Abstract：
:Rare-disease drug development is both scientifically and commercially challenging. This case study highlights Agilis Biotherapeutics (Agilis), a small private biotechnology company that has developed the most clinically advanced adeno-associated virus (AAV) gene therapy for the brain. In an international collaboration led by Agilis with National Taiwan University (NTU) Hospital and the Therapeutics for Rare and Neglected Diseases (TRND) program of the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health, Agilis' gene therapy for aromatic l-amino acid decarboxylase deficiency (AADC), AGIL-AADC, was granted biologics license application (BLA)-ready status by the FDA in 2018, only 18 months after being licensed from NTU by Agilis. Here, we highlight the factors that enabled this remarkable pace of successful drug development for an ultra-rare disease.
journal_name
Drug Discov Todayjournal_title
Drug discovery todayauthors
Das S,Huang S,Lo AWdoi
10.1016/j.drudis.2018.12.006subject
Has Abstractpub_date
2019-03-01 00:00:00pages
678-684issue
3eissn
1359-6446issn
1878-5832pii
S1359-6446(18)30340-4journal_volume
24pub_type
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
doi：10.1016/s1359-6446(03)02826-5
更新日期：2003-09-15 00:00:00
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
doi：10.1016/j.drudis.2016.02.010
更新日期：2016-06-01 00:00:00
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
doi：10.1016/s1359-6446(03)02623-0
更新日期：2003-03-15 00:00:00
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
doi：10.1016/j.drudis.2018.03.013
更新日期：2018-08-01 00:00:00
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
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更新日期：2018-05-01 00:00:00
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
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journal_title：Drug discovery today
pub_type： 杂志文章,评审
doi：10.1016/j.drudis.2018.08.009
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