Selecta Biosciences, Partner Get FDA Rare-Pediatric Designation for MMA-101
Selecta Biosciences, Partner Get FDA Rare-Pediatric Designation for MMA-101
DJ Selecta Biosciences, Partner Get FDA Rare-Pediatric Designation for MMA-101
DJ Selecta Biosciences，合作伙伴获得FDA稀有儿科MMA-101称号
By Colin Kellaher
科林·凯莱赫(Colin Kellaher)著
Selecta Biosciences Inc. and Asklepios BioPharmaceutical Inc. on Tuesday said the U.S. Food and Drug Administration granted rare-pediatric-disease designation to MMA-101, the gene therapy they are developing for a rare metabolic disorder.
Selecta Biosciences Inc.和Asklepios生物制药公司周二说，美国食品和药物管理局(FDA)批准MMA-101为罕见儿科疾病，这是他们正在开发的一种针对一种罕见代谢疾病的基因疗法。
Selecta, a Watertown, Mass., clinical-stage biotechnology company, and privately held Asklepios said the designation covers MMA-101 for the treatment of isolated methylmalonic acidemia due to methylmalonyl-CoA mutase gene mutations.
位于马萨诸塞州沃特敦的临床期生物技术公司Selecta和私人持股的Asklepios公司表示，该名称涵盖了MMA-101，用于治疗由于甲基丙二酰辅酶A变位酶基因突变而导致的孤立的甲基丙二酸血症。
The companies said they expect to initiate a phase 1 clinical trial of MMA-101 with Selecta's ImmTOR immune tolerance platform in the first half of 2021.
两家公司表示，他们预计将在2021年上半年利用Selecta的ImmTOR免疫耐受平台启动MMA-101的1期临床试验。
The FDA awards priority-review vouchers to drugmakers upon approval of drugs that are granted the rare-pediatric-disease designation. Those vouchers can be used to obtain priority review for another drug or sold to other companies.
FDA在批准被授予罕见儿科疾病称号的药物时，将优先审查凭证授予制药商。这些代金券可以用来获得另一种药物的优先审查，或者出售给其他公司。
Write to Colin Kellaher at colin.kellaher@wsj.com
写信给Colin Kellaher，电子邮件：colin.kellaher@wsj.com
(END) Dow Jones Newswires
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October 20, 2020 08:55 ET (12:55 GMT)
2020年10月20日东部时间08：55(格林尼治标准时间12：55)
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