Acceleration of rare disease therapeutic development: a case study of AGIL-AADC.

Sonya Das, Samuel Huang, Andrew W Lo

Journal: Drug discovery today 2019;24(3):678-684

PMID: 30610920

Full Text on Elsevier Science View on PubMed

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.

Address: MIT Laboratory for Financial Engineering, Sloan School of Management, Cambridge, MA, USA; MIT Department of Mathematics, Cambridge, MA, USA; MIT Department of Electrical Engineering and Computer Science, Cambridge, MA, USA.; MIT Laboratory for Financial Engineering, Sloan School of Management, Cambridge, MA, USA; MIT Department of Chemical Engineering, Cambridge, MA, USA.; MIT Laboratory for Financial Engineering, Sloan School of Management, Cambridge, MA, USA; MIT Computer Science and Artificial Intelligence Laboratory, Cambridge, MA, USA; Santa Fe Institute, Santa Fe, NM, USA. Electronic address: [email protected].

DOI: 10.1016/j.drudis.2018.12.006

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MeSH Terms: Amino Acid Metabolism, Inborn Errors, Aromatic-L-Amino-Acid Decarboxylases, Biotechnology, Genetic Therapy, Humans, Interinstitutional Relations, National Center for Advancing Translational Sciences (U.S.), Rare Diseases, Universities