一文看懂基因编辑和基因疗法七大最新进展

1、缓解率达97%!血友病基因疗法效果显著

Spark Therapeutics宣布,12名甲型血友病患者在接受基因疗法SPK-8011治疗后,缓解率达到97%。Spark研发的SPK-8011是一种使用Spark200衣壳且含有密码子优化的人源因子VIII基因的新型生物工程腺相关病毒(AAV)载体。它可以更有效地转导人肝细胞,并表达密码子优化的B结构域缺失的因子VIII基因,内源性产生因子VIII,用于治疗甲型血友病。Spark总裁兼研发负责人Katherine High博士说:“这些早期数据进一步支持了因子活性水平超过12%对患者预后造成的巨大影响,使我们更接近消除自发性出血的目标,使甲型血友病患者不再需要定期输注。”基于到目前为止的总体结果,Spark打算在今年第四季度启动3期试运行研究。在试运行研究完成后,3期参与者将接受2×10^12 vg/kg的SPK-8011。

2、黄斑变性基因疗法效果显著,有望摆脱定期注射

Regenxbio公司公布了基因疗法RGX-314颇具前景的1期临床试验中期数据,该疗法用于治疗湿性年龄相关性黄斑变性(wAMD)。AMD可用的疗法包括靶向VEGF(血管内皮生长因子)的眼内注射药物(例如再生元的Eylea和罗氏的Avastin和Lucentis)。这些药物可防止眼内渗漏血管的生长,但定期进行眼内注射会对患者的生活造成困扰。

Regenxbio带来的一次性基因疗法RGX-314有望为AMD患者带来全新的治疗体验。RGX-314使用腺相关病毒来提供编码可以中和VEGF活性的蛋白质的基因,旨在阻止因在眼睛中形成新血管而引起的视力丧失,从而减少抗VEGF眼内注射药物的需求。研究结果发现,基因疗法RGX-314在所有剂量下都是安全且耐受良好的,所有三组的蛋白质表达水平均具有剂量依赖性,抗VEGF药物的注射也呈剂量依赖性减少。

3、罗氏反义核酸疗法获EMA优先药品资格

罗氏(Roche)宣布,欧洲药品管理局(EMA)授予其在研药物RG6042(曾用名IONIS-HTTRx)优先药品(PRIME)资格,用于治疗亨廷顿病(HD)。在一项1/2a期研究中,RG6042展示了减少致病性突变亨廷顿蛋白(mHTT)的能力。与FDA突破性疗法认定类似,EMA的优先药品资格将加速潜力药物审批,使其能尽快用于患者。

RG6042是第二代修饰反义寡核苷酸(ASO,antisense oligonucleotide),通过靶向人HTT mRNA来降低mHTT蛋白的合成和表达水平。RG6042是罗氏和Ionis之间全面深入的药物筛选计划的成果,该计划专注于优化靶向人HTT mRNA的ASO的有效性,特异性和耐受性。来自RG6042的初步数据第一次展示了成人HD患者的mHTT水平可以被降低。按计划,罗氏将开展关键3期临床试验,旨在评估在较大患者群体中,RG6042的安全性和减缓成人HD的进展的有效性。

4、基因编辑新锐克睿基因获1700万美元A轮融资

专注于开发CRISPR基因编辑技术平台的公司——苏州克睿基因生物科技有限公司宣布完成1700万美元A轮融资,用于公司技术平台完善、生产研发中心建设和推进新药项目IND申报,以及产品线的扩充。本轮由启明创投领投,清松资本、盛鼎投资共同投资。

克睿基因成立于2016年,位于苏州生物医药产业园(BioBAY),由CRISPR基因编辑技术应用的先驱者联合创办。公司专注于新型医药、分子诊断领域中CRISPR技术原创性应用的开发,致力于解决难治愈性肿瘤、复杂遗传性疾病的临床需求。目前,克睿基因已经搭建了全球顶尖的基因编辑技术平台及基因递送平台。本轮融资后,克睿基因将进一步完善技术平台和生产研发中心建设,推进新药项目IND申报,并持续扩充产品管线。

5、启函生物完成780万美元A轮融资,致力解决异种器官移植

据报道,美国eGenesis姐妹公司杭州启函生物科技有限公司(“启函生物”)已于近日完成780万美元A轮融资。本轮投资由红杉资本中国基金领投,Arch Venture、北极光创投、 树兰医疗资本、Biomatics Capital Partners、Alta Partner等参与融资。接下来,启函生物将加快建立其基因修改平台,以推动异种器官移植的研发。

据悉,启函生物由哈佛大学异种器官移植课题带头人杨璐菡博士,以及美国科学院和工程院双院院士、哈佛大学知名遗传学家George Church 教授共同创建,是一家将最新的基因编辑技术来获取可用于人体移植的安全有效的异种细胞、组织和器官,并用于异种器官移植的创新型生物科技公司。

6、基因疗法公司A轮融资5000万美元,开发听力损失药物

专注于基因疗法的初创公司Akouos宣布完成5000万美元的A轮融资,以推动其首个在研项目进入人体临床试验,并加速开发多条研发管线,通过已经过验证的机制和基因靶标来治疗感觉神经性听力丧失。此轮融资由5AM Ventures和New Enterprise Associates共同领投。Akouos公司正在针对单基因引起的感觉神经性听力损失来开发创新疗法。Akouos成立于2016年,通过其开发的基于腺相关病毒载体(AAV)的基因治疗平台,致力于开发感觉神经性听力损失的治疗药物。

7、CRISPR基因编辑公司A轮融资2300万美元,利用智能手机检测疾病

Mammoth Biosciences近日宣布完成了2300万美元的A轮融资,该轮融资由Mayfield领投,NFX和8VC参与投资。此外,苹果公司首席执行官Tim Cook先生和从事癌症早期筛查的GRAIL公司首席执行官Jeff Huber先生也参与了此次融资。值得注意的是,基因编辑专家Jennifer Doudna博士是这家公司的创始人之一。据悉,Mammoth将利用这笔融资资金进行平台的开发、人才招聘和知识产权组合的扩充。

Mammoth又被称为“Google of CRISPR”,专注于将CRISPR作为搜索基因序列的方法,而不是基因编辑能力本身。Mammoth采用的CRISPR专利技术旨在通过简单且经济实惠的即时检测。首款产品旨在通过信用卡大小的即时检测设备来实现疾病检测的大众化,以便快速、轻松、经济地检测多种疾病,且同时可在医院和家中使用。这个过程只需要30分钟的时间和一台智能手机。

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