Narrow-Moat Alnylam Navigates a Highly Competitive ATTR Market
Alnylam Pharmaceuticals is a pioneer in designing therapeutics based on RNA interference, or RNAi, a mechanism that occurs naturally in cells and silences overexpressed genes, which often cause protein misfolding and/or protein accumulation. Alnylam seeks to capitalize on the therapeutic potential of RNAi by creating small interfering RNA, or siRNA, that can treat diseases at the genetic level. When siRNA reaches its intended target, it can silence the gene that it contains code for, making it a potentially game-changing technology for difficult- to-treat diseases caused by genetic mutations.
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