Science

regRNA

Every Protein Counts

More than a thousand rare and non-rare diseases are caused by genetic changes that prevent the body from making enough of a critical protein, leading to potentially life-threatening complications.

For these diseases, even a modest increase in protein production has the potential to profoundly impact the lives of patients. Yet today, many of these patients rely on medicines that fail to address the underlying cause of their disease.

At CAMP4, the solution lies in our RNA

RNA plays a central role in gene expression, acting as the essential messenger that translates DNA instructions into functional proteins.

Recent discoveries in molecular biology have revealed a new class of RNA, called regulatory RNA (regRNA), that controls protein production. We have built a comprehensive platform to identify and characterize virtually every regRNA that regulates a protein-coding gene in the human genome.

This foundational insight enables us to rapidly develop antisense oligonucleotides (ASOs) that selectively target specific regRNAs to precisely increase the expression of deficient proteins, moving quickly from target identification to therapeutic candidates designed to amplify protein production and tackle the root cause of diease.

CAMP4 has developed the industry’s most advanced platform capable of systematically mapping regRNAs across the genome and has generated a catalog of tens of thousands of regRNAs from key cell types underlying human disease states.

Illustration of how regulatory RNAs (regRNAs) and transcription factors regulate gene transcription inside the nucleus. A DNA strand extends from the nucleus and contains an enhancer region, a promoter region, and a nearby labeled target gene. An RNA polymerase II complex sits at the promoter, producing an mRNA transcript from the target gene. A central magnified inset shows an active enhancer where regRNA is transcribed and physically associated with RNA polymerase II, an activator protein, and a repressor protein, with arrows indicating dynamic interactions among them. Two numbered captions state that (1) regRNAs originate from enhancers and promoters of actively transcribed protein‑coding genes, and (2) activating and repressive transcription factors bind to enhancers, promoters, and regRNAs to control gene transcription.

Learn About Our Platform

Opening the aperture for diseases of haploinsufficiency

Our RAP Platform^® is designed to address genetic diseases where a modest upregulation of protein levels could yield meaningful therapeutic benefit. This includes haploinsufficiency disorders, where mutation of one functional gene copy results in a 50% reduction in protein levels. By upregulating expression of the remaining functional gene, we aim to restore protein levels to the therapeutic range.

Haploinsufficient Diseases

The Problem

A genetic mutation causes one gene allele to stop functioning

Leading to ~50% of normal protein expression

CAMP4’s Approach

Upgregulating the healthy allele, generating up to a 2x increase in protein production

Resulting in near-normal protein levels

Learn About Our Pipeline