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美國同業(yè)Alnylam Pharmaceuticals Inc.(ALNY)  
Alnylam是一家生物制藥公司，基于 RNA干擾 或RNAi開發(fā)新穎的療法。公司正在將其治療方面的專門技術應用于RNAi，以滿足重大的醫(yī)療需求，而目前的主要藥物類別小分子或抗體藥物無法滿足其中的許多需求。Alnylam正在率先將RNAi轉化為一類新的創(chuàng)新性藥物，其同儕審評的研究成果已在全世界一流的科學雜志包括《自然》、《自然藥物》和《細胞》中發(fā)表。公司正在運用這些能力，建立一種廣泛的 RNAi療法管道；其最前沿的計劃是治療呼吸道合胞病毒(RSV)感染的二期人體臨床試驗，該試驗與Cubist和Kyowa Hakko合作進行。此外，公司正在開發(fā)RNAi療法，治療多種疾病，包括肝癌、高膽固醇血癥、亨廷頓舞蹈病和TTR 淀粉樣變性病。公司在RNAi方面的基本專利、技術和專門知識上占據(jù)領先地位，從而得以與Medtronic、Novartis、Biogen Idec、Roche、Takeda、Kyowa Hakko Kirin和Cubist等首屈一指的公司結為主要聯(lián)盟。為了反映公司對主要的科學、臨床和商業(yè)倡議的看法，Alnylam在2008年1月建立“RNAi 2010”，包括公司的以下規(guī)劃：顯著擴展 RNAi療法給藥方法解決方案范圍，擁有四個或更多的臨床開發(fā)規(guī)劃，建立四項或更多的主要業(yè)務新合作關系，所有這一切都在2010年年底之前完成。Alnylam和Isis共同擁有Regulus Therapeutics Inc.，Regulus Therapeutics Inc.側重于微RNA療法的發(fā)現(xiàn)、開發(fā)和商業(yè)化。Alnylam于2002年創(chuàng)建，總部設于麻省劍橋。

Phil Sharp

Alnylam的創(chuàng)始人之一

美國生物學家菲利普·夏普的主要研究領域是植物學、生物化學、核糖核酸調控。他是獲得美國國內院士稱號最多的科學家之一，因發(fā)現(xiàn)斷裂基因而獲得1993年諾貝爾生理學或醫(yī)學獎。他曾任麻省理工學院生物系主任和癌癥研究中心主任。夏普教授在生物醫(yī)學領域作出了巨大貢獻，在國際上享有盛譽。他因開發(fā)新的醫(yī)藥產品而獲得美國國家技術獎章。目前，他的主要研究方向為RNA干擾素，為治療疾病帶來新思路。


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.