biomarin-pharma

## 历史时间线 - 1997 Jean-Jacques Bienaime在加州创立 - 2001 首款药物Naglazyme获批治疗MPS VI - 2003 Kuvan获批治疗苯丙酮尿症 - 2014 与Sangamo合作开发血友病基因疗法 - 2019 FDA批准血友病A基因疗法VOXVOXA - 2022 Voxzogo获批治疗软骨发育不全

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Install skill "biomarin-pharma" with this command: npx skills add hanxueyuan/biomarin-pharma

历史时间线

  • 1997 Jean-Jacques Bienaime在加州创立
  • 2001 首款药物Naglazyme获批治疗MPS VI
  • 2003 Kuvan获批治疗苯丙酮尿症
  • 2014 与Sangamo合作开发血友病基因疗法
  • 2019 FDA批准血友病A基因疗法VOXVOXA
  • 2022 Voxzogo获批治疗软骨发育不全

商业模式

专注超罕见病,患者往往少于5000人。核心收入来自Naglazyme、Kuvan、Palynziq等酶替代和小分子药物。基因治疗是未来增长引擎。

护城河分析

罕见病领域25年积累,酶替代生产技术平台,FDA孤儿药独占期保护。管线中20+临床阶段候选药。

关键数据

2023年收入约22亿美元;7款获批药物;全球员工约4000人;市值约160亿美元

有趣事实

BioMarin是少数在基因疗法领域同时布局多个适应症的生物公司,AAV载体技术理论上可以换基因来治疗不同疾病。

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