CAMP4 Brings $100M to Target the Dark Side of the Genome with regRNA Platform

Nearly one year after securing $45 million to usher in a new era in RNA-based therapeutics, Cambridge, Massachusetts-based CAMP4 Therapeutics raised another $100 million in financing to advance its approach into the “dark side of the genome” and drive two therapeutics into clinical trials next year.

Founded in 2016, CAMP4 Therapeutics has combined its RNA Actuating Platform (RAP) to map regulatory RNA (regRNA) molecules that are associated with every protein-coding gene in any cell type, with cutting-edge oligonucleotide technology in order to develop precise and programmable therapeutics that enable “tunable upregulation” of gene expression to treat disease. The company’s target is the so-called “dark side” of DNA that regulates the small bit of the genome that codes for proteins. Targeting these regRNA molecules creates a pathway to target more than 1,000 different genetic diseases at their core, CAMP4 said.

Josh Mandel-Brehm, chief executive officer of CAMP4, noted that over the past several years, there have been significant advancements in the field of regRNAs and being able to target the dark side molecules. By doing so, CAMP4 can “elegantly control the expression of genes for therapeutic purposes,” he 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 (ASO) 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,” Mandel-Brehm said in a statement.

Proceeds from the Series B financing round will be used to advance the company’s lead programs in central nervous system and liver diseases into the clinic by the middle of 2023. CAMP4’s lead programs are aimed at Dravet syndrome, a genetic form of epilepsy, and urea cycle disorder, which is a set of liver diseases with life-threatening neurological effects. The company anticipates filing an Investigational New Drug Application for its Dravet syndrome asset first, followed by the urea cycle disorder therapeutic toward the end of 2023.

Beyond Dravet syndrome and urea cycle disorders, the company’s preclinical pipeline is also focused on diseases such as primary biliary cholangitis, an autoimmune disease that causes progressive destruction of the bile ducts and frontotemporal dementia, a group of disorders that impacts the frontal and temporal lobes of the brain and causes problems with behavior and language.

In addition to advancing development of the company’s lead assets, the Series B funds will also be used to expand its technology platform, enabling it to build a broad pipeline of RNA actuators, and construct a 5,000 square foot innovation space in Boulder, Colorado that is focused on ASO chemistry optimization. The company also plans to expand its headcount by the end of the year with the hiring of more than 25 employees.

“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,” Mandel-Brehm added.

The $100 million financing round was led by Enavate Sciences, a biotech investment firm that launched earlier this year. It was supported by the Gaingels, an LGBTQIA+/Allies investment syndicate, 5AM Ventures, Polaris Partners, Northpond Ventures, Andreessen Horowitz, The Kraft Group and others.

James Boylan, CEO of Enavate and a new member of the CAMP4 Board of Directors, said there is “immense opportunity and value” in CAMP4’s approach to upregulating gene expression with antisense oligonucleotides.

“Groundbreaking insights into regRNAs, powered by internally derived machine learning algorithms and our ability to drug targets with ASOs (antisense oligonucleotides) 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,” Boylan said in a statement.