Son teknoloji gen terapileri geli\u015ftirmeye odaklanan yenilik\u00e7i bir biyoteknoloji \u015firketi olan Belief BioMed (BBM), bug\u00fcn \u015firketin Duchenne Musk\u00fcler Distrofi (DMD) gen terapisi aday\u0131 olan BBM-D101 i\u00e7in bir Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 (IND) ba\u015fvurusu i\u00e7in Ulusal T\u0131bbi \u00dcr\u00fcnler \u0130daresi'nden (NMPA) onay ald\u0131\u011f\u0131n\u0131 duyurdu. \u015eimdiye kadar, BBM-D101 i\u00e7in IND hem NMPA hem de Amerika G\u0131da ve \u0130la\u00e7 \u0130daresi (FDA) taraf\u0131ndan onayland\u0131.<\/p>\n\n\n\n
Belief BioMed'nin Kurucu Orta\u011f\u0131, Ba\u015fkan\u0131 ve Ba\u015f Bilim Sorumlusu Dr. Xiao Xiao, "NMPA'n\u0131n BBM-D101 i\u00e7in IND onay\u0131 almas\u0131yla ilgili g\u00fczel haberi sizlerle payla\u015fmaktan mutluluk duyuyoruz. NMPA'n\u0131n IND incelemesini takdir ediyoruz ve klinik denemeyi y\u00fcr\u00fctmek i\u00e7in ajansla i\u015f birli\u011fi i\u00e7inde \u00e7al\u0131\u015fmaya devam etmeyi d\u00f6rt g\u00f6zle bekliyoruz. Daha \u00f6nce FDA taraf\u0131ndan IND onay\u0131 al\u0131nm\u0131\u015ft\u0131. DMD hastalar\u0131na m\u00fcmk\u00fcn olan en k\u0131sa s\u00fcrede yeni tedavi se\u00e7enekleri sunmak i\u00e7in klinik ara\u015ft\u0131rmalar\u0131n ilerlemesini h\u0131zland\u0131raca\u011f\u0131z." dedi.<\/p>\n\n\n\n
Bu klinik \u00e7al\u0131\u015fma, 4-9 ya\u015f aras\u0131 DMD hastalar\u0131nda tek bir intraven\u00f6z inf\u00fczyonun g\u00fcvenli\u011fini ve etkinli\u011fini de\u011ferlendirmek i\u00e7in tasarlanm\u0131\u015f a\u00e7\u0131k etiketli ve tek dozlu bir klinik \u00e7al\u0131\u015fmad\u0131r. Tasarlanm\u0131\u015f kapsid nedeniyle, BBM-D101 kas dokusunu d\u00f6n\u00fc\u015ft\u00fcrmek i\u00e7in daha y\u00fcksek bir verimlili\u011fe sahiptir. Bu nedenle, \u00f6nerilen klinik dozaj, onaylanm\u0131\u015f benzer adeno-ili\u015fkili vir\u00fcs (AAV) gen terapisi \u00fcr\u00fcnlerinden daha d\u00fc\u015f\u00fckt\u00fcr ve BBM-D101'in iyi bir g\u00fcvenli\u011fe sahip olmas\u0131 beklenmektedir.<\/p>\n\n\n\n
BBM-D101, BBM'nin ba\u011f\u0131ms\u0131z fikri m\u00fclkiyet haklar\u0131na sahip AAV tabanl\u0131 bir gen terapisidir. Tasarlanm\u0131\u015f AAV vekt\u00f6r\u00fcne dayanarak, optimize edilmi\u015f geni t\u00fcm v\u00fccudun kaslar\u0131na iletmek i\u00e7in tek bir intraven\u00f6z olarak uygulan\u0131r ve b\u00f6ylece DMD'nin "tek doz, uzun vadeli etki" tedavisi elde edilir.<\/p>\n\n\n\n
Daha fazla bilgi edin:<\/strong> Duchenne Musk\u00fcler Distrofisi \u0130\u00e7in Yeni Gen Terapileri<\/a><\/p>\n\n\n\n Ara\u015ft\u0131rma Ama\u00e7l\u0131 Yeni \u0130la\u00e7 Ba\u015fvurusu (IND), G\u0131da ve \u0130la\u00e7 Dairesi'nden (FDA) insanlara ara\u015ft\u0131rma ama\u00e7l\u0131 bir ila\u00e7 veya biyolojik \u00fcr\u00fcn uygulanmas\u0131 i\u00e7in yetki talebidir. (Daha fazlas\u0131n\u0131 okuyun: IND \u0130zninin Anlam\u0131<\/a>)<\/p>","protected":false},"excerpt":{"rendered":" Belief BioMed (BBM), an innovative biotechnology company focused on developing cutting-edge gene therapies, today announced that it has received National Medical Products Administration (NMPA) clearance of an Investigational New Drug (IND) application for BBM-D101, the company\u2019s Duchenne Muscular Dystrophy (DMD) gene therapy candidate. Thus far, the IND for BBM-D101 has been approved by both NMPA […]<\/p>\n","protected":false},"author":1,"featured_media":3710,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[281],"tags":[199,197,123,61,354],"class_list":["post-3708","post","type-post","status-publish","format-standard","has-post-thumbnail","category-press-releases","tag-bbm-d101","tag-belief-biomed","tag-china","tag-gene-therapy","tag-nmpa"],"subtitle":"Belief BioMed'nin BBM-D101 gen terapisine Ulusal T\u0131bbi \u00dcr\u00fcnler \u0130daresi (NMPA) taraf\u0131ndan IND izni verildi.","_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3708","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=3708"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/posts\/3708\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media\/3710"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/media?parent=3708"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/categories?post=3708"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/tr\/wp-json\/wp\/v2\/tags?post=3708"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}IND \u0130zni Ne Demektir?<\/h2>\n\n\n\n