A new drug approval for the vanguard of RNA-targeted small molecules
Dark Matter Blog
AUGUST 27, 2020
A few years ago, at Arrakis Therapeutics, we set out to conquer a strange new territory, drugging RNA structures with small molecules. Using gene therapy, their approach was beautifully straightforward – “Missing an important gene? The agent, onasemnogene abeparvovec, was approved by the FDA in 2019 and is marketed as Zolgensma.
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