Camp4 Therapeutics (CAMP) investor relations material

Platform overview and scientific approach

• Focuses on regulatory RNAs (regRNAs) that control gene expression via promoters and enhancers, enabling targeted upregulation for haploinsufficiency disorders.

• Antisense oligonucleotides (ASOs) are designed to bind regRNAs, increasing protein expression by about twofold, ideal for diseases with partial gene loss.

• Platform allows rapid mapping and analytical assessment of gene regulation in any cell type, expediting candidate selection.

• Efficient ASO design enables quick progression from discovery to development for restoring missing proteins.

Lead program: Syngap1-related disorders (CMP002)

• 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, with about 10,000 diagnosed in the US and similar numbers globally.

• Diagnosis now relies on genetic epilepsy panels, with improved awareness and patient registries aiding identification.

• Preclinical data show full protein recovery in patient-derived cells and reversal of cognitive symptoms in humanized mouse models.

• GLP-tox studies began in Q3; clinical entry expected in the second half of next year, with regulatory preparations underway.

Pipeline and CNS focus

• CNS haploinsufficiencies, especially developmental and epileptic encephalopathies (DEEs), are the primary focus due to the platform's allele-agnostic upregulation capability.

• Additional CNS targets are under consideration, with Syngap1 as the cornerstone of a broader DEE franchise.

• Opportunities exist beyond rare diseases, with CNS offering a rich pool of potential targets.