CAMP4 harnesses regulatory RNAs (regRNAs) and a proprietary RNA Actuating Platform (RAP™) to map regRNAs controlling protein-coding genes and program antisense oligonucleotides (ASOs) to increase gene expression. Its approach targets the “dark side” of the genome to upregulate gene output, aiming to treat diseases where increased protein expression is therapeutic, with a focus on haploinsufficient disorders. The company is initially concentrating on central nervous system and liver indications. CAMP4 expects to enter the clinic with a lead candidate for Dravet syndrome by mid-2023 and plans to progress a urea cycle disorder program into the clinic. The platform is positioned to build an expansive pipeline of RNA actuators across multiple tissues. The company announced a $100 million Series B to accelerate advancement of its lead programs and expansion of its regRNA Actuating Platform. CAMP4 combines a proprietary RNA Actuating Platform (RAP) with oligonucleotide technology to create programmable therapeutics called RNA Actuators that tunably increase gene expression. The company focuses on targeting cis-acting regulatory RNAs (regRNAs) to adjust transcriptional output for diseases where modest increases in gene expression are therapeutic. CAMP4 has used next-generation sequencing and AI to map transcriptional machinery across tissues including liver, muscle, brain and heart to identify gene-specific regulatory RNA targets. Its current pipeline prioritizes indications where oligonucleotides have proven safe and effective, notably CNS and liver diseases, with potential expansion to muscle and heart. CAMP4 expects to file an Investigational New Drug application by late next year and is advancing multiple preclinical RNA therapies toward human testing. The company raised $45 million to expand its platform and accelerate those programs; it was founded in 2016. Camp4 Therapeutics has developed a Gene Circuitry Platform that amplifies the value of cellular and genetic insights to understand how genes are controlled by signaling pathways in specific disease states. The platform generates proprietary 4D maps to identify de-risked druggable targets, produce actionable insights and improve therapeutic predictability. The company says this approach could potentially address hundreds of diseases and benefit millions of patients globally. Camp4 was co-founded in 2016 by Dr. Richard Young and Dr. Leonard Zon and is led by CEO Josh Mandel-Brehm. The company is based in Cambridge, MA. It raised financing to expand its development efforts.

Total raised

$175M

Funding rounds

3

Latest round

Series B

Latest activity

Jul 2022