CAMP4 is combining its proprietary RNA Actuating Platform with state-of-the-art oligonucleotide technology to develop precise and programmable therapeutics that enable tunable upregulation of gene expression to treat disease.
CAMP4's approach uniquely targets a new class of RNA known as regulatory RNAs that control the expression of proteins, making this approach applicable to any genetic disease whereby a small increase in gene output can lead to meaningful therapeutic outcomes.
5AM Ventures and Northpond Ventures led the financing alongside existing investors Andreessen Horowitz, Polaris Partners and The Kraft Group.
Since the company's founding in 2016, CAMP4 has used next-generation sequencing techniques fueled by AI to map the transcriptional machinery of human cells across a variety of tissues including liver, muscle, brain and heart.
These tissues underpin hundreds of well-characterized genetic haploinsufficient diseases whereby one gene does not function properly, and, in many cases, a successful therapy needs to reliably increase gene output within a narrow range.
Insights gleaned from the initial drug discovery work and recent seminal findings by CAMP4's founder Richard Young, Ph.D. (Whitehead Institute/MIT) and others in the newly emerging field of cis-acting gene regulatory RNAs revealed a superior approach to generating specific and tunable gene control.
Their studies revealed the power of RNA to act as a specific rheostat in a gene-specific manner that can reliably tune up gene expression upstream of mRNA transcription, but not to excessive levels that might generate toxicity.
CAMP4 leverages its extensive genomic data to efficiently and systematically identify gene-specific regulatory RNA targets and generate precise oligonucleotide drug candidates, or RNA Actuators, to drug them. CAMP4's current pipeline is focused on diseases where oligonucleotides have proven safe and effective, including CNS and liver diseases, with the potential to expand to muscle and heart diseases.
The company expects to file an Investigational New Drug application by late next year.
Drugging regRNAs using RNA Actuators offers potential therapeutic benefits over current gene modulating approaches, including adjustable dosing, reduced risk of off-target effects and the opportunity to address a broader range of diseases, in addition to overcoming significant manufacturing limitations of gene therapy.
As part of the financing, two new members, Andy Schwab, Managing Partner, 5AM Ventures and Shaan Gandhi, M.D., D. Phil., director, Northpond Ventures, will join CAMP4's board of directors.
At CAMP4, we are pioneering a novel approach to programmable therapeutics. We combine a deep understanding of regulatory RNA and gene expression with a complementary and customizable oligonucleotide modality.
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