Camp4 Therapeutics (CAMP) Leerink Global Healthcare Conference 2026 summary

Technology platform overview

• Utilizes antisense oligonucleotides (ASOs) to upregulate gene expression by targeting regulatory RNAs, specifically enhancers and promoters from the non-coding genome.

• Achieves precise, gene-specific upregulation, typically resulting in a twofold increase, ideal for haploinsufficient diseases.

• Platform enables cataloging of regulatory RNAs for any cell type and has been validated across over 40 target genes.

• Developed robust assays to distinguish true biological upregulation from noise, addressing unique challenges in this field.

• Uses established ASO chemistries with pharmacokinetics and dosing schedules similar to approved drugs like SPINRAZA.

Program reprioritization and learnings

• 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 provided key insights into pharmacokinetics, safety, and regulatory processes, with no safety issues observed.

• Partnership discussions for UCD are ongoing, aiming to find a suitable external partner to advance the program.

• Experience from UCD clinical execution informs future regulatory and operational strategies.

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 protein levels in patient-derived cells.

• Target clinical population is children as young as 2–3 years, focusing initially on the 80% with truncating mutations and high seizure rates.

• 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 are informing trial design and inclusion criteria.