CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders
Pharmaceutical Technology
JULY 29, 2022
In 2013, the US Food and Drug Administration (FDA) approved Ravicti (glycerol phenylbutyrate), manufactured by Hyperion Therapeutics, which has since then been acquired by Horizon Pharma. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Future Possibilities.
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