RARE Daily

CAMP4 Raises $100 Million to Accelerate Expansion of Regulatory RNA-Targeting Platform and Advance Rare Disease Pipeline

July 20, 2022

CAMP4 Therapeutics said it closed a $100 million series B round to advance the company’s lead regRNA programs and accelerate the expansion of its regRNA Actuating Platform.

Photo: Josh Mandel-Brehm, CEO of CAMP4

Enavate Sciences, a portfolio company created by Patient Square Capital, led the round. Additional investors in the series B financing include 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 5AM Ventures, Polaris Partners, Northpond Ventures, Andreessen Horowitz, The Kraft Group, and others also joined the round.

CAMP4’s approach uniquely targets regulatory RNAs (regRNAs), considered to be part of the “Dark Side of the Genome,” the 98 percent of our genome that does not encode proteins. Through breakthroughs in molecular biology, it is now known that regRNAs control the expression of nearby protein-encoding genes. CAMP4’s RNA Actuating Platform (RAP) maps regRNAs associated with every protein-coding gene in any cell type, and its programmable antisense oligonucleotide (ASO) therapeutics target the controlling regRNAs to upregulate gene expression to treat disease. This approach is applicable to a range of genetic diseases in which tunable increases in gene output can lead to meaningful therapeutic outcomes.

“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,” said Josh Mandel-Brehm, CEO of CAMP4. “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 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. While the company is initially focusing on treating diseases of the central nervous system and liver, its platform has the potential to address a broad range of genetic indications across multiple tissues, with a focus on haplo-insufficient diseases.

“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,” says James Boylan, CEO of Enavate Sciences. “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.”

In conjunction with the financing, Boylan will join CAMP4’s Board of Directors.

Author: Rare Daily Staff

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