Camp4 Therapeutics (CAMP) 25th Annual Needham Virtual Healthcare Conference summary

Platform and scientific approach

• Utilizes antisense oligonucleotides (ASOs) to upregulate gene expression by targeting regulatory RNAs, aiming to treat haploinsufficient diseases with a focus on upregulation rather than downregulation.

• Proprietary mapping of regulatory elements in primary human cells enables identification of optimal ASO binding sites and lead generation, with all data generated internally.

• Chemistry optimization is tailored for CNS delivery, currently using naked oligos delivered intrathecally without conjugates.

Pipeline and lead program

• Lead program targets SYNGAP1-related disorder, a rare genetic epilepsy, with plans to enter the clinic in the second half of the year.

• Early discovery programs focus on other genetic epilepsies, and a deprioritized urea cycle disorder asset is available for partnership.

• Multi-target discovery collaboration with GSK announced, spanning several therapeutic areas.

SYNGAP1 disease overview and patient journey

• SYNGAP1 is a true haploinsufficiency disorder with over 20,000 patients globally, characterized by intellectual disability, seizures, and significant behavioral and sleep challenges.

• No approved disease-modifying therapies exist; management is symptomatic and often involves complex polypharmacy.

• Diagnostic journey has improved with increased awareness and access to genetic testing, reducing time to diagnosis to about a year.