- Proceeds to fuel expansion of RNA Actuating Platform™ designed to harness the power of regulatory RNA to restore healthy gene expression

- Company advancing preclinical pipeline initially focused on CNS and liver diseases; first IND expected in 2022

- Funding led by 5AM Ventures and Northpond Ventures, alongside existing investors Polaris Partners, Andreessen Horowitz, The Kraft Group and others

CAMP4 Therapeutics, a biotechnology company harnessing the power of RNA to restore healthy gene expression, today announced that it has raised $45 million to propel the next phase of its scientific strategy, significantly expand its platform and advance multiple preclinical RNA therapies into human testing. CAMP4 is combining its proprietary RNA Actuating Platform (RAP) 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 (“regRNAs”) 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.

“Over the last few years, we’ve been quietly learning a great deal about the power of regRNAs to precisely influence gene expression and we are now using these insights, combined with a powerful oligonucleotide modality, to develop truly transformative medicines in a way that’s never been done before,” said Josh Mandel-Brehm, CEO of CAMP4. “Our new and current investors share our enthusiasm for the science and possibilities before us; their support forms the foundation of our efforts to build the industry’s only platform for directly upregulating gene expression at the transcriptional level and rapidly advancing a pipeline of precise, potent and durable therapeutics that can be programmed to meet the exact needs of genetic diseases.”

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^TM, 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.

“Upregulation of gene expression holds immense potential, both in terms of the impact on patients’ lives and the vast number of diseases that can be addressed using this approach,” said Mr. Schwab. “Previous methods of achieving this goal have faced significant limitations. We believe CAMP4’s approach, using a precise, proven and customizable oligonucleotide technology to target regulatory RNA, can succeed in bringing valuable new medicines to treat a range of diseases.”

Dr. Gandhi added: “The biology underpinning CAMP4’s scientific strategy is quite exciting and largely unexploited territory in terms of drug development. Plus, the technology is highly scalable. We are excited to support the CAMP4 team in creating new therapeutic approaches for patients who currently have few options available to help them.”

About CAMP4 Therapeutics

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. Our RNA Actuating Platform’s proprietary insights enable us to harness the power of RNA to upregulate the expression of genes and unlock the potential to create treatments for hundreds of diseases affecting millions of patients. Learn more about us at www.CAMP4tx.com and follow us @CAMP4tx.

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