{"title":"夏尔与Nimbus Discovery合作开发溶酶体贮积症的口服疗法","authors":"Heather Cartwright","doi":"10.3833/PDR.V2013I5.1941","DOIUrl":null,"url":null,"abstract":"In the first deal to emerge from its tie-up with Atlas Venture, Shire has partnered with Nimbus Discovery to develop disease-altering small molecule therapies for lysosomal storage disorders (LSDs). Using its computational chemistry platform, Nimbus will profile molecules against a single disease target and will deliver a drug candidate ready to enter late preclinical studies. At this point Shire will have an exclusive option to acquire the programme. Shire already markets a portfolio of intravenously administered enzyme replacement therapies for LSDs, including Replagal® (agalsidase alpha) for Fabry disease, Vpriv® (velaglucerase alpha) for Gaucher disease and Elaprase® (idursulphase) for Hunter syndrome.","PeriodicalId":19951,"journal":{"name":"Pharmadeals Review","volume":"30 1","pages":""},"PeriodicalIF":0.0000,"publicationDate":"2013-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":"{\"title\":\"Shire Partners with Nimbus Discovery to Develop Oral Therapies for Lysosomal Storage Disorders\",\"authors\":\"Heather Cartwright\",\"doi\":\"10.3833/PDR.V2013I5.1941\",\"DOIUrl\":null,\"url\":null,\"abstract\":\"In the first deal to emerge from its tie-up with Atlas Venture, Shire has partnered with Nimbus Discovery to develop disease-altering small molecule therapies for lysosomal storage disorders (LSDs). Using its computational chemistry platform, Nimbus will profile molecules against a single disease target and will deliver a drug candidate ready to enter late preclinical studies. At this point Shire will have an exclusive option to acquire the programme. Shire already markets a portfolio of intravenously administered enzyme replacement therapies for LSDs, including Replagal® (agalsidase alpha) for Fabry disease, Vpriv® (velaglucerase alpha) for Gaucher disease and Elaprase® (idursulphase) for Hunter syndrome.\",\"PeriodicalId\":19951,\"journal\":{\"name\":\"Pharmadeals Review\",\"volume\":\"30 1\",\"pages\":\"\"},\"PeriodicalIF\":0.0000,\"publicationDate\":\"2013-05-20\",\"publicationTypes\":\"Journal Article\",\"fieldsOfStudy\":null,\"isOpenAccess\":false,\"openAccessPdf\":\"\",\"citationCount\":\"0\",\"resultStr\":null,\"platform\":\"Semanticscholar\",\"paperid\":null,\"PeriodicalName\":\"Pharmadeals Review\",\"FirstCategoryId\":\"1085\",\"ListUrlMain\":\"https://doi.org/10.3833/PDR.V2013I5.1941\",\"RegionNum\":0,\"RegionCategory\":null,\"ArticlePicture\":[],\"TitleCN\":null,\"AbstractTextCN\":null,\"PMCID\":null,\"EPubDate\":\"\",\"PubModel\":\"\",\"JCR\":\"\",\"JCRName\":\"\",\"Score\":null,\"Total\":0}","platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pharmadeals Review","FirstCategoryId":"1085","ListUrlMain":"https://doi.org/10.3833/PDR.V2013I5.1941","RegionNum":0,"RegionCategory":null,"ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"","JCRName":"","Score":null,"Total":0}
Shire Partners with Nimbus Discovery to Develop Oral Therapies for Lysosomal Storage Disorders
In the first deal to emerge from its tie-up with Atlas Venture, Shire has partnered with Nimbus Discovery to develop disease-altering small molecule therapies for lysosomal storage disorders (LSDs). Using its computational chemistry platform, Nimbus will profile molecules against a single disease target and will deliver a drug candidate ready to enter late preclinical studies. At this point Shire will have an exclusive option to acquire the programme. Shire already markets a portfolio of intravenously administered enzyme replacement therapies for LSDs, including Replagal® (agalsidase alpha) for Fabry disease, Vpriv® (velaglucerase alpha) for Gaucher disease and Elaprase® (idursulphase) for Hunter syndrome.