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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

In preparation for this, drug manufacturing will begin later this year and will be outsourced to external contract manufacturing organisations (CMOs). The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. David Bumcrot PhD, CSO, CAMP4 Therapeutics.

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The evolution of the RNA therapeutics landscape 

Drug Discovery World

RNA interference: silencing gene expression The second category of RNA therapeutics is a class that has also yielded marketable drugs in recent years. These drugs, known as RNA interference (RNAi)-based therapeutics use Watson-Crick base-pairing to form an RNA helix, short duplexes of double-stranded RNA molecules.

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