辉瑞签署2笔协议宣布进入罕见病基因治疗领域

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辉瑞（Pfizer）周一宣布2项战略决策，通过组建基因治疗平台调查潜在的基因疗法，扩大qixia罕见病研发活动。在首笔交易中，辉瑞与生物技术公司Spark Therapeutics签署协议，开发SPK-FIX项目，该项目纳入一种生物工程化腺相关病毒（AAV）载体用于B型血有病的潜在治疗，辉瑞预计于2015上半年将该项目推进至I/II期临床试验。Spark专注基因治疗领域，目前正在开发针对眼科遗传性疾病的基因疗法，今年成功上榜FierceBiotech的《2014最热门的15家生物技术公司》。

根据协议，Spark将获得一笔2000万美元的现金并有资格获得高达2.6亿美元的额外款项。在第二笔交易中，辉瑞将委任英国伦敦大学国王学院教授兼伦敦大学学院基因治疗联盟主任Michael Linden博士，在公司进行为期2年的借调，领导罕见病领域的基因治疗研究。

基因治疗领域已开展了超过20年的试验，但一直被一系列挫折和安全性问题所困扰。然而现在，科学家已解决了一些早期问题，并已经推进至临床。近日，西方世界首个基因疗法Glybera在德国上市，该药售价高达110万欧元（约合140万美元），创下了治疗罕见病药价的新纪录。Glybera由荷兰生物技术公司UniQure花费了25年的时间才研发成功，该药用于治疗一种名为脂蛋白酯酶缺乏遗传病（LPLD）的超罕见血液疾病，在整个欧洲只有150~200名患者可能符合使用Glybera的条件。（相关阅读：重磅！首种基因治疗药物将上市 价格高达140万美元）

辉瑞全球研发部总裁Mikael Dolsten博士表示，将遗传性罕见病最根本的知识与基因技术中的科学进步结为一体，借助腺相关病毒（AAV）作为基因运载工具，为调查新一轮潜在改变患者生活的基因疗法提供了一个成熟的机会。

根据协议，总部位于费城的Spark Therapeutics将与辉瑞合作推进SPK-FIX临床项目。Spark将负责临床开发的I/II研究，而辉瑞则负责关键III期临床试验、监管批准及产品的潜在商业化

此外，从2014年12月1日期，Michael Linden教授正式加入辉瑞进行为期2年的借调并领导辉瑞罕见病领域的基因治疗研究。Michael Linden教授在基因治疗领域开展了20多年的研究，在AAV技术方面拥有极其丰富的专业知识。辉瑞将在Michael Linden教授的领导下成立基因治疗研究组，帮助辉瑞探索将这一重要技术应用于罕见病领域。

英文原文：Pfizer bets on gene therapy as technology comes of age

LONDON, Dec 8 (Reuters) - Pfizer Inc is moving into the gene therapy space in the latest sign that the technology for fixing faulty genes may finally be ready for prime time, following earlier setbacks.

The U.S. drugmaker said on Monday it was establishing a gene therapy platform to study potential treatments, led by a top UK expert, and had struck a deal with privately owned U.S. biotech firm Spark Therapeutics to develop a treatment for haemophilia.

The Spark programme is expected to enter early-stage clinical trials for haemophilia B in the first half of 2015. Spark will be responsible for the early Phase I/II tests, with Pfizer taking over late-stage studies, any regulatory approvals and potential commercialisation.

Spark will get $20 million upfront and be eligible for additional payments based on product success worth up to $260 million.

Pfizer's research effort in gene therapy will be led by Michael Linden, a professor from King's College London and director of the University College London Gene Therapy Consortium. Linden is joining Pfizer on a two-year secondment.

Gene therapy has seen more than 20 years of experiments but research has been dogged by a series of disappointments and safety concerns.

Now, however, scientists have solved some of the earlier problems and treatments are starting to reach the clinic, with a the Western world's first gene therapy set to go on sale in Germany to treat and ultra-rare blood disease.

"The fundamental understanding of the biology of hereditary rare diseases, coupled with advances in the technology to harness disarmed viruses as gene delivery vehicles, provide a ripe opportunity to investigate the next wave of potential life-changing therapies for patients," said Pfizer research head Mikael Dolsten.

Among other major pharmaceutical companies, Bayer AG struck a gene therapy deal with Dimension Therapeutics in June, while Novartis AG recently established a new cell and gene therapies unit, and Sanofi SA has a long-standing tie-up with Oxford BioMedica .
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