Empowered Patient Podcast Significance of Regulatory RNA in Protein Production in Specific Genes with Dr. Yuri Maricich CAMP4 Therapeutics
Dr. Yuri Maricich, Chief Medical Officer at CAMP4 Therapeutics, describes regulatory RNA, a new area of biology that recognizes the role of Reg RNA in the production of proteins from specific genes. This technology is well-suited for haploinsufficient diseases such as SYNGAP1-related disorders, in which there is a lack of healthy protein and both parents carry a copy of the mutated gene. The goal is to create disease-modifying treatments that correct the underlying genetic cause rather than treating the symptoms.
Yuri explains, "What was really unique about CAMP4's scientific approach is that we're focused on a whole new and emerging area of biology called regulatory RNA. And these are control elements for the expression of genes. In other words, how much protein we get from a particular gene. And there's been a lot of work in the past on how to have less protein made, particularly if it's a protein that has a mutation that causes a problem. But in medicine, there have been very few opportunities to actually increase the amount of protein, but there are many diseases that need more healthy protein."
"The backdrop of CAMP4 is that there was work done just over eight years ago at the Whitehead Institute at the Massachusetts Institute of Technology in a lab by Rick Young, and he was working with a colleague at Boston Children's Hospital, Lenson, and they noticed that there was this group of so-called regRNAs. These were non-coding regions that historically have been really ignored. And as they looked and explored their function further, what they discovered was that, in fact, these regRNA elements play a critical role in controlling how much protein is produced. And so the story of CAMP4 has been to continue to understand and map different cell lines so that we could take a particular target gene and, by using tools or established medicines, for example, like antisense oligonucleotides, we could actually increase the amount of a gene's protein back up to normal."
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