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About Alnylam

Alnylam is leading the translation of RNAi as a new class of innovative medicines, with a core focus on RNAi therapeutics toward genetically defined targets for the treatment of serious, life-threatening diseases with limited treatment options for patients and their caregivers.

Our ‘Alnylam 5×15′ and genetic medicine product strategy establishes a path for development and commercialization of novel RNAi therapeutics. With RNAi therapeutics, we believe that we’ve engineered a modular and reproducible approach for development and commercialization of innovative genetic medicines.

The products candidates we are developing with this approach share several key characteristics including:

  • the potential to have a major impact in a high unmet need population;
  • a genetically defined, liver-expressed target gene involved in disease and validated in human genetics;
  • the ability to leverage the existing Alnylam RNAi delivery platform;
  • the opportunity to monitor an early, blood-based biomarker with strong disease correlation in Phase 1 clinical trials for human proof of concept; and
  • the existence of clinically relevant endpoints for the filing of a new drug application (NDA) with a focused patient database and possible accelerated paths for commercialization.

We launched our ‘Alnylam 5×15’ product strategy in early 2011. At that time, we guided that by the end of 2015, we would have five such RNAi therapeutic programs in clinical development, including programs in advanced stages. In early 2014, we announced that we plan on exceeding that original guidance, with six to seven programs in clinical development, at least two programs in Phase 3 trials, and five to six programs with human proof-of-concept by the end of 2015.

At Alnylam, we are focused on developing and commercializing our ‘Alnylam 5×15’ and genetic medicine programs in North America and Europe. In early 2014, we formed a transformational alliance with Genzyme, a Sanofi company, where Genzyme will develop and commercialize Alnylam’s ‘5×15’ and other future genetic medicine programs – up until 2020 – in the rest of world. This new strategic partnership is intended to significantly expand and accelerate the global value of our  product pipeline, and also facilitates an increased investment in our genetic medicine discovery pipeline for future products.