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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

Ultragenyx uses adeno-associated virus 8 (AAV8) gene therapy to induce stable OTC gene expression. This treatment activates the OTC gene so that ammonia can be removed from the blood. According to Bumcrot, regRNAs are “RNAs that arise out of the non-coding genome”. Future Possibilities.

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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