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

Pharmaceutical Technology

JULY 29, 2022

However, in patients with urea cycle disorders, genetic defects result in inadequate amounts of the enzymes needed to convert nitrogen into urea. Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. Their platform identifies which regRNAs control the specific gene of interest.

RNA 245
RNA Gene Expression Gene Gene Therapy 245
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Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

APRIL 17, 2024

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. DS: How easy was it to transfer theoretical research into a viable business?

Gene Expression 59
Gene Expression Gene Genetic Disease RNA 59
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