In 2013, Ted W. Love — a former drug developer at Onyx, Genentech and elsewhere and cardiologist by training — came out of retirement to take on sickle cell disease. This inherited blood disorder, in which sickled red blood cells clump together, blocking blood flow and the delivery of oxygen through the body, afflicts nearly 100,000 people in the USA and millions more throughout the world. And yet, until recently, these patients have had few therapeutic options for preventing the organ damage, risk of stroke and severe pain associated with disease. Last year, as CEO of Global Blood Therapeutics, Love secured an FDA approval for his company’s voxelotor, a first-in-class anti-sickling agent that at last takes aim at the underlying pathophysiology of the disease. He spoke with Asher Mullard about the science of voxelotor, his hopes of being able to turn sickle cell disease into a quiescent condition and the FDA’s embrace of surrogate end points in more and more indications.

中文翻译：

---

特德·W·爱

2013年，Ted W. Love（曾在Onyx，Genentech和其他地方担任药物开发人员，并且是心脏病专家，通过培训）从退休中退休，接受镰状细胞病治疗。这种遗传性血液疾病使镰状红细胞聚集在一起，阻止血液流动和氧气通过人体的输送，在美国折磨了近100,000人，在世界范围内折磨了数百万。然而，直到最近，这些患者还没有用于预防器官损伤，中风和与疾病相关的严重疼痛的治疗选择。去年，洛夫（Love）作为全球血液疗法公司（Global Blood Therapeutics）的首席执行官，为其公司的voxelotor药获得了FDA的批准，该药是一种一流的抗-药，最终针对该疾病的潜在病理生理学。他与Asher Mullard谈到了voxelotor的科学，