一文看懂基因编辑和基因疗法四大最新进展

1. Amicus公司1亿美元收获10款在研基因疗法

9月20日,Amicus Therapeutics公司宣布,通过收购Celenex公司,它从全国儿童医院(Nationwide Children’s Hospital)和俄亥俄州立大学(Ohio State University)获得10个基因疗法项目的全球研发许可。其中治疗CLN6,CLN3,和CLN8 Batten病(Batten disease)的主打研究项目可能成为治疗这些严重罕见病的治愈性疗法。

Amicus公司收获的10项基因疗法都是靶向溶酶体贮积症(LSD)的研究项目。它们通过使用AAV载体将功能正常的基因通过鞘内注射(intrathecal injection)运送到中枢神经系统内部,希望能够达到一次治疗就能长期,甚至终身缓解患者症状的效果。这10个研究项目中,治疗因为CLN6和CLN3的基因突变导致的Batten病的基因疗法已经进入临床阶段,其中治疗CLN6 Batten病的基因疗法已经获得了积极的初步临床结果。根据合作协议,Amicus公司将付出1亿美元的先期付款。根据研发和监管里程碑,Celenex可能获得高达2.77亿美元的后续付款。

2. 针对杜氏肌营养不良症,Sarepta基因疗法临床试验重获FDA放行

9月24日,Sarepta Therapeutics公司宣布FDA解除对该公司的杜氏肌营养不良症(DMD)微型抗肌萎缩蛋白基因疗法项目的临床试验禁令。Sarepta此前于2018年7月25日宣布,由于第三方提供的质粒中存在痕量DNA片段,FDA叫停该项目的临床实验。作为回应,其与全国儿童医院(Nationwide Children’s Hospital)合作向FDA递交了行动计划,将对质粒供应商进行审查并且承诺使用GMP水平的质粒生产以后的产品。在FDA禁令解除后,Sarepta公司预计将在2018年底启动使用微抗肌萎缩蛋白基因疗法治疗DMD患者的临床试验。

3. 提高婴幼儿运动能力,基因泰克SMA新药中期结果优良

10月3日,基因泰克(Genentech)公司宣布了其针对脊髓性肌萎缩症(SMA)的在研药物risdiplam(RG7916)的两项关键临床试验FIREFISH和SUNFISH的积极中期数据。RG7916是一款在研的口服SMA疗法,有望直接靶向致病原因,通过调节SMN2基因的剪接,进而产生更多完整的SMN mRNA,增加神经系统、肌肉及其他组织中的SMN蛋白水平。

在针对1型SMA的试验FIREFISH中,参与试验的14名婴儿患者中6名(43%)可以在有支持或无支持的情况下顺利坐起,更有3名(21%)在接受治疗8个月后可以无需协助地持续坐住。此外,试验结束后,4名婴儿患者(29%)可以翻身侧躺,7名(50%)可以踢腿,6名(43%)可以控制头部保持直立。

在SUNFISH试验第一部分中,接受治疗12个月后,患者血液中的SMN蛋白中值增加了两倍以上。在接受治疗至少一年后(n=30),作为主要终点的运动功能评估量表(MFM)分数对比基线的中值变化为3.1。63%的患者在一年后获得了对比基线上升了大于等于3的MFM分数。值得一提的是,70%的患者的MFM分数都获得了增加。

4. 治疗地中海贫血症,基因疗法获欧盟优先药品资格

10月5日,Orchard Therapeutics宣布其在研自体离体慢病毒基因疗法OTL-300获得了欧洲医药管理局(EMA)颁发的优先药品(PRIME)资格。OTL-300用于治疗输血依赖性β-地中海贫血症(TDBT)。Orchard的自体离体基因疗法是,通过造血干细胞移植的程序,将经过遗传修饰的拥有正常HBB基因拷贝的干细胞输回患者体内,从而生长并分裂成新的细胞,产生更高水平的血红蛋白。

此次PRIME资格的颁发基于OTL-300的临床前和早期临床项目数据。在7名接受了至少的12月后续追踪的TDBT患者中,5名患者显著降低了输血频率和输血量。4名儿童患者之中的3名,实现了治疗一个月后无需输血,而一名成人患者到达了9个月无需输血。此外,OTL-300展示了良好的耐受性和安全性。

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