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Royalty Pharma plc (NASDAQ:RPRX)
Finance & Acquisitions: Royalty Pharma
Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY)*
Organization & Developments: Alnylam
*Organization: Alnylam
Parents
Organization & Developments: Alnylam
Children
*ATTR amyloidosis can lead to significant morbidity, disability, and mortality.
*Isis and Alnylam Pharmaceuticals are joint owners of Regulus Therapeutics Inc., a company focused on the discovery, development of microRNA
*RNA interference-based pharmaceuticals
*RNAi creates the opportunity to silence the production of disease-causing proteins and therefore, represents a whole new approac
*This disease is a progressive, life-threatening disease caused by misfolded transthyretin ("TTR") proteins that accumulate as amyloid fibrils in multiple organs, but primarily in the peripheral nerves and heart.
2016: Its most advance trials are in Phase III targeting hereditary ATTR amyloidosis.
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*Developments: Alnylam