Camp4 Therapeutics (CAMP) Chardan's 9th Annual Genetic Medicines Conference summary

Advances in non-coding RNA research

• Non-coding RNAs, once considered genomic 'dark matter,' are now recognized as key regulators of gene expression, genome organization, and protein function.

• Regulatory RNAs (regRNAs) form three-dimensional structures and act locally within gene loci, serving as landing pads for transcription factors and influencing gene transcription.

• Long non-coding RNAs (lncRNAs) can control cell states and are implicated in diseases such as cardiac fibrosis and various cancers.

Platform technologies and target identification

• Systematic platforms use functional genomics, computational tools, and AI/ML to map enhancer-gene relationships and identify disease-driving non-coding RNAs.

• Platforms enable the design of highly specific antisense oligonucleotides (ASOs) or small molecules to modulate gene expression or disrupt RNA-protein interactions.

• Multimodal approaches allow rapid identification and validation of targets, with some platforms achieving candidate nomination in under nine months.

Preclinical and clinical progress

• Robust preclinical data in patient-derived cells and animal models show upregulation of target genes and reversal of disease phenotypes, such as in SYNGAP1-related disorders and cardiac fibrosis.

• Intrathecal delivery of ASOs in non-human primates demonstrated effective gene upregulation and favorable safety profiles.

• Early clinical studies in healthy volunteers have established safety and pharmacodynamic properties, informing future patient trials.