加拿大细胞和基因疗法的商业化:当前的局面,挑战和机遇
Logan S Germain1, Louise M Winn2
1Department of Biomedical and Molecular Sciences, Queen's University, Kingston, Ontario K7L 3N6, Canada.
Drug discovery today
|August 15, 2025
概括
加拿大加拿大加拿大加拿大加拿大加拿大
科学领域:
- 生物技术和制药科学 生物技术和制药科学
- 卫生政策和管理卫生政策和管理
背景情况:
- 细胞和基因疗法 (CGTs) 在治疗遗传疾病和癌症方面取得了重大进展.
- 全球对CGT的批准正在增加,但与美国和欧洲相比,加拿大在商业化方面面临挑战.
研究的目的:
- 确定加拿大CGT商业化的主要障碍.
- 提出政策解决方案,以提升加拿大的CGT创新,可负担性和可访问性,借鉴国际实例.
主要方法:
- 进行了以政策为导向的环境扫描.
- 数据来源包括监管文件,同行评审文献,政府报告和行业出版物.
主要成果:
- 在四个关键领域确定了障碍:监管,制造,定价和补偿,以及获取和公平.
- 尽管最近投资面临挑战,但加拿大批准基于CRISPR的疗法和CAR T细胞扩张表明增长潜力.
结论:
- 战略政策改革对于克服已识别的障碍至关重要.
- 这些改革可以促进国内的CGT创新,提高负担能力,并确保加拿大人获得公平的机会.
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