將DNA植入眼睛治療視力喪失的療法有望在美獲批

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.

一種將DNA直接植入眼睛以逆轉(zhuǎn)某類兒童視力喪失的開創(chuàng)性療法，有望得到美國(guó)監(jiān)管機(jī)構(gòu)的批準(zhǔn)。此前，一個(gè)由科學(xué)家組成的顧問委員會(huì)對(duì)這種由Spark Therapeutics研發(fā)的“基因療法”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.

如果美國(guó)食品藥品監(jiān)督管理局(FDA)接受其顧問委員會(huì)的建議(往往會(huì)接受)，將標(biāo)志著該機(jī)構(gòu)首次批準(zhǔn)此類療法，并預(yù)示著一個(gè)新藥物時(shí)代的到來(lái)，即通過(guò)植入缺失或變異基因的運(yùn)轉(zhuǎn)正常的副本來(lái)治療疾病。

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.

基因療法尋求從疾病的生物源頭治療疾病，這使得生產(chǎn)出只需投用一次(而不是要定期投用)的藥物成為可能。Spark在納斯達(dá)克(Nasdaq)上市的股票的代碼是ONCE，指代這種療法可能藥到病除的特性。

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.

這種療法還使得為一系列由基因?qū)е碌募膊≌业礁玫闹委熯x擇成為可能，這類疾病很難依靠傳統(tǒng)藥物治療，例如囊性纖維化和亨廷頓氏病(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.

盡管這代表著巨大的科學(xué)進(jìn)步，但這種一次性療法的價(jià)格預(yù)計(jì)將極其高昂，可能會(huì)給本已資金緊張的醫(yī)療系統(tǒng)帶來(lái)壓力，這些醫(yī)療系統(tǒng)的設(shè)計(jì)初衷是為慢性療法買單。

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.

總部位于費(fèi)城的Spark要等到藥物獲批后才會(huì)宣布價(jià)格，但分析師和投資者預(yù)計(jì)，其價(jià)格將超過(guò)100萬(wàn)美元，令其成為有史以來(lái)最昂貴的療法之一。

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.

這種名為L(zhǎng)uxturna的療法，是為部分患有名為“萊伯氏先天性黑蒙”(LCA)的罕見遺傳性疾病的患者研發(fā)的。在美國(guó)、歐洲和其他市場(chǎng)，這種患者的數(shù)量約為6000人，Spark希望進(jìn)入這塊市場(chǎng)?；加羞@種疾病的人視力嚴(yán)重喪失，有很高風(fēng)險(xiǎn)變得徹底失明。