美国同业Alnylam Pharmaceuticals Inc.(ALNY)  
Alnylam是一家生物制药公司，基于 RNA干扰 或RNAi开发新颖的疗法。公司正在将其治疗方面的专门技术应用于RNAi，以满足重大的医疗需求，而目前的主要药物类别小分子或抗体药物无法满足其中的许多需求。Alnylam正在率先将RNAi转化为一类新的创新性药物，其同侪审评的研究成果已在全世界一流的科学杂志包括《自然》、《自然药物》和《细胞》中发表。公司正在运用这些能力，建立一种广泛的 RNAi疗法管道；其最前沿的计划是治疗呼吸道合胞病毒(RSV)感染的二期人体临床试验，该试验与Cubist和Kyowa Hakko合作进行。此外，公司正在开发RNAi疗法，治疗多种疾病，包括肝癌、高胆固醇血症、亨廷顿舞蹈病和TTR 淀粉样变性病。公司在RNAi方面的基本专利、技术和专门知识上占据领先地位，从而得以与Medtronic、Novartis、Biogen Idec、Roche、Takeda、Kyowa Hakko Kirin和Cubist等首屈一指的公司结为主要联盟。为了反映公司对主要的科学、临床和商业倡议的看法，Alnylam在2008年1月建立“RNAi 2010”，包括公司的以下规划：显著扩展 RNAi疗法给药方法解决方案范围，拥有四个或更多的临床开发规划，建立四项或更多的主要业务新合作关系，所有这一切都在2010年年底之前完成。Alnylam和Isis共同拥有Regulus Therapeutics Inc.，Regulus Therapeutics Inc.侧重于微RNA疗法的发现、开发和商业化。Alnylam于2002年创建，总部设于麻省剑桥。

Phil Sharp

Alnylam的创始人之一

美国生物学家菲利普·夏普的主要研究领域是植物学、生物化学、核糖核酸调控。他是获得美国国内院士称号最多的科学家之一，因发现断裂基因而获得1993年诺贝尔生理学或医学奖。他曾任麻省理工学院生物系主任和癌症研究中心主任。夏普教授在生物医学领域作出了巨大贡献，在国际上享有盛誉。他因开发新的医药产品而获得美国国家技术奖章。目前，他的主要研究方向为RNA干扰素，为治疗疾病带来新思路。


Alnylam is developing an entirely new class of innovative medicines based on a breakthrough discovery in biology known as RNA interference, or RNAi. With RNAi technology, we have the opportunity to treat disease and impact the lives of patients in a fundamentally new way by silencing disease-causing genes upstream of today's medicines.

The meaning of our name, Alnylam(al-NIGH-lam)
Located in the constellation Orion, Alnylam is the name of the center star of Orion's belt. The star has a luminosity that is 250,000 times greater than the sun, representative of the potential strength that RNAi therapeutics could bring to bear for human health.
As scientists continue to investigate the human genome, where we now have the complete genetic sequence, there have been remarkable developments in the understanding of disease and consequently a large increase in the number of molecular disease targets to impact a broad range of human disease. Paradoxically, with all these new opportunities to significantly impact disease, the challenge for the industry has been that today's therapeutic modalities, such as small molecules and monoclonal antibodies cannot access most (~70%-80%) of these new disease targets. With RNAi, we can target virtually any gene in the genome involved in the causal pathway of disease. The possibility of targeting these previously "undruggable" targets with RNAi is transformative for new drug discovery.

We have made very significant strides in the development and advancement of RNAi therapeutics. For example, in 2004. Alnylam scientists demonstrated the ability to deliver RNAi therapeutics in mice mice achieving a desired therapeutic effect, and in 2006, we achieved similar results in non-human primates; both of these landmark studies were published in the journal Nature. In 2008, with our Phase II GEMINI study, we showed for the first time that RNAi works in man when we demonstrated that an RNAi therapeutic achieved statistically significant efficacy in a randomized, double-blind, placebo-controlled human clinical trial. By all measures, this represents clear and very rapid progress.

Our strategy at Alnylam is to lead the translation of the science of RNAi into a robust drug discovery capability and to build a significant product pipeline of innovative medicines that we commercialize alone or with our partners. The pace of biomedical discovery is faster than ever and the hunger for innovative medicines is greater than ever. This environment, when combined with Alnylam's unparalleled intellectual property position for RNAi therapeutics and our industry-leading alliances, creates a unique opportunity for Alnylam to build a leading biopharmaceutical company.