About Alnylam
Harnessing a revolution in biology for human health®
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 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.
