CAMP4 Therapeutics presents preclinical data demonstrating the utility of its RNA Actuating Platform (RAP) and the therapeutic potential of oligonucleotide-mediated gene upregulation targeting a new class of RNA known as regulatory RNAs (“regRNAs”).

The data describe this novel treatment approach for two genetically defined diseases, one being HAE.

“These data demonstrate that by using oligonucleotides to target regRNA, we can directly upregulate endogenous gene expression at the transcriptional level and achieve a therapeutically relevant result,” says David Bumcrot, Ph.D., Chief Scientific Officer of CAMP4. “With the results of these studies, we have the early proof points that our novel approach to harness the power of regRNA for gene upregulation works across multiple genes, fueling our conviction as we advance the development of precise, potent, programmable and durable therapeutics for genetic diseases that lack disease-modifying therapies.”

RegRNAs act as gene-specific rheostats that can finely modulate gene expression upstream of mRNA transcription, but not to excessive levels that might generate toxicity. CAMP4 leverages its extensive set of proprietary genomic data to efficiently and systematically identify gene-specific regRNA targets and generate precise oligonucleotide drug candidates, or RNA ActuatorsTM, to drug them. This approach is applicable to any genetic disease whereby a small increase in gene output can lead to meaningful therapeutic outcomes.

In primary mouse hepatocytes, RNA Actuators increased Serping1 expression by 2-to-3-fold with minimal impact on neighboring genes. The RNA Actuators also normalized the levels of Serping1 in a HAE mimic in vitro model and led to an increase in Serping1 protein in mice.
(Source: CAMP4 Therapeutics)