{"id":3833,"date":"2025-05-13T11:37:31","date_gmt":"2025-05-13T08:37:31","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=3833"},"modified":"2025-05-13T11:39:47","modified_gmt":"2025-05-13T08:39:47","slug":"pharma-giants-pull-back-from-aav-based-gene-therapy-research","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/tr\/pharma-giants-pull-back-from-aav-based-gene-therapy-research\/","title":{"rendered":"\u0130la\u00e7 Devlerinin AAV Tabanl\u0131 Gen Terapisi Ara\u015ft\u0131rmalar\u0131ndan \u00c7ekilmesinin Ard\u0131ndan Duchenne Musk\u00fcler Distrofi (DMD) Hastalar\u0131n\u0131 Ne Bekliyor?"},"content":{"rendered":"<p class=\"wp-block-paragraph\">Adeno ili\u015fkili vir\u00fcs (AAV) vekt\u00f6rleri bir zamanlar gen terapileri sunman\u0131n cevab\u0131 olarak duyuruluyordu, ancak son y\u0131llarda \u00f6nemi azald\u0131. \u0130lk olarak birka\u00e7 kal\u0131tsal hastal\u0131\u011f\u0131 tedavi etme yetene\u011fine sahip oldu\u011fu duyuruldu, ancak ara\u015ft\u0131rmalardaki d\u00fc\u015f\u00fc\u015f zaten fark edildi. Vertex Pharmaceuticals, AAV ara\u015ft\u0131rmalar\u0131na yat\u0131r\u0131m\u0131n\u0131 azaltan en son biyofarmas\u00f6tiktir ve bu teknoloji end\u00fcstrisi i\u00e7in bir aksiliktir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Endpoints News, uluslararas\u0131 biyofarmas\u00f6tiklerin t\u00fcm AAV ara\u015ft\u0131rmalar\u0131n\u0131 kesti\u011fini bildirdi. Bu, \u015f\u00fcphesiz tRNA terapileri \u00fcreticisi Tevard Biosciences ve ila\u00e7lar\u0131n\u0131 \u00fcretmek i\u00e7in AAV vekt\u00f6rlerinin \u00fcretimine ba\u011fl\u0131 bir gen terapisi i\u015fletmesi olan Affinia terapileri ile olan ortakl\u0131klar\u0131n\u0131 etkileyecektir. \u2013 <a href=\"https:\/\/endpts.com\/vertex-drops-aav-gene-therapy-research-trna-therapy-partnership\/\" target=\"_blank\" rel=\"noreferrer noopener\">Vertex, AAV gen terapisi ara\u015ft\u0131rmalar\u0131n\u0131 b\u0131rakt\u0131<\/a> \u2013<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u0130\u00e7indekiler<\/h2><nav><ul><li><a href=\"#history-of-aav-based-gene-therapy\">AAV Tabanl\u0131 Gen Terapisinin Tarih\u00e7esi<\/a><\/li><li><a href=\"#vertex-breaks-off-partnerships-with-affinia-therapeutics-and-tevard-biosciences\">Vertex, Affinia Therapeutics ve Tevard Biosciences ile ortakl\u0131klar\u0131n\u0131 sonland\u0131r\u0131yor<\/a><\/li><li><a href=\"#risks-of-aav-based-gene-therapies\">AAV Tabanl\u0131 Gen Terapilerinin Riskleri<\/a><\/li><li><a href=\"#roche-abandons-the-gene-therapy-unit-and-pfizer-discontinues-beqvez\">Roche gen terapisi \u00fcnitesini terk ediyor ve Pfizer Beqvez&#039;i sonland\u0131r\u0131yor<\/a><\/li><li><a href=\"#aa-vs-are-constrained-by-manufacturing-costs-and-payload-size\">AAV&#039;ler \u00fcretim maliyetleri ve y\u00fck boyutuyla s\u0131n\u0131rl\u0131d\u0131r<\/a><\/li><li><a href=\"#beyond-aav-research-looking-for-more-effective-ways-to-deliver-drugs\">AAV ara\u015ft\u0131rmas\u0131n\u0131n \u00f6tesinde: \u0130la\u00e7lar\u0131 ula\u015ft\u0131rman\u0131n daha etkili yollar\u0131n\u0131 ar\u0131yoruz<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"history-of-aav-based-gene-therapy\">AAV Tabanl\u0131 Gen Terapisinin Tarih\u00e7esi<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Vir\u00fcs\u00fcn ola\u011fan d\u0131\u015f k\u0131l\u0131f\u0131na sahip olmad\u0131klar\u0131 i\u00e7in, AAV&#039;ler genellikle \u00e7\u0131plak vir\u00fcsler veya zarfs\u0131z vir\u00fcsler olarak adland\u0131r\u0131l\u0131r. Bunun yerine, vir\u00fcs\u00fcn DNA&#039;s\u0131n\u0131 kaplayan bir protein kabu\u011fu olan bir kapsidleri vard\u0131r.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">1965&#039;te, adenovir\u00fcs preparatlar\u0131 haz\u0131rlarken, bilim insanlar\u0131 bu vir\u00fcsleri nispeten kas\u0131ts\u0131z bir \u015fekilde ke\u015ffettiler. \u0130la\u00e7lar\u0131 iletmek i\u00e7in AAV&#039;leri tasarlama konsepti, kaps\u00fcllenmi\u015f viral DNA nedeniyle ortaya \u00e7\u0131kt\u0131. Bir AAV vekt\u00f6r\u00fc, ke\u015ffedilmesinden otuz y\u0131l sonra, v\u00fccutta mukus birikmesine neden olan kal\u0131tsal bir durum olan kistik fibrozlu bir hastada ilk kez kullan\u0131ld\u0131.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha sonra, 2012&#039;de Avrupa Birli\u011fi (AB), onaylanan ilk gen terapisi olan Glybera&#039;y\u0131 onaylad\u0131. Glybera&#039;da gen, v\u00fccudun kan lipitlerini par\u00e7alayan bir enzimden yoksun oldu\u011fu nadir bir genetik bozukluk olan kal\u0131tsal lipoprotein lipaz eksikli\u011fini tedavi etmek i\u00e7in bir AAV vekt\u00f6r\u00fc arac\u0131l\u0131\u011f\u0131yla iletildi. Ancak be\u015f y\u0131l sonra, yarat\u0131c\u0131s\u0131 UniQure, y\u00fcksek maliyetler ve d\u00fc\u015f\u00fck talep nedeniyle tedaviyi piyasadan kald\u0131rd\u0131\u011f\u0131nda, tedaviyi \u00e7evreleyen co\u015fku suya d\u00fc\u015ft\u00fc.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ancak d\u00fczenleyici onay\u0131, bir\u00e7o\u011fu tedavi edici olan daha fazla AAV gen terapisinin piyasaya s\u00fcr\u00fclmesini m\u00fcmk\u00fcn k\u0131ld\u0131. Bunlar aras\u0131nda kas kayb\u0131na neden olan Duchenne kas distrofisi (DMD) i\u00e7in Elevidys, nadir g\u00f6r\u00fclen kal\u0131tsal g\u00f6z hastal\u0131\u011f\u0131 Leber&#039;in konjenital amorozisi i\u00e7in Luxturna, omurili\u011fi etkileyen n\u00f6rodejeneratif bir durum olan spinal kas atrofisi i\u00e7in Zolgensma ve nadir g\u00f6r\u00fclen kan hastal\u0131klar\u0131 hemofili B ve A i\u00e7in Hemgenix ve Roctavian yer al\u0131yor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"vertex-breaks-off-partnerships-with-affinia-therapeutics-and-tevard-biosciences\">Vertex, Affinia Therapeutics ve Tevard Biosciences ile ortakl\u0131klar\u0131n\u0131 sonland\u0131r\u0131yor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Ancak AAV gen tedavilerinin zaman\u0131n\u0131n ge\u00e7mi\u015f oldu\u011fu anla\u015f\u0131l\u0131yor. 2020&#039;de Affinia ile yap\u0131lan $1.6 milyarl\u0131k bir anla\u015fmada Vertex, DMD ve kistik fibrozu daha iyi tedavi etmek i\u00e7in AAV&#039;ler bulma s\u00f6z\u00fc vermi\u015fti. Ancak Affinia ge\u00e7en hafta Endpoints&#039;e DMD vekt\u00f6rlerinin haklar\u0131n\u0131 geri ald\u0131\u011f\u0131n\u0131 bildirdi. 2022&#039;de Vertex, kistik fibrozu hedef alan vekt\u00f6rlerin haklar\u0131ndan \u00e7oktan vazge\u00e7mi\u015fti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Di\u011fer \u015feylerin yan\u0131 s\u0131ra, Tevard ge\u00e7en y\u0131l\u0131n sonlar\u0131nda Vertex&#039;in DMD s\u00f6zle\u015fmesinden \u00e7ekildi\u011fi konusunda bilgilendirildi. Ayr\u0131ca, bu ay tRNA program\u0131n\u0131n haklar\u0131n\u0131 geri ald\u0131. Grup, bu hafta New Orleans&#039;ta d\u00fczenlenen Amerikan Gen ve H\u00fccre Terapisi Derne\u011fi (ASGCT) kongresinde tRNA terapisini desteklemek i\u00e7in verilerini sunmaya haz\u0131rlan\u0131rken, Tevard program konusunda iyimser.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"risks-of-aav-based-gene-therapies\">AAV Tabanl\u0131 Gen Terapilerinin Riskleri<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">B\u00fcy\u00fck ila\u00e7 \u015firketleri, Vertex&#039;in sahneden \u00e7ekilmesini de i\u00e7eren AAV ara\u015ft\u0131rma ve geli\u015ftirme (Ar-Ge) i\u015finden \u00e7ekiliyor. \u0130ki y\u0131l \u00f6nce, Japon \u00e7okuluslu ila\u00e7 \u015firketi Takeda, AAV gen tedavileri i\u00e7in klinik \u00f6ncesi ara\u015ft\u0131rmas\u0131n\u0131 sonland\u0131rd\u0131. O zamanlar pek fazla \u015fey bilinmese de, 2020 ile 2022 aras\u0131nda imzalad\u0131\u011f\u0131 on gen terapisi anla\u015fmas\u0131ndan ikisi olan Kaliforniya merkezli Codexis ve Massachusetts merkezli Selecta Biosciences ile AAV&#039;ye \u00f6zg\u00fc ittifaklar\u0131n\u0131n \u015f\u00fcpheli olaca\u011f\u0131 tahmin ediliyordu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Artan AAV g\u00fcvenli\u011fi endi\u015feleri kar\u015f\u0131s\u0131nda para tasarrufu yapmak amac\u0131yla ila\u00e7 devi Biogen, ayn\u0131 y\u0131l klinik \u00f6ncesi AAV gen terapisi ara\u015ft\u0131rmalar\u0131n\u0131 durdurdu.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Frans\u0131z biyoteknoloji \u015firketi EG 427&#039;nin kurucusu ve CEO&#039;su Philippe Chambon, vekt\u00f6rlerin g\u00fcvenlik profilinin ila\u00e7 da\u011f\u0131t\u0131m\u0131 a\u00e7\u0131s\u0131ndan kritik \u00f6neme sahip oldu\u011funu s\u00f6yledi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Chambon, &quot;AAV tabanl\u0131 gen terapileri, nadir hastal\u0131klar d\u0131\u015f\u0131nda etkili ve ekonomik uygulamalar\u0131n\u0131 engelleyen bir dizi sorun nedeniyle ila\u00e7 \u015firketleri aras\u0131nda ra\u011fbet g\u00f6rm\u00fcyor,&quot; dedi. &quot;Gen terapisinin, \u00f6nemli bir ila\u00e7 modalitesi olarak tam potansiyeline ula\u015fabilmesi i\u00e7in nadir hastal\u0131klar alan\u0131n\u0131n d\u0131\u015f\u0131ndaki kronik endikasyonlar\u0131 ve hastal\u0131klar\u0131 tedavi edebilmesi gerekiyor.&quot;<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Dahas\u0131, Chambon, kronik hastal\u0131klar\u0131 tedavi etmenin tek yolunun &quot;AAV tabanl\u0131 tedavilerde g\u00f6r\u00fclen ana engellerin&quot; \u00fcstesinden gelmek oldu\u011funu s\u00f6yl\u00fcyor. Bunlar aras\u0131nda, g\u00fcvenlik profilini iyile\u015ftirmek i\u00e7in daha az imm\u00fcnojenik ve m\u00fcmk\u00fcn oldu\u011funda yerel y\u00f6ntemler kullanmak yer al\u0131yor. Tekrar dozlanabilir ila\u00e7lar \u00fcretmek ve t\u0131bbi-ekonomik ve terap\u00f6tik rejim zorluklar\u0131 yaratan tek seferlik paradigmadan ka\u00e7\u0131nmak i\u00e7in daha iyi tolere edilebilirlik de \u00f6nemlidir.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"roche-abandons-the-gene-therapy-unit-and-pfizer-discontinues-beqvez\">Roche gen terapisi \u00fcnitesini terk ediyor ve Pfizer Beqvez&#039;i sonland\u0131r\u0131yor<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Daha yak\u0131n zamanda, Pfizer ve Roche, 2023&#039;te Takeda ve Biogen hari\u00e7 olmak \u00fczere AAV Ar-Ge&#039;sini terk etti. Hemofili B&#039;yi tedavi etmek i\u00e7in ABD G\u0131da ve \u0130la\u00e7 Dairesi (FDA) taraf\u0131ndan onaylanan AAV tabanl\u0131 bir gen terapisi olan Beqvez, bir y\u0131l \u00f6nce Pfizer taraf\u0131ndan durduruldu. \u015eubat ay\u0131nda, kanama durumu i\u00e7in gen terapisine ilginin eksikli\u011finden bahsedildi.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">B\u00fcy\u00fck \u0130svi\u00e7reli ila\u00e7 \u015firketi Roche, Philadelphia merkezli AAV odakl\u0131 gen terapisi b\u00f6l\u00fcm\u00fc Spark Therapeutics&#039;de \u00f6nemli bir yeniden yap\u0131lanmaya gitti. \u00c7al\u0131\u015fanlar\u0131n yar\u0131s\u0131ndan fazlas\u0131 i\u015ften \u00e7\u0131kar\u0131ld\u0131 ve Roche&#039;a yeniden entegre edilen 310 ki\u015fi kald\u0131. \u015eirket Mart ay\u0131nda Spark&#039;\u0131n etrafta olmas\u0131n\u0131n &quot;\u00f6nemli bir gelecek sinerjik faydas\u0131&quot; olmad\u0131\u011f\u0131n\u0131 a\u00e7\u0131klad\u0131\u011f\u0131ndan beri, bunun herhangi bir kayb\u0131 \u00f6nlemek i\u00e7in yap\u0131ld\u0131\u011f\u0131 d\u00fc\u015f\u00fcn\u00fcl\u00fcyor.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"aa-vs-are-constrained-by-manufacturing-costs-and-payload-size\">AAV&#039;ler \u00fcretim maliyetleri ve y\u00fck boyutuyla s\u0131n\u0131rl\u0131d\u0131r<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">Chambon&#039;a g\u00f6re, AAV&#039;lerin k\u00fc\u00e7\u00fck y\u00fck kapasitesi, azalan talebin nedenlerinden biridir. AAV&#039;ler asl\u0131nda di\u011fer viral ve viral olmayan vekt\u00f6rlerden \u00e7ok daha b\u00fcy\u00fck y\u00fckleri ta\u015f\u0131yabilir ve yakla\u015f\u0131k 4,7 Kb&#039;lik bir paketleme kapasitesine sahiptir. Di\u011ferleri birden fazla gene sahip olabilir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Chambon, DMD i\u00e7in gen terapisinde kullan\u0131lan distrofin proteininin yo\u011funla\u015ft\u0131r\u0131lm\u0131\u015f bir bi\u00e7imi olan etkili bir AAV mikrodistrofinin geli\u015ftirilmesinin y\u0131llar ald\u0131\u011f\u0131n\u0131 ve hala geli\u015ftirilmesi gerekebilece\u011fini belirtti. Chambon, &quot;Terap\u00f6tik DNA ifadesinin daha iyi kontrol edilmesi ve b\u00fcy\u00fck proteinlerin tam dizilerinin dahil edilmesi i\u00e7in y\u00fck boyutu \u00f6nemlidir&quot; dedi. Ayr\u0131ca, y\u00fck boyutu sorununu a\u015fmak i\u00e7in ikili ve \u00fc\u00e7l\u00fc AAV kombinasyonlar\u0131 deneniyor, ancak bunlar daha y\u00fcksek dozajlar gerektirdi\u011fi ve do\u011fas\u0131 gere\u011fi daha az verimli oldu\u011fu i\u00e7in cevap de\u011fil.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Ayr\u0131ca, Chambon, AAV vekt\u00f6rlerinin \u00fcretiminin karma\u015f\u0131kl\u0131\u011f\u0131 nedeniyle \u00f6l\u00e7eklendirmenin maliyetli oldu\u011funu a\u00e7\u0131klad\u0131. Bu nedenle, iyile\u015ftirilmi\u015f AAV&#039;ler ve belki de AAV d\u0131\u015f\u0131ndaki vekt\u00f6rler i\u00e7in arama uzun zamand\u0131r gecikti.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazla bilgi edin: <a href=\"https:\/\/dmdwarrior.com\/tr\/dmd-warriors-turkish-representative-shares-his-views-on-elevidys-gene-therapy-is-it-effective-and-why-is-it-expensive\/\">DMD WarrioR&#039;un T\u00fcrkiye Temsilcisi Elevidys Gen Terapisi Hakk\u0131ndaki G\u00f6r\u00fc\u015flerini Payla\u015f\u0131yor: Etkili mi ve Neden Pahal\u0131?<\/a><\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"beyond-aav-research-looking-for-more-effective-ways-to-deliver-drugs\">AAV ara\u015ft\u0131rmas\u0131n\u0131n \u00f6tesinde: \u0130la\u00e7lar\u0131 ula\u015ft\u0131rman\u0131n daha etkili yollar\u0131n\u0131 ar\u0131yoruz<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">\u00d6rne\u011fin, Vertex Pharmaceuticals&#039;nin ilgisizli\u011fine ra\u011fmen, Affinia Therapeutics AAV&#039;lerini daha yayg\u0131n olarak bilinir hale getirmeye kendini adam\u0131\u015ft\u0131r. &quot;Toksisite b\u00f6lgelerinden ka\u00e7\u0131n\u0131rken istenen etkinlik b\u00f6lgeleri olan dokular\u0131 ve h\u00fccreleri daha iyi hedeflemek&quot; i\u00e7in kapsidler tasarlanmaktad\u0131r. AAV9&#039;u yeniden tasarlamak i\u00e7in k\u0131sa peptitler AAV genetik koduna dahil edilir. Kapsidlerini di\u011fer biyoteknolojilere lisanslamay\u0131 d\u00fc\u015f\u00fcn\u00fcrken, y\u00fck kapasitesini art\u0131rmak i\u00e7in \u00fcniversite kurumlar\u0131 ve di\u011fer biyofarmas\u00f6tiklerle de \u00e7al\u0131\u015fmay\u0131 d\u00fc\u015f\u00fcnmektedir.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Bu arada, replikatif olmayan Herpes simpleks vir\u00fcs\u00fc-1 (HSV-1) vekt\u00f6rleri Chambon&#039;un EG 427 genetik ila\u00e7 hatt\u0131n\u0131n temelini olu\u015fturdu. Di\u011fer viral vekt\u00f6rlerle kar\u015f\u0131la\u015ft\u0131r\u0131ld\u0131\u011f\u0131nda, bunlar neredeyse 30 kb&#039;lik daha b\u00fcy\u00fck bir genetik y\u00fck ta\u015f\u0131yor. Ayr\u0131ca ba\u011f\u0131\u015f\u0131kl\u0131k tepkilerini bast\u0131rd\u0131klar\u0131 ke\u015ffedilerek \u015fimdiye kadar g\u00fcvenli bir profil s\u00fcrd\u00fcrd\u00fcler.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Chambon, &quot;Bu stratejinin daha \u00f6nce klinik g\u00fcvenli\u011fi g\u00f6sterilmi\u015f, yeniden dozajlamaya izin veren, \u00fcretimi ekonomik olarak \u00f6l\u00e7eklendirmeyi basitle\u015ftiren ve AAV&#039;den en az alt\u0131 kat daha b\u00fcy\u00fck bir y\u00fck kapasitesine sahip bir alternatif sundu\u011funu d\u00fc\u015f\u00fcn\u00fcyoruz. HSV tabanl\u0131 vekt\u00f6rlerin, \u00f6zellikle Krystal Bio olmak \u00fczere, di\u011fer i\u015fletmeler taraf\u0131ndan g\u00fcvenli bir \u015fekilde yeniden dozajlanabilece\u011fi bilimsel olarak g\u00f6sterilmi\u015ftir&quot; diye belirtti.<\/strong><\/p>\n\n\n\n<p class=\"wp-block-paragraph\">FDA Komiseri Marty Makary&#039;nin Nisan ay\u0131nda nadir hastal\u0131k ila\u00e7lar\u0131n\u0131n onaylar\u0131n\u0131 h\u0131zland\u0131rmak i\u00e7in \u00e7abalar duyurmas\u0131 g\u00f6z \u00f6n\u00fcne al\u0131nd\u0131\u011f\u0131nda (\u00e7o\u011fu zaten gen terapileri kullan\u0131yor), mevcut AAV&#039;lere alternatif aray\u0131\u015f\u0131 devam ederken bunun genel olarak gen terapisi ara\u015ft\u0131rmalar\u0131n\u0131 yava\u015flatmas\u0131 pek olas\u0131 de\u011fil. Ancak, ila\u00e7 da\u011f\u0131t\u0131m sekt\u00f6r\u00fcn\u00fcn \u00f6n\u00fcm\u00fczdeki y\u0131llarda nereye gitti\u011fini bekleyip g\u00f6rmemiz gerekecek.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">Daha fazlas\u0131n\u0131 ke\u015ffedin: <a href=\"https:\/\/dmdwarrior.com\/tr\/tag\/full-length-dystrophin\/\" target=\"_blank\" rel=\"noreferrer noopener\">Tam Boy Distrofin DMD Tedavisi \u0130\u00e7in M\u00fcmk\u00fcn Olabilir<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Adeno-associated virus (AAV) vectors were once heralded as the answer to delivering gene therapies, but in recent years, their prominence has diminished. It was first hailed as having the ability to treat several hereditary illnesses, but the decline in research has already been recognized. Vertex Pharmaceuticals is the most recent biopharma to reduce its investment [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":3843,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[282],"tags":[368,359,61],"class_list":["post-3833","post","type-post","status-publish","format-standard","has-post-thumbnail","category-editorials","tag-aav-based-gene-therapy","tag-full-length-dystrophin","tag-gene-therapy"],"subtitle":"AAV tabanl\u0131 Duchenne kas distrofisi (DMD) gen terapisine y\u00f6nelik ara\u015ft\u0131rmalar, y\u00fcksek maliyeti ve s\u0131n\u0131rl\u0131 etkinli\u011fi nedeniyle g\u00f6zden d\u00fc\u015fm\u00fc\u015f durumda. \u0130la\u00e7 \u015firketlerinin, daha d\u00fc\u015f\u00fck maliyetlerle tam uzunlukta distrofin \u00fcretmek i\u00e7in tRNA ve mRNA tabanl\u0131 ila\u00e7lar geli\u015ftirmeye odaklanmas\u0131 bekleniyor.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3833","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/comments?post=3833"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3833\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3843"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3833"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3833"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3833"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}