00fcm noktas\u0131n\u0131 gururla duyurur: Spirovant Sciences, bir klinik a\u015fama... <\/p>\n

<\/div>\n

Daha fazla bilgi edinin<\/a><\/p>","protected":false},"author":1719,"featured_media":0,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_seopress_robots_primary_cat":"none","_seopress_titles_title":"%%post_title%%","_seopress_titles_desc":"","_seopress_robots_index":"","_monsterinsights_skip_tracking":false,"_monsterinsights_sitenote_active":false,"_monsterinsights_sitenote_note":"","_monsterinsights_sitenote_category":0,"_jetpack_memberships_contains_paid_content":false,"footnotes":""},"categories":[178],"tags":[198,197,199,200,201],"class_list":["post-23831","post","type-post","status-publish","format-standard","hentry","category-press-releases","tag-cf-clinical-trial","tag-cf-research","tag-clinical-trial","tag-sp-101","tag-spirovant"],"acf":[],"jetpack_featured_media_url":"","jetpack_sharing_enabled":true,"post_mailing_queue_ids":[],"_links":{"self":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/23831","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/users\/1719"}],"replies":[{"embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/comments?post=23831"}],"version-history":[{"count":5,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/23831\/revisions"}],"predecessor-version":[{"id":23841,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/posts\/23831\/revisions\/23841"}],"wp:attachment":[{"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/media?parent=23831"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/categories?post=23831"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.emilysentourage.org\/tr\/wp-json\/wp\/v2\/tags?post=23831"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}