Camp4 Therapeutics (CAMP) Leerink Global Healthcare Conference 2026 summary

Technology platform overview

• Utilizes antisense oligonucleotides (ASOs) to increase gene expression by targeting regulatory RNAs, offering precise upregulation distinct from traditional gene silencing approaches.

• Platform catalogs regulatory RNAs for each gene in any cell type, enabling selective drugging for controlled gene expression increases.

• Achieves a twofold increase in gene expression, ideal for haploinsufficient diseases, with cellular mechanisms preventing excessive overexpression.

• Pharmacokinetics and pharmacodynamics align with established ASO drugs, supporting similar dosing schedules and durability.

• Developed robust assays to distinguish true biological upregulation from noise, critical for this novel therapeutic area.

Program reprioritization and pipeline strategy

• Shifted focus from urea cycle disorder (UCD) to SYNGAP1 due to stronger preclinical data, larger unmet need, and better fit for platform strengths.

• UCD program was well-executed with no safety issues and is now being considered for partnership with interested parties.

• Learnings from UCD, including regulatory and clinical execution, inform ongoing and future programs.

• Internal pipeline prioritizes CNS haploinsufficiencies with high unmet need, while non-core or non-haploinsufficient indications are considered for business development partnerships.

• Plans to announce additional CNS programs similar to SYNGAP1, aiming to build a multi-billion dollar franchise.

SYNGAP1 program details

• SYNGAP1 is a severe, lifelong haploinsufficient disorder with no approved therapies, characterized by cognitive impairment, seizures, and mobility issues.

• Preclinical data show reversal of disease symptoms in humanized mouse models and restoration of gene expression in patient-derived cells.

• Target clinical population is children as young as 2–3 years, focusing initially on the most homogeneous group with truncating mutations.

• Clinical development will use multiple ascending dose (MAD) studies from the outset, supported by regulatory comfort with CNS oligo therapies.

• Natural history studies and collaborations with foundations inform trial design, endpoints, and inclusion criteria.