Importance of RNA Binding in Gene Regulation Validates CAMP4’s Approach

CAMP4 Therapeutics, a biotechnology company harnessing the power of regulatory RNA to restore healthy gene expression, announced research published by the lab of co-founder Richard Young, Ph.D., in the journal Molecular Cell showing that transcription factors (TFs) interact with non-coding RNA to finely tune the regulation of gene expression. This research validates CAMP4’s therapeutic approach of targeting regulatory RNAs (regRNAs) to affect gene expression.

For the last 40 years, it’s been known that TFs, which orchestrate a cell’s gene expression programs, bind to DNA and proteins. The new research published by Dr. Young’s lab reveals that many TFs also bind to RNA through a previously unrecognized domain.

“Until now, our knowledge of transcription factors binding to RNA was limited to a few isolated examples,” said Dr. Young. “Our research shows that many transcription factors need to interact with DNA, protein, and RNA to conduct their normal gene regulation functions. This finding opens up a whole new world of thinking about how RNA molecules regulate gene expression in all of our cells. These insights underpin CAMP4’s regulatory RNA-based approach to developing new therapeutic across a range of diseases.”

Dr. Young and lead authors Ozgur Oksuz, Ph.D., and Jonathan E. Henninger, Ph.D., and colleagues discovered that at least half of the approximately 1,600 TFs in the human genome also bind to RNA. Their research found that RNA binding contributes to TF function by promoting the dynamic association between DNA, RNA, and TF on chromatin. It also shows that these interactions are a conserved feature essential for healthy development but disrupted in disease.

“This breakthrough research elucidates a key underlying mechanism of RNA’s role in gene regulation and provides biological validation of our approach,” said Josh Mandel-Brehm, CEO of CAMP4. “At CAMP4, we are exploiting these regulatory interactions to very specifically control the expression of genes tied to disease. Our proprietary platform targeting regRNAs has the potential to address hundreds of genetic diseases in which amplifying healthy protein can offer therapeutic benefits.”

The Molecular Cell article “Transcription factors interact with RNA to regulate genes” was published on July 3, 2023. Dr. Young is a Member of the Whitehead Institute and a Professor of Biology at the Massachusetts Institute of Technology. His lab is highly regarded for exploring gene regulation in health and disease. He serves on the Board of Directors of CAMP4 and several other biotech companies.

About CAMP4 Therapeutics

CAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplifying healthy protein can offer therapeutic benefits. Our approach amplifies mRNA by harnessing a fundamental mechanism of how genes are controlled. To amplify mRNA, our therapeutic candidates target regulatory RNAs (regRNAs), which act locally on transcription factors and are the master regulators of gene expression. Our RAP Platform™, which allows us to map regRNAs and select the optimal chemistry to generate safe and effective therapeutics, has the potential to address hundreds of genetic diseases across multiple tissues. Learn more about us at www.CAMP4tx.com and follow us @CAMP4tx.

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