CAMP4 Therapeutics closes $100m round to advance lead regRNA programmes

The funding round was led by Enavate Sciences, a portfolio company created by Patient Square Capital.

The round also saw participation of Gaingels, along with other undisclosed investors.

Existing investors, such as Polaris Partners, 5AM Ventures, Andreessen Horowitz, Northpond Ventures, The Kraft Group, took part in the Series B round.

The biotechnology company’s approach targets regulatory RNAs (regRNAs), which is seen as part of the ‘Dark Side of the Genome’, the 98% of genome without encoding proteins.

These regRNAs control the expression of closeby protein-encoding genes.

Mapping regRNAs related with each protein-coding gene in any cell kind, the CAMP4’s RNA Actuating Platform’s (RAP) programmable antisense oligonucleotide (ASO) therapeutics target the controlling regRNAs to upregulate gene expression to treat disease.

This process is applicable to various genetic diseases in which tunable increases in gene output can lead to therapeutic results.

CAMP4 CEO Josh Mandel-Brehm said: “We have made massive advancements in the field of regRNAs and our ability to target these ‘dark side’ molecules to elegantly control the expression of genes for therapeutic purposes. For any disease-associated gene, we can identify the regRNA controlling it with our proprietary RAP platform and then rapidly develop an antisense oligonucleotide to precisely increase gene output.

“This is an efficient, repeatable approach for more than a thousand known genetic diseases in which a patient is under-expressing a key protein. The incredible support we’ve received in our Series B round is a testament to the promise and vast potential of our transformative regulatory RNA platform and the impact it could have for patients with genetic diseases.”

The firm expects to enter the clinic with its lead candidate for the treatment of Dravet syndrome by mid of next year.

The funding round will bolster the progress of CAMP4’s drug candidates, including advancing its urea cycle disorder programme into the clinic and continuing to build a pipeline of RNA actuators.

Although the firm is initially concentrating on treating diseases of the central nervous system and liver, its platform has the potential to tackle a wide range of genetic indications across several tissues, with a focus on haploinsufficient diseases.

Enavate Sciences CEO James Boylan said: “We see immense opportunity and value in CAMP4’s truly unique approach of upregulating gene expression using antisense oligonucleotides, a proven modality for regulating gene expression.

“Groundbreaking insights into regRNAs, powered by internally-derived machine learning algorithms, and our ability to drug targets with ASOs have merged together in CAMP4’s proprietary platform to advance an entirely new class of medicines. We’re excited to lead this financing and partner with CAMP4 to help realise the full potential of RNA actuators for patients with genetic diseases.”

Along with the financing, Boylan will join CAMP4’s board of directors.