- Proceeds to Support Clinical Development of Lead CNS & Liver Programs; IND for Dravet Syndrome is Anticipated by Mid-2023

- New Capital Will Also Fuel Expansion of CAMP4’s regRNA Actuating Platform™, Harnessing the Power of Regulatory RNA to Upregulate Gene Expression

- Funding Round Led by Enavate Sciences, a Portfolio Company of Patient Square Capital; Enavate CEO Jim Boylan Joins CAMP4’s Board of Directors

CAMP4 Therapeutics, a biotechnology company harnessing the power of RNA to restore healthy protein expression, today announced the closing of a $100 million Series B round to advance the company’s lead regRNA programs and accelerate the expansion of the company’s regRNA Actuating Platform. 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% 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,” says 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 haploinsufficient 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, Chief Executive Officer 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. We’re excited to lead this financing and partner with CAMP4 to help realize the full potential of RNA actuators for patients with genetic diseases.”

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

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.

About Enavate Sciences

Enavate Sciences is a platform created by Patient Square Capital dedicated to supporting therapeutic companies advancing medicines and enabling technologies with transformative potential to address patient need. Through the application of capital support and operational experience, Enavate strives to enable and empower a diverse portfolio of therapeutics companies to accelerate innovation. To learn more about Enavate, please visit www.enavatesciences.com.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220720005150/en/

"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," says Josh Mandel-Brehm, CEO.