Camp4 Therapeutics (CAMP) Piper Sandler 37th Annual Healthcare Conference summary

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.