The Problem
A genetic mutation causes one gene allele to stop functioning
Leading to ~50% of normal protein expression
Solving problems is in our RNA
Genetic changes leading to sub-optimal protein levels underlie many human diseases. Amplifying mRNA, leading to increased production of healthy protein, can lead to meaningful therapeutic benefits for hundreds of diseases. At CAMP4, the solution lies in our RNA.
We are focused on a form of RNA that regulates gene expression and has been unexploited as a therapeutic target, until now: regRNA.
We have built the industry’s only platform to map every regRNA for the tunable amplification of genes. Our approach enables the efficient and systematic creation of antisense drug candidates to control any regRNA.
regRNAs Defined
Regulating Gene Expression
We are pioneering a new approach to restore healthy protein levels.
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.
regRNAs are transcribed from promoters and enhancers, the regulatory elements of the genome which come together at every actively expressed gene. By controlling the local concentration of transcriptional regulatory proteins, regRNAs function as rheostats to specifically control the level of every gene.
Vast Frontiers
Amplifying mRNA with programmable ASO drugs has the potential to be game-changing for hundreds of genetic diseases. Our therapeutic approach is applicable to any disease where increasing protein expression is beneficial.
Haploinsufficient Diseases
CAMP4's Approach
Upregulate the healthy allele, generating a 2x increase in protein production
Resulting in near-normal protein levels
Recessive or Loss-of-Function Diseases
The Problem
A genetic mutation causes both alleles to function poorly or not at all Leading to <25% of normal protein expression
CAMP4's Approach
Upregulate either the mutated gene with residual function or a gene with compensatory function, generating a 2x increase in protein expression
Resulting in near-normal protein levels
Our initial focus is on diseases of the liver and central nervous system, where proven technology exists to deliver antisense therapies to those cells and tissues. Beyond that, we see potential in heart, muscle, lung, kidney and eye diseases.