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一种药物,多种罕见病:让罕见病药物研发更具盈利能力的变革探秘One drug, many rare diseases. Inside the movement to make rare drug development more profitable

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CNBC咨询委员会成员安娜·格雷卡(Anna Greka)博士的职业生涯一直建立在寻找不同罕见病之间联系的基础上。作为哈佛大学教授、麻省总医院布莱根医疗系统的医师,格雷卡也是博德研究所的成员。在她的实验室里,她负责监督“通往治愈加速器”(Ladders to Cures Accelerator)项目,该倡议旨在加速罕见病患者治疗方法的开发。她希望这种模式能够吸引更多投资进入这一领域。

CNBC Advisory Board member Dr. Anna Greka has built her career on finding connections between different rare diseases. Greka, a Harvard professor and physician at Mass General Brigham, is also a member of the Broad Institute. At her lab, she oversees the Ladders to Cures Accelerator, an initiative designed to speed up the development of treatments for rare disease patients. It's a model she hopes is able to attract more investment to the space.

格雷卡和她的团队正致力于识别不同罕见病汇聚并共享单一生物学机制的地方。格雷卡将这种方法称为“节点生物学”(nodal biology)。通过识别罕见病重叠的领域,“通往治愈加速器”希望开发出可用于治疗多种病症的新型药物。通过开发能够治疗多种疾病的单一药物,这些药物的目标患者群体将变得更加庞大。这可以使药物更具盈利能力,格雷卡认为这是建立经济上可持续的罕见病药物研发模式的关键。

Greka and her team are working on identifying where different rare diseases converge and share a single biological mechanism. Greka calls this approach "nodal biology". By identifying areas where rare diseases overlap, Ladders to Cures hopes to develop new types of drugs that can be used to treat multiple conditions. By developing single drugs capable of treating multiple diseases, the addressable patient population of those drugs becomes much bigger. That can make a drug more profitable, which Greka says is key to building a rare drug development model that is economically sustainable.

格雷卡已经证明了节点方法的可行性。2019年,她和团队发表了一篇论文,概述了他们为一种罕见肾病发现的机制,该机制也在影响眼睛和大脑的疾病中发挥作用。通过“通往治愈加速器”,格雷卡希望发现更多节点连接,并开始开发能够治疗这些疾病的药物。

Greka has already shown that the nodal approach can work. In 2019, she and her team published a paper outlining a mechanism they discovered for a rare kidney disease that also plays a role in diseases that impact the eye and brain. With the Ladders to Cures Accelerator, Greka hopes to discover even more nodal connections and start to develop drugs that can treat them.

10月27日,博德研究所将举办第三届年度“通往治愈”研讨会。格雷卡将出席,她的同事大卫·刘(David Liu)博士、温斯顿·严(Winston Yan)博士以及麻省理工学院经济学家安德鲁·洛(Andrew Lo)也将一同出席。

On October 27th, the Broad Institute will host the 3rd Annual Ladders to Cures Symposium. Greka will be there, along with her colleagues Dr. David Liu, Dr. Winston Yan, and MIT economist Andrew Lo.

在活动之前,贝基·奎克(Becky Quick)与格雷卡进行了深入交谈,询问了“通往治愈加速器”正在开展的工作以及她希望该加速器对罕见病群体产生的影响。

Ahead of the event, Becky Quick sat down with Greka to ask about the work being done at Ladders to Cures and the impact she hopes the accelerator can have on the rare community.

CNBC Cures栏目由阿斯利康罕见病业务亚力克森(Alexion)赞助支持。

CNBC Cures is underwritten by Alexion, AstraZeneca Rare Disease.