CAMP4 Therapeutics

CAMP4 Therapeutics, founded in 2016 and based in Cambridge, Massachusetts, is a clinical-stage biotechnology company focused on developing novel RNA-based therapies to restore healthy gene expression. The company's proprietary RNA Actuating Platform (RAP) targets regulatory RNAs (regRNAs) within the non-coding genome to upregulate gene expression and treat genetic diseases. CAMP4's approach uses antisense oligonucleotides (ASOs) to precisely control gene expression, potentially addressing over 1,000 genetic disorders where patients under-express key proteins.

The company's lead program for Dravet syndrome aims to upregulate the SCN1A gene to reduce seizures and preserve neurodevelopmental milestones. CAMP4 expects this candidate to enter clinical trials in mid-2023. Another key program targets urea cycle disorders (UCDs) by upregulating ornithine transcarbamylase (OTC) production to improve ammonia processing. In March 2024, CAMP4 dosed the first participant in a Phase 1 study of CMP-CPS-001, its lead candidate for UCDs. The company is also developing treatments for primary biliary cholangitis and frontotemporal dementia.

CAMP4 occupies approximately 30,000 square feet of lab and office space in Cambridge and employed about 55 people as of August 2022, with plans to expand. The company has raised over USD 180 million in funding, including a USD 100 million Series B round in July 2022 led by Enavate Sciences. CAMP4 utilizes its RAP platform to map regRNAs associated with protein-coding genes and design targeted ASO therapies. The company believes its approach can address haploinsufficiency disorders and recessive diseases by increasing protein production from remaining functional genes.

Key customers and partnerships

In July 2023, Fulcrum Therapeutics entered an exclusive worldwide license agreement with CAMP4 for the rights to develop therapies for Diamond-Blackfan Anemia based on CAMP4's preclinical work. Under this agreement, Fulcrum will pay CAMP4 an undisclosed upfront amount plus up to USD 70 million in milestone payments and tiered royalties on future sales.