Rare Daily Staff

CAMP4 Therapeutics, a therapeutics developer harnessing RNA to restore healthy gene expression, completed a  $45 million financing to fund the expansion of its platform and advance multiple preclinical RNA therapies into human testing.

CAMP4 is combining its proprietary RNA Actuating Platform (RAP) with oligonucleotide technology to develop precise and programmable therapeutics that enable tunable upregulation of gene expression to treat disease. The company’s approach 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.

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.

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 application to begin human clinical studies by late next year. It said its approach 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 while overcoming significant manufacturing limitations of gene therapy.

As part of the financing, two new members, Andy Schwab, managing partner of 5AM Ventures and Shaan

Gandhi, director of Northpond Ventures, will join CAMP4’s board of directors.

Photo: Josh Mandel-Brehm, CEO of CAMP4

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