Reg Actuators • CAMP4

Solving problems is in our RNA

The disruption of normal gene expression underlies many human diseases. Restoring gene expression to normal levels could provide a path to treating these diseases. At CAMP4, the solution lies in our RNA.

We are focused on a form of RNA that regulates gene expression and has largely been unexploited as a therapeutic target, until now.

We have built the industry’s only platform for the tunable upregulation of genes. Our approach enables the efficient and systematic identification of gene specific regulatory RNA targets and generates bespoke oligonucleotide drug candidates to reach them.

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In the nucleus, genes and their regulatory elements are organized into conserved 3D DNA structures known as insulated neighborhoods to control gene expression. regRNAs are uniquely transcribed within these loops and act as rheostats for gene expression.

regRNAs Defined

Regulating Gene Expression

Abnormal gene expression – resulting in too much or too little of a certain protein – can cause disease. Many therapies aim to interfere with the disease process by modifying how or when genes are expressed. We are pioneering a new approach.

In all of us, RNA is the messenger between DNA and proteins. Recent discoveries in molecular biology have revealed that RNA is also involved in the regulation of genome organization and gene expression. These regulatory RNAs – or regRNAs – play a crucial role in controlling gene expression. By precisely targeting regRNA, we can increase gene expression in a specific and tunable way.

Adjusting the Output

RNA actuators can open the faucet, increasing regRNA activity to restore levels of mRNA expression.

Vast Frontiers

Upregulating gene expression with intentionally-designed drugs that target regRNA opens up a vast frontier of opportunity to develop truly transformative medicines. Our therapeutic approach is applicable to hundreds of diseases caused by genetic anomalies that can be addressed by increasing the output of a certain gene.

Haploinsufficient Diseases

The Problem

A genetic mutation damages one gene copy, eliminating protein expression

Leading to ~50% of normal protein levels.

CAMP4’s Approach

Upregulate gene expression, causing a 2x increase in protein production

Resulting in near-normal protein levels.

Recessive or Loss-of-Function Diseases

The Problem

Genetic mutations damage both gene copies, reducing protein production

Leading to <25% of normal protein levels.

CAMP4’s Approach

Upregulate expression of the mutated genes or a compensatory gene

Restoring or compensating for sufficient protein expression.

Our initial focus is on diseases of the liver and central nervous system. We believe our platform is applicable to every tissue in the human body and we see vast potential.