宾夕法尼亚州荷兰乡村等不及生物制药公司，自建药企
STAT+: Pennsylvania Dutch country, tired of waiting for biopharma, builds drug company of its own
宾夕法尼亚州兰开斯特的荷兰乡村地区因长期等待生物制药公司无果，决定自建药企。研究人员Kevin Strauss用携带新基因的病毒治疗了患枫糖尿症（MSUD）的小牛Petunia，证明MSUD基因疗法可行，但迄今无一名患者接受该治疗，临床试验亦未开展。
LANCASTER, Pa. — They saved Petunia first. She was the second cow. Her brother Louie had died within hours of his birth. But over two harrowing, sleepless nights, the three researchers kept Petunia alive to wheel her from the barn to the veterinary hospital and inject her with a virus carrying new genes.
Petunia had a bovine form of maple syrup urine disease (MSUD), a life-threatening condition that is also unusually prevalent within the Mennonite community here. The goal wasn’t to save the 79-pound calf. That would be a bonus. The goal, for Kevin Strauss, was to prove that an MSUD gene therapy could work — that after two decades he could finally offer the desperate families who entered his medical clinic here a simple, one-time fix.
It worked. Today, Petunia is a feisty, 5-year-old, hay-munching, 1,500 pound heifer. Yet not a single one of Strauss’s patients has received the treatment that saved her life, not even in a clinical trial.
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来源：STAT News · statnews.com