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The development of RNA interference (RNAi) treatments has been difficult to say the least.

Early on there were many clinical trials with serious side effects and patient deaths. 1 One of the
most problematic was Alnylam’s phase 3 trial for revusiran, its RNAi therapeutic for treating
transthyretin amyloidosis with cardiomyopathy, one of the two main forms of the disease. An
imbalance in the number of deaths—19 in total—caused the Cambridge, Massachusetts–based
biotech to end the program on October 4, 2016. One day after the announcement, the company's
stock price fell by almost half, wiping out $3.6 billion in market value.2

These difficulties resulted in several large pharmaceutical companies pulling out of RNAi
research and development completely. However, Alnylam and a few other companies continued.
On August 10, 2018, the first RNAi therapy was approved by the FDA when Alnylam
announced that the FDA had approved Onpattro (patisiran) for the treatment of polyneuropathy
of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Like many disruptive technologies, the RNAi

1
https://www.labiotech.eu/in-depth/rnai-new-developments/
2
https://www.nature.com/articles/nbt1216-1213

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