将DNA植入眼睛治疗视力丧失的疗法有望在美获批

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直接植入眼睛以逆转某类儿童视力丧失的开创性疗法，有望得到美国监管机构的批准。此前，一个由科学家组成的顾问委员会对这种由Spark Therapeutics研发的“基因疗法”表示了一致的支持。

If the US Food and Drug Administration accepts the recommendation of its advisory committee, as it tends to, it would mark the agency’s 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)接受其顾问委员会的建议（往往会接受），将标志着该机构首次批准此类疗法，并预示着一个新药物时代的到来，即通过植入缺失或变异基因的运转正常的副本来治疗疾病。

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. In a reference to the potentially curative properties of such treatments, Spark’s shares are listed on Nasdaq under the ticker ONCE.

基因疗法寻求从疾病的生物源头治疗疾病，这使得生产出只需投用一次（而不是要定期投用）的药物成为可能。Spark在纳斯达克(Nasdaq)上市的股票的代码是ONCE，指代这种疗法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 Huntington’s disease.

这种疗法还使得为一系列由基因导致的疾病找到更好的治疗选择成为可能，这类疾病很难依靠传统药物治疗，例如囊性纤维化和亨廷顿氏病(Huntington’s disease)。

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.

尽管这代表着巨大的科学进步，但这种一次性疗法的价格预计将极其高昂，可能会给本已资金紧张的医疗系统带来压力，这些医疗系统的设计初衷是为慢性疗法买单。

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.

总部位于费城的Spark要等到药物获批后才会宣布价格，但分析师和投资者预计，其价格将超过100万美元，令其成为有史以来最昂贵的疗法之一。

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.

这种名为Luxturna的疗法，是为部分患有名为“莱伯氏先天性黑蒙”(LCA)的罕见遗传性疾病的患者研发的。在美国、欧洲和其他市场，这种患者的数量约为6000人，Spark希望进入这块市场。患有这种疾病的人视力严重丧失，有很高风险变得彻底失明。