CAMP4 Outlines 2025 Milestones: MAD Safety Data in H2, GLP Toxicity Studies for SYNGAP1 Disorders, New GBA1 Discovery for Parkinson's, and Strategic Partnership Expansion.

Benzinga · 01/07 21:11

Expected Milestones in 2025

  • MAD safety, pharmacokinetic and key pharmacodynamic biomarker data in healthy volunteers in the second half of 2025.
  • GLP toxicity studies of lead ASO candidate for neurodevelopmental disorders caused by SYNGAP1 mutations to be initiated this year.
  • Advance a new discovery program targeting a GBA1 regRNA to increase gene expression for the treatment of Parkinson's disease (PD) caused by mutations in GBA1, with potential for application in sporadic PD.
  • Company to focus on expanding its strategic partnerships to continue maximizing the value of its RAP platform.