将DNA植入眼睛治疗视力丧失的疗法有望在美获批|ml米乐

本文摘要：A pioneering treatment that can reverse a form of childhood vision loss by inserting DNA directly into the eye is on track for regulatory approval in the US, after a panel of scientists endorsed unanimously the gene therapy being developed by Spark Therapeutics.一种将DNA必要植入眼睛以反败为胜某类儿童视力丧失的开创性疗法，未来将会获得美国监管机构的批准后。

A pioneering treatment that can reverse a form of childhood vision loss by inserting DNA directly into the eye is on track for regulatory approval in the US, after a panel of scientists endorsed unanimously the gene therapy being developed by Spark Therapeutics.一种将DNA必要植入眼睛以反败为胜某类儿童视力丧失的开创性疗法，未来将会获得美国监管机构的批准后。此前，一个由科学家构成的顾问委员会对这种由Spark Therapeutics公司研发的“基因疗法”回应了完全一致的反对。If the US Food and Drug Administration accepts the recommendation of its advisory committee, as it tends to, it would mark the agencys first approval of such a treatment and herald a new era of medicine where diseases are tackled by inserting functioning copies of genes that are missing or mutated.如果美国食品药品监督管理局(FDA)拒绝接受其顾问委员会的建议（往往不会拒绝接受），将标志着该机构首次批准后此类疗法，并伴随着一个新的药物时代的来临，即通过植入缺陷或变异基因的运转长时间的副本来化疗疾病。

Gene therapies seek to tackle illness at its biological source, opening up the possibility of drugs that can be delivered just once rather than on a regular basis.基因疗法谋求从疾病的生物源头化疗疾病，这使得生产出有只需投用一次（而不是要定期投用）的药物沦为有可能。In a reference to the potentially curative properties of such treatments, Sparks shares are listed on Nasdaq under the ticker ONCE.Spark在纳斯达克(Nasdaq)上市的股票的代码是ONCE，指代这种疗法有可能药到病除的特性。Such therapy also opens up the possibility of finding better treatment options for a string of genetically-driven illnesses that have been hard to tackle with traditional medicine, such as cystic fibrosis and Huntingtons disease.这种疗法还使得为一系列由基因造成的疾病寻找更佳的化疗自由选择沦为有可能，这类疾病很难依赖传统药物化疗，例如囊性纤维化和亨廷顿氏病。While representing a huge scientific advance, such one-off treatments are expected to be hugely expensive and threaten to put a strain on already cash-strapped healthcare systems that are structured to pay for chronic therapies.尽管这代表着极大的科学变革，但这种重复使用疗法的价格预计将极为高昂，可能会给本已资金紧张的医疗系统带给压力，这些医疗系统的设计想法是为慢性疗法买单。

Philadelphia-based Spark will not announce a price until after the drug has been approved but analysts and investors expect it to be in excess of $1m, which would make it one of the most expensive therapies of all time.总部坐落于费城的Spark要等到药物获批后才不会宣告价格，但分析师和投资者预计其价格将多达100万美元，令其其沦为有史以来最便宜的疗法之一。The therapy, known as Luxturna, is being developed for a subset of patients with a rare inherited disorder known as Leber congenital amaurosis, who number about 6,000 in the US, Europe and other markets that Spark hopes to enter. People with LCA suffer severe vision loss and are at high risk of going completely blind.这种取名为Luxturna的疗法，是为部分患上取名为“莱伯氏先天性黑蒙”(LCA)的少见遗传性疾病的患者研发的。在Spark期望转入的美国、欧洲和其他市场，这种患者的数量大约为6000人。患上这种疾病的人视力相当严重失去，有很高风险显得完全失聪。

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