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See the 18-slide pitch deck a genetics startup used to raise $100 million and explore the ‘dark side’ of the genome
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- The genetics startup CAMP4 Therapeutics just raised a $100 million Series B round.
- The company hopes to treat disease by drugging a neglected area of the human genome.
- The Massachusetts biotech aims to start human testing in 2023.
A biotech in Cambridge, Massachusetts, raised $100 million to explore what its leaders call the “dark side” of the genome.
On Wednesday, CAMP4 Therapeutics announced its Series B round, which it said would help it start initial human testing next year. The startup, named after the camp closest to Mount Everest’s summit, was founded in 2016 and is researching a special type of RNA called regulatory RNA.
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There are multiple types of RNA. The best-known type is messenger RNA — the basis of the Pfizer and Moderna COVID-19 vaccines — which acts as a courier within the cell to carry instructions about how to make proteins from DNA.
Regulatory RNA, or regRNA, is part of the 98% of the human genome sequence that doesn’t make proteins, said David Bumcrot, CAMP4’s chief scientific officer. Instead, these molecules control the expression of the protein-making genes.
Building off research from Rick Young, an MIT researcher who cofounded CAMP4, the company is shining a light on this neglected part of the genome, its leaders said. The company plans to design drugs to bind to regRNA, effectively controlling how much of a protein is made.
Its CEO, Josh Mandel-Brehm, says the company’s platform can be applied to more than 1,000 genetic diseases. The startup hopes to ask regulators in early 2023 to start human testing for its lead drug candidate: a therapy for Dravet syndrome, a rare genetic disorder. Mandel-Brehm said that CAMP4 is also prioritizing partnerships and that early talks are ongoing with Big Pharma.
“Now that this fundraise is in the bank, we are ready to partner and we are absolutely focused on putting at least one major partnership in place,” he said, adding that he hopes to strike a partnership deal in the next six to nine months.
See the 18-slide pitch deck CAMP4 used to raise money from VCs including Patient Square Capital, Andreessen Horowitz, and 5AM Ventures.
CAMP4 is designing drugs that target regRNA molecules. The startup hopes to treat genetic diseases with this approach.
CAMP4 Therapeutics
To do this, the company uses artificial intelligence and genetic sequencing to map out which regRNAs interact with specific genes.
CAMP4 Therapeutics
These regRNAs help determine which genes are switched on and off within the body.
CAMP4 Therapeutics
CAMP4 is focused on diseases that occur because only one gene is producing proteins; healthy people have two working copies of each gene.
CAMP4 Therapeutics
While CAMP4 hasn’t started human testing, it says it’s completed test-tube and animal studies that support its regRNA research.
CAMP4 Therapeutics
CAMP4 is led by Mandel-Brehm, a Biogen veteran, and Bumcrot, who worked at the genetic pioneers Alnylam Pharmaceuticals and Editas Medicine.
CAMP4 Therapeutics
“For any gene that you are interested in regulating, our platform tells you this is where the regulatory RNAs are going to come from,” Bumcrot told Insider.
CAMP4 Therapeutics
Transcription factors are special proteins that turn genes on and off. RegRNAs play a key role in binding to nearly half of these proteins, CAMP4 says.
CAMP4 Therapeutics
CAMP4 is using antisense oligonucleotides, or ASOs, to bind to regRNA molecules. There are several approved ASO-based medicines on the market, such as Spinraza, Biogen’s spinal-muscular-atrophy drug.
CAMP4 Therapeutics
In addition to Dravet syndrome, the company is researching how to treat other diseases in the nervous system and the liver.
CAMP4 Therapeutics
There is no cure for Dravet syndrome, which causes routine seizures that typically start within the first few months of life.
CAMP4 Therapeutics
Most Dravet cases are caused by a mutation in one of the two copies of the SCN1A gene. CAMP4 is hoping its approach can increase protein production from the functional SCN1A gene.
CAMP4 Therapeutics
CAMP4 says that to produce more SCN1A protein it hopes to modify regRNA to “remove the breaks” on the working gene. That fix could make up for the nonworking copy of the SCN1A gene.
CAMP4 Therapeutics
CAMP4 says its research suggests its approach led to significantly fewer seizures in mice.
CAMP4 Therapeutics
CAMP4 has also tested this approach in monkeys — it says primates given the experimental drug had higher levels of the SCN1A protein.
CAMP4 Therapeutics
CAMP4 estimates its Dravet drug could be a $1 billion-plus market opportunity.
CAMP4 Therapeutics
The research firm PitchBook indicates that after this Series B raise, CAMP4 has a valuation of $280 million. Mandel-Brehm declined to comment on the valuation.
CAMP4 Therapeutics
While CAMP4’s initial research focus is on liver and central-nervous-system diseases, Mandel-Brehm said the team would eventually study other diseases. “We’re applying our platform beyond just the tissues where you can safely deliver today, because we’re betting on delivery getting solved,” he said.
CAMP4 Therapeutics