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.

With RNAi therapeutics, we believe that we’ve created a modular and reproducible approach for development and commercialization of innovative 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.

In December 2014, we established an evolution in our strategy with a focus on development and commercialization of investigational RNAi therapeutics in 3 Strategic Therapeutic Areas (STArs):

  • Genetic Medicine STAr, where we are advancing a broad pipeline of RNAi therapeutics for the treatment of rare diseases;
  • Cardio-Metabolic STAr, focused on programs toward genetically validated, liver-expressed disease targets for unmet needs in dyslipidemia, hypertension, non-alcoholic steatohepatitis (NASH), and type 2 diabetes; and
  • Hepatic Infectious Disease STAr, where we are advancing a pipeline of programs that address major global health challenges, including hepatitis B virus (HBV) and hepatitis D virus (HDV) infections, amongst other infectious disease opportunities.

In January 2015, we launched our “Alnylam 2020” guidance that reflects our expected transition from a late-stage clinical development company to a multi-product commercial-stage company with a sustainable development pipeline.  Specifically, by the end of 2020, we expect to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs – including 4 in late stages of development – across our 3 STARs.