US regRNA player gets $100m in Series B funding to treat genetic diseases at their core

By Jane Byrne

- Last updated on GMT

© GettyImages/koto_feja
© GettyImages/koto_feja

Related tags genetic diseases Rna CNS Dna Gene expression

US biotech, CAMP4 Therapeutics, has secured US$100m in a Series B financing round that it says will be used to accelerate expansion of its regulatory RNA (regRNA) platform.

The capital will also go towards advancing the Cambridge, Massachusetts company’s lead programs into the clinic.

The financing round was led by Enavate Sciences, with additional investors including a large national managed care organization and the Gaingels, an LGBTQIA+/Allies investment syndicate supporting diversity in venture capital, along with other unnamed investors.

Existing investors such as 5AM Ventures, Polaris Partners, Northpond Ventures, Andreessen Horowitz, The Kraft Group, and others also participated.

The biotech says it has made massive advancements in the field of regRNAs and its ability to target these molecules to “elegantly”​ control the expression of genes for therapeutic purposes.

Through breakthroughs in molecular biology pioneered by CAMP4 co-founder, Dr Rick Young, it said it is now known that the 'dark side of the genome,'​ what was once thought to be ‘junk’ DNA, actually regulates the small sliver of the genome that codes for proteins.

And by targeting these ‘dark side’ molecules - regulatory RNA (regRNA), CAMP4 is able to fine-tune expression of nearby genes, treating certain genetic diseases at their core. 

Josh Mandel-Brehm, CEO of CAMP4, said: “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 1,000 known genetic diseases in which a patient is under-expressing a key protein.”

Disease focus

The company expects to enter the clinic with its lead candidate to treat Dravet syndrome by mid-2023. The financing round will further support the advancement of CAMP4’s drug candidates, including progressing its urea cycle disorder program into the clinic and continuing to build an expansive pipeline of RNA actuators.

Initially focusing on treating diseases of the central nervous system (CNS) and liver, it said its platform also has the potential to address a broad range of genetic indications across multiple tissues, with a focus on haploinsufficient diseases.

CAMP4 said the proceeds of the funding will also support its recruitment drive: it is looking to hire an additional 25 employees this year.

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