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Camp4 Therapeutics (CAMP) investor relations material
Camp4 Therapeutics Piper Sandler 37th Annual Healthcare Conference summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Piper Sandler 37th Annual Healthcare Conference summary3 Dec, 2025
Platform overview and scientific approach
Focuses on regulatory RNAs (regRNAs) that control gene expression via promoters and enhancers.
Drugging specific regRNAs with antisense oligonucleotides can upregulate protein-coding genes, ideal for haploinsufficiency disorders.
Platform enables efficient mapping and analytical targeting of gene regulation in any cell type.
Antisense oligonucleotides allow rapid development of candidates to restore missing proteins.
Lead program: SYNGAP1-related disorders
SYNGAP1 is critical for synaptic function; deficiency leads to seizures, learning disabilities, and motor issues.
Most patients have haploinsufficient mutations, producing only 50% of normal protein levels.
Estimated over 10,000 patients in the U.S. and a similar number globally.
Diagnosis now more streamlined due to genetic panels and patient advocacy efforts.
Preclinical and clinical progress
Demonstrated full recovery of SYNGAP protein in patient-derived cells and reversal of cognitive symptoms in humanized mouse models.
Achieved dose-responsive, significant increases in SYNGAP in critical brain regions in primates.
GLP-tox studies for CMP-002 began in Q3; clinical entry expected in the second half of next year.
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Next Camp4 Therapeutics earnings date
Q4 202527 Mar, 2026
Next Camp4 Therapeutics earnings date
Q4 202527 Mar, 2026
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Frequently asked questions
Camp4 Therapeutics Corp is a biotechnology company focused on developing RNA-based therapeutics. The company leverages a proprietary platform to identify and regulate gene expression through RNA technologies, aiming to address a range of diseases driven by dysregulated gene activity. Camp4 Therapeutics' research and development activities span multiple therapeutic areas, applying its platform to discover potential treatments targeting specific genetic mechanisms. The company is headquartered in Cambridge, Massachusetts, and its shares are listed on the NASDAQ.
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