BBIO - RNAi: Winning In The Clinic Now For The Market

2024-06-25 06:45:00 ET

Summary

• Alnylam has been extremely persistent over the years in getting their RNA interference ideas developed and tested.
• That original RNAi therapy (patisiran) was directed towards neuropathy, but Alnylam has been hoping that such treatments would show benefit for cardiovascular hATTR.
• It'll take some time to sort out what the best combination of therapies will be, and at the moment it's not clear at all, not least because of the number of different mutations that can lead to the disease in the first place.

Alnylam ( ALNY ) has been extremely persistent over the years in getting their RNA interference ideas developed and tested. The company had a big moment in 2017 when they had successful results in treating hATTR amyloidosis by blocking production of the troublesome protein behind it. That was certainly good news because there was basically nothing that could be done for those patients at the time. That acronym stands for "hereditary transthyretin amyloidosis", and the underlying cause is some mutation in the TTR gene causing less-stable forms of transthyretin protein to be produced. There are at least 114 (!) known mutations that lead to this problem, and the resulting TTR ends up forming tetrameric amyloid fibrils that can cause pathology in the kidneys, liver, GI tract, CNS, and cardiovascular system. There are different historical names for several of these diseases, which were gradually realized to be part of a similar etiology....

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