RNA interference drug development
Alnylam Pharmaceuticals (ALNY) is a Cambridge, Massachusetts company focused on the development and commercialization of novel RNAi therapeutics for the treatment of diseases with high unmet medical need and genetically defined targets.
Alnylam expects to have five RNAi programs in clinical development, or in commercial stages, by the end of 2015. The company's core product strategy, called "Alnylam 5x15", involves developing all five program candidates with the following shared characteristics:
- Specificity for genetically defined target and disease.
- Potential to significantly impact high unmet need populations.
- Ability to leverage the company's existing RNAi delivery platform.
- Opportunity to monitor an early biomarker in Phase 1 clinical trials for human proof of concept.
- Existence of clinically relevant
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