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主题:PRO051/GSK2402968 近况

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PRO051/GSK2402968 近况  发贴心情 Post By:2011/10/13 21:58:25

PRO051/GSK2402968

In October 2009, Prosensa partnered with GSK for the development of its lead compound PRO051. 2009年10月, Prosensa与葛兰素史克合作开发的铅化合物PRO051。 Both parties are working closely together to make this drug available to patients.双方正紧密合作,使这种药物提供给患者。

Prosensa's lead compound PRO051/GSK2402968 is highly sequence-specific, ie no 100% full length hits elsewhere in the human genome, reducing the risk for off-target effects. Prosensa PRO051/GSK2402968的先导化合物是高度序列特异性,即没有100%的全长点击在人类基因组,减少脱靶效应的风险。 PRO051/GSK2402968 thus specifically induces exon 51 skipping in the DMD gene, which, given the frequencies in various international DMD mutation databases, could in principle correct the reading frame in ~13% of all DMD patients, including patients with deletions of exon 50, exon 52, exons 45-50, exons 48-50, and exons 49-50. PRO051/GSK2402968从而明确诱导外显子51中,在各种国际DMD基因突变数据库的频率,可以在原则上是正确的阅读框?13%的所有DMD患者,包括外显子50缺失患者的DMD基因,跳绳,外显子52,外显子,外显子48-50,45-50和49-50个外显子。

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In vitro studies in series of cultured patient cells affected by different relevant deletions demonstrated that PRO051/GSK2402968 induces exon 51 skipping independent of the type of mutation.在体外研究显示PRO051/GSK2402968诱导外显子51跳绳独立的突变类型不同有关删除受影响的细胞培养病人。 It was also successfully tested in the hDMD mouse model expressing full length human dystrophin.也有人试验成功hDMD鼠标模型表达全长人类的dystrophin。 Clinical proof of concept was obtained in four DMD patients receiving a single intramuscular 0.8 mg dose of PRO051/GSK2402968 [van Deutekom et al., 2007].临床证明的概念,得到四个DMD患者在接受单次肌注0.8毫克剂量的PRO051/GSK2402968 [面包车Deutekom等,2007]。 In this study PRO051/GSK2402968 was safe, well-tolerated, and effective in specifically inducing exon 51 skipping and dystrophin restoration (up to 35% of normal) in the majority of muscle fibers (up to 94%) in the treated area.在这项研究中,PRO051/GSK2402968专门诱导大部分肌纤维(高达94%)51外显子跳跃的dystrophin恢复正常(可达35%)在治疗部位是安全的,耐受性良好,和有效。

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In a subsequent Phase I/II dose-ranging safety study, PRO051/GSK2402968 was administered subcutaneously during 5 weeks in 12 patients at two European clinical centers.在随后的阶段I / II期剂量范围的安全性研究,PRO051/GSK2402968管理在5周12例皮下在两个欧洲的临床中心。 The study demonstrated that PRO051/GSK2402968 was well tolerated in all patients and that novel dystrophin expression was detected in each treated patient.该研究表明,PRO051/GSK2402968是很好的耐受性在所有患者在每个治疗的病人,新颖的抗肌萎缩蛋白表达检测。

An extension study is ongoing in all 12 patients in order to collect longer term safety data before enrolling patients for a large international multi-center pivotal study in 2010.扩展研究所有12名患者正在为了一个大型的国际多中心关键研究在2010年之前要收集招收患者更长的长期安全性数据。 Prosensa is also preparing for a safety and PK study in non-ambulatory DMD boys. Prosensa还准备为安全和在日间非DMD的男孩PK研究。 Prosensa has partnered with GSK for the further clinical development of PRO051/GSK2402968. Prosensa与葛兰素史克合作PRO051/GSK2402968临床的进一步发展。

PRO051/GSK2402968 has obtained an orphan drug designation in the EU and the US. PRO051/GSK2402968已取得孤儿药在欧盟和美国


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