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タイトル
和文: 
英文:The gap between development and manufacturing in gene therapy: Strategic options for overcoming traps 
著者
和文: 西田優, 児玉 耕太, 仙石愼太郎.  
英文: Yu Nishida, Kota Kodama, Shintaro Sengoku.  
言語 English 
掲載誌/書名
和文: 
英文:Drug Discovery Today 
巻, 号, ページ Volume 28    Issue 2    103429
出版年月 2022年11月2日 
出版者
和文: 
英文:Elvevier BV 
会議名称
和文: 
英文: 
開催地
和文: 
英文: 
DOI https://doi.org/10.1016/j.drudis.2022.103429
アブストラクト Gene therapy has been one of the most promising therapeutic approaches in recent years. This study analyzed a research and development (R&D) system for adeno-associated virus (AAV)-based gene therapies, and confirmed that there was a gap between the development and manufacturing capabilities. Although a start-up company that has no academic or manufacturing facilities can begin the clinical development process, it cannot successfully continue development activities without forming alliances and capital investment or, at a certain stage, without appropriate manufacturing and marketing strategies. We reviewed a series of case studies to categorize the acquisition patterns of pharmaceutical companies that are engaged in AAV gene therapy. These results provide insights into the R&D structures for AAV gene therapies from a technological management perspective.

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