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About RNAi & Alnylam

About RNAi & Alnylam

  • Posted by ISPE Boston
  • On August 15, 2018

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.

RNAi therapeutics are a new class of medicines that harness the natural biological process of RNAi. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing proteins – thus preventing them from being made. This is a revolutionary approach in developing medicines to improve the care of patients with genetic and other diseases. (Source: Alnylam Website, 10 August, 2018)

 

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