基因君官网
2015年4月6日讯 /生物谷BIOON/ –百时美施贵宝（BMS）近日与人类基因治疗领域的领导者——荷兰生物技术公司uniQue签署了一份高达10亿美元的独家合作协议，获得了uniQue公司的基因治疗技术平台，开发基因疗法用于心血管疾病领域多达10个靶标。
根据协议，百时美将支付1亿美元的预付款，获得uniQue公司候选基因疗法S100A1及另外3个潜在基因疗法的商业化权利。uniQue将负责所有发现及生产活动，并有资格获得S100A1高达2.54亿美元的里程碑款项及另外3个基因疗法每个2.17亿美元的里程碑款项，使得该笔合作的金额达到了10亿美元之多。百时美则负责所有的研发成本，并支付产品在未来商业化销售的特许权使用费。
uniQue公司S100A1基因疗法是2014年收购德国InoCard公司获得，利用了一种无害的病毒递送钙结合蛋白S100A1，这是一种心脏功能的主要调节器。通过增加S100A1，缓解充血性心力衰竭（CHF）的疾病症状。除了S100A1基因疗法，双方合作的其余项目，将利用uniQue公司研发的基因治疗平台，利用腺相关病毒（AAV）递送矫正性基因至疾病靶标，同时将依赖基于昆虫细胞的生产技术，生产工业化规模剂量的基因治疗产品。
尽管uniQue是一家小型生物技术公司，但在基因治疗领域却一直处于领先地位，该公司在2012年推出了欧洲首个基因治疗产品Glybera。Glybera利用腺相关病毒（AAV）将一个功能性脂蛋白脂肪酶（LPL）基因递送至骨骼肌，用于治疗一种极其罕见的遗传性代谢性疾病——脂蛋白脂肪酶缺乏症（LPLD）。Glybera在欧洲的上市，标志着人类基因治疗领域的一个重大里程碑。
相对于传统的蛋白替代策略，基因疗法能够提供更高的临床治疗利益，这是因为基因疗法能够恢复自然的身体机能，而不仅仅是短期修复。然而，到目前为止，基因治疗领域的大部分工作主要集中于罕见性遗传病领域。
而S100A1基因疗法则是为数不多的几个靶向常见疾病的基因疗法之一，开发用于治疗充血性心力衰竭（CHF），患者群体非常庞大。若S100A1基因疗法能够成功，将进一步提振目前正在缓慢复苏的人类基因治疗领域。
该笔交易，也标志着百时美开始涉足基因治疗领域，过去多年该领域一直被药物安全性和有效性所困扰。不过，该领域正在慢慢复苏。就在去年，辉瑞、赛诺菲、拜耳均与其他基因治疗公司在该领域达成了合作，而百健（Biogen）则设立了一个独立的研发单元专注于基因治疗。（基因宝jiyinbao.com）
英文原文：Bristol-Myers bets big on gene therapy with a $1B uniQure deal
Bristol-Myers Squibb ($BMY) is the latest pharma heavyweight to buy into gene therapy’s renaissance, signing a deal with uniQure ($QURE) worth as much as $1 billion and getting its hands a novel cardiovascular treatment.
Under the agreement, Bristol-Myers will hand over $100 million in up-front payments, licensing fees and equity investments in exchange for the commercial rights to uniQure’s lead candidate and three more potential therapies. The Holland headquartered-uniQure will handle all discovery and manufacturing in the collaboration, eligible for up to $254 million tied to top prospect S100A1 and $217 million for each of the three other assets. Bristol-Myers is on the hook for all R&D costs, promising its partner a royalty on future sales.
UniQure’s lead candidate, picked up in an acquisition of Germany’s InoCard last year, is a gene therapy that uses a harmless virus to deliver the calcium-binding protein S100A1, a master regulator of cardiac function. By boosting S100A1, the treatment has the potential to relieve symptoms of congestive heart failure, according to uniQure, and Bristol-Myers is betting its cardio expertise can push the program forward.
Beyond the top prospect, each of the pair’s subsequent collaborations will use uniQure’s in-house gene therapy platform, which uses adeno-associated viruses (AAV) to shuttle corrective genes to their targets and relies on insect-cell-based manufacturing technology to produce doses at an industrial scale. UniQure has long been a leader among the new generation of gene therapy companies, making history in 2012 when its Glybera became the first such treatment to win a global approval.
To date, the majority of gene therapy work has focused on rare genetic diseases–including Glybera, a treatment for familial lipoprotein lipase deficiency. S100A1 stands out as one of the few targeting a major indication with a huge patient population, and success could further bolster the resurgent gene therapy space, uniQure CEO Joern Aldag has said.
“Bristol-Myers Squibb’s strength in the cardiovascular area and its commitment to gene therapy will allow them to leverage the full breadth and capacity of our platform for cardiovascular diseases,” Aldag said in a statement. “This collaboration will accelerate the application of gene therapy for large patient populations suffering from heart diseases and will complement the further development of uniQure’s internal pipeline in two focus areas: liver diseases, including hemophilia; and CNS, including lysosomal storage diseases.”
News of the agreement sent uniQure’s shares soaring up 50% on Monday morning.
With the deal, Bristol-Myers joins a long line of traditional biopharma powers taking the plunge on gene therapy, a field long dogged by safety and efficacy concerns. Over the past year, Pfizer ($PFE), Sanofi ($SNY), Bayer and others have inked deals with pioneering biotechs to get some irons in the fire, while Biogen ($BIIB) has made gene therapy a standalone R&D vertical.