2015年1月14日讯 /生物谷BIOON/ --2015年刚刚到来，Moderna的管理人员恐怕睡觉都会笑出声来。继Moderna公司在与武田药业签订了价值3亿美元的研发协议之后，其长期合作伙伴默沙东又与Moderna公司签订了价值1亿美元的RNA药物研发协议。接连两笔大单或许预示着2015年Moderna公司将迎来一个快速发展期。
根据此次与默沙东公司签订的协议，默沙东公司将向Moderna公司支付5000万美元的现金以交换五种处于临床前研究的抗病毒候选药物分子。此外，默沙东公司还向Moderna公司许诺了数额未名的里程碑资金。而Moderna公司之所以如此受到默沙东青睐的利器之一就是公司开发的输送RNA药物技术。
一直以来，科学家们都在考虑如何将蛋白质药物、小分子药物输送至病灶内。但是这种输送方式仍然会面临着药物被很快分解，药效降低以及毒副作用大等问题。近年来兴起的RNA疗法研究则为这一问题提供了一个新的思路。科学家们设想将特定的mRNA输送至患者细胞内，这样就可以将一个个细胞转变为"药物加工厂"，使其自动生成治疗所需药物。这样，一方面药物的药效将大大延长；另一方面也使得药物蛋白能够富集在需要治疗的部位，从而降低药物副作用。Moderna公司目前正在进行的类似研究共有约45项左右。
近几年来，许多生物医药产业分析人士都将RNA疗法看做是未来生物医药产业的重要发展方向，许多生物医药巨头也都在这一领域投入了大量的人力物力。但是，受限于药物的输送技术和安全性考虑，一些企业对这一领域也有不小的担忧。去年，诺华公司退出这一领域就代表了这些企业的消极看法。
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Pharma giant Merck ($MRK) is buying into the allure ofModerna Therapeutics and its radical promise to transform patients' cells into drug factories, committing $100 million and joining a host of collaborators and investors betting the early-stage biotech is sitting on transformational technology.
Under the deal, Merck will pay $50 million in cash to partner up on 5 antiviral treatments in preclinical development, promising undisclosed milestone payments and royalties down the line. In tandem, the company is making a $50 million equity investment in Moderna, piling onto the $450 million round the biotech closed earlier this month.
The big draw is Moderna's flagship technology, which uses messenger RNA (mRNA) to spur the production of human proteins or antibodies within patient cells, effectively creating an in vivo factory for targeted therapies. The company believes its proprietary approach to mRNA has the potential to treat previously undruggable targets in a wide range of disease areas, touting 45 preclinical programs in its pipeline.
Merck and Moderna are now in a three-year collaboration in which the pair will craft mRNA vaccines that spur the production of antibodies against viruses and other infectious ailments, fighting the spread of disease from within the body.
"Given the tremendous potential for messenger RNA therapeutics across a wide range of therapeutic applications, establishing long-term strategic relationships with world leaders in their fields will accelerate our ability to bring mRNA products to patients in need," Moderna CEO Stéphane Bancel. "Merck's worldwide leadership in vaccines and anti-infective treatments make them an ideal collaborator for us, particularly given their strong commitment to innovation and new approaches to prevent and treat serious viral diseases."
Merck's interest is a valuable co-sign for Moderna, which, despite raising unprecedented sums of money, has yet to get any of its candidates into the clinic and faces some skepticism around the industry. After wrapping its latest funding round, the biotech pledged to buckle down and start marshaling its pipeline assets toward Phase I, and Bancel told Xconomy the company expects to begin its first human trials in the next 12 to 24 months. In the meantime, Moderna is looking to hire more than 100 scientists and researchers to bolster its existing staff of 145.
The Merck deal is Moderna's third major industry partnership, following a $420 million deal with AstraZeneca ($AZN), signed in 2013, in which a whopping $240 million of that total came in up-front cash, among the largest down payments in industry history. Months later, rare disease specialist Alexion ($ALXN) stepped in with a $125 million deal to get its hands on 10 mRNA candidates. In total, Moderna has banked more than $950 million in venture and collaboration cash since its 2011 foundation by Flagship Ventures.