Maps tell you where things are and how to get there. CAMP4 Therapeutics is using maps of human cells, created by its computational technology, to find its way to new drugs. The biotech startup is still preclinical, but perhaps not for long. It now has $45 million to help bring its RNA therapies closer to their first tests in humans.
The financing was led by Northpond Ventures and 5AM Ventures.
Cambridge, Massachusetts-based CAMP4, which takes its name from the final camp before mountaineers summit Mount Everest, builds its maps of human cells with next-generation sequencing techniques and artificial intelligence. These maps show signaling pathways that control the expression of genes.
A map can tell you where to go but it doesn’t necessarily tell you the means to get there. CAMP4 first tried small molecules. That approach led to too many things happening when the company was trying to achieve a specific therapeutic effect, said CEO Josh Mandel-Brehm. So CAMP4 regrouped, and turned to Richard Young, the MIT biology professor who cofounded the startup. Young had newer understanding of biology from his RNA research.
The convergence of CAMP4’s maps and Young’s insights brought the company to regulatory RNA (regRNA). Unlike the messenger RNAs that encode proteins, regRNAs acts to precisely control a gene, similar to how a rheostat adjusts an electrical current, Mandel-Brehm said. This approach can dial up a gene’s expression, but not so much that it causes toxic effects. CAMP4 aims to harness this mechanism for therapeutic applications by addressing haploinsufficient diseases, which are disorders in which one gene of a gene pair is not functioning properly.
“If you were to increase gene [expression] by twofold, you would theoretically move back to a normal state,” Mandel-Brehm said. “Even a small increase can have a meaningful impact in these diseases.”
CAMP4 calls its drugs RNA actuators. They’re made from RNA molecules called oligonucleotides. We already have oligonucleotide drugs. Biogen drug Spinraza treats spinal muscular atrophy by getting a gene to increase the production of a protein at the root of the rare disorder. Another biotech, Stoke Therapeutics, is also developing oligonucleotide drugs for haploinsufficiencies. But that biotech’s drugs target mRNA.
There are an estimated 650 haploinsufficient diseases. To narrow things down, CAMP4 is focusing on haploinsufficiencies of the liver and brain, both rare and common disorders, Mandel-Brehm said. The liver and the central nervous system are also areas that scientists know can be targeted by oligonucleotides, he added. So far, CAMP4 has done some animal testing. With the new funding, the company will continue the preclinical research leading up to an application to begin clinical testing. The goal is to file two investigational new drug applications next year, one for a liver disease and the other for a CNS disorder.
The funding will support CAMP4 through the end of 2022. By then the company will have more data, and perhaps collaborations with larger companies. CAMP4 partnered with Biogen last year, an alliance that dissolved after the startup’s initial small molecule research didn’t pan out. Mandel-Brehm said that if CAMP4 can show its RNA therapies work, the company will want to position the technology in a way it can be used more broadly.
CAMP4 was founded in 2016 based on the research of Young and Leonard Zon, director of the stem cell program at Boston Children’s Hospital and a professor at Harvard Medical School. The startup emerged from stealth two years later with a $30 million Series A round of funding. The latest capital is what Mandel-Brehm calls a Series A prime financing, which brings Northpond and 5AM alongside the company’s earlier investors. Those investors— Andreessen Horowitz, Polaris Partners and The Kraft Group—also participated in the latest financing.
The next stop on CAMP4’s journey could be a much larger financing round to support clinical testing, and then perhaps a move to the public markets. Mandel-Brehm said this next capital raise could be a crossover round, an investment that includes firms that back both private and public companies. Crossover rounds are one of the final steps toward an IPO, though Mandel-Brehm added that in today’s financing environment, a biotech startup has many different options.
“To do what we’re doing, we’re going to need access to more capital to deliver on the promise,” he said.
Photo by CAMP4 Therapeutics