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某類兒童視力喪失的開創性療法，有望得到美國監管機構的批准。此前，一個由科學家組成的顧問委員會對這種由Spark Therapeutics公司研發的“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.
如果美國食品藥品監督管理局(FDA)接受其顧問委員會的建議（往往會接受），將標誌着該機構首次批准此類療法，並預示着一個新藥物時代的到來，即通過植入缺失或變異基因的運轉正常的副本來治療疾病。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.
基因療法尋求從疾病的生物源頭治療疾病，這使得生產出只需投用一次（而不是要定期投用）的藥物成爲可能。
In a reference to the potentially curative properties of such treatments, Spark's shares are listed on Nasdaq under the ticker ONCE.
Spark在納斯達克(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.
這種療法還使得爲一系列由基因導致的疾病找到更好的治療選擇成爲可能，這類疾病很難依靠傳統藥物治療，例如囊性纖維化和亨廷頓氏病。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.
儘管這代表着巨大的科學進步，但這種一次性療法的價格預計將極其高昂，可能會給本已資金緊張的醫療系統帶來壓力，這些醫療系統的設計初衷是爲慢性療法買單。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.
總部位於費城的Spark要等到藥物獲批後纔會宣佈價格，但分析師和投資者預計其價格將超過100萬美元，令其成爲有史以來最昂貴的療法之一。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.
這種名爲Luxturna的療法，是爲部分患有名爲“萊伯氏先天性黑蒙”(LCA)的罕見遺傳性疾病的患者研發的。在Spark希望進入的美國、歐洲和其他市場，這種患者的數量約爲6000人。患有這種疾病的人視力嚴重喪失，有很高風險變得徹底失明。
