Targeting the BFPs of the CNS, Biogen licenses map of genetic neighborhoods in the brain
Searching for new methods of attacking serious central nervous system diseases, Biogen has signed a deal that could be run into the hundreds of millions of dollars to essentially license a transcriptional map of the brain and scan it for new drug targets.
The partnership, worth $15 million upfront and far more in milestones, is with a new bioinformatics startup called CAMP4 Therapeutics. Founded in 2018 year by the Whitehead Institute’s Richard Young and Harvard Medical School’s Leonard Zon, CAMP4 takes genes considered affiliated with a disease and maps out the various ways cells express those genes and turn them on or off. They then take that map and – in the biological equivalent of stretching a war map across a table in a bunker — mark up all the different methods of attack.
“The more we know about a gene, it doesn’t mean we can drug that gene,” CEO Josh Mandel-Brehm, who worked at Biogen before joining CAMP4, told Endpoints News. “Many genes are not quote-un-quote druggable, so then we have an interesting problem: I think I understand the genetics of the disease, but how do I drug it? And that’s where our map solves for this problem.”
Alongside the $15 million upfront payment, CAMP4 will be eligible for up to $96 million in milestones for each of the initial targets it supplies, and up to $173 million for each subsequent one.
Biogen is not disclosing what genes or diseases they will target, but the collaboration will focus on microglial cells. These macrophages are found in the central nervous system and have long been considered a key factor in neurodegenerative diseases such as Alzheimer’s and Parkinson’s.
Biogen will bring CAMP4 the genes it believes are central to diseases in the central nervous system, and CAMP4 will inform them all the ways the gene is being expressed and where Biogen might go about turning up or down the expression.
“So you say ‘hey I want to move this particular gene for this disease, how should I do that? I want to change the expression of it,'” Mandel-Brehm said, describing how they work with partners. “We’ll show you the different ‘nodes,’ if you will: ‘hey you can drug this, you can drug this, you can drug this.’ It’s modality agnostic.”
CAMP4, founded out of Polaris Partners, is one of a couple of new biotechs that build on a discovery Young had back in 2015. The longtime transcriptional biologist published work showing that cells modify gene expression using a series of “insulated neighborhoods,” 3-D loops of DNA that control gene expression within them. CAMP4 maps those (along with other aspects of the transcriptome). Flagship Pioneering’s new Omega Therapeutics also tries to drug them.
Since launching in 2018, CAMP4 has done extensive work on mapping genes for liver diseases, including NASH, as part of an in-house development program. They’ve also partnered with synthetic RNAi company Alnylam and are working to expand their maps of cells in the brain, heart, muscles, immune system, blood and kidneys, which they could then license out or use to develop drugs in-house.
“The way I think about the diseases we’re going after is BFP: Big Fucking Problems,” Mandel-Brehm said, citing NASH and other diseases that lack effective treatments. And “It’s well-validated and understood: If you’re drugging something that’s tied to the gene, you’re much more likely to be successful.”