Targeting the Central cAMP Signaling Defect in ADPKD to Advance a Genotype-Agnostic Small-Molecule Therapy into the Clinic
9:30 am - Tuesday 16th March 2027
- Directly targeting abnormally elevated cAMP through Mironid’s first-in-class LoAc small molecules to intervene in a central driver of cyst formation and growth
- Building on preclinical evidence supporting reductions in cyst formation and kidney enlargement to translate PDE4- mediated cAMP modulation into a clinical, orally available treatment strategy for ADPKD
- Advancing a genotype-agnostic approach designed for patients across PKD1, PKD2 and other genetic backgrounds to broaden therapeutic reach and complement increasingly mutation-specific genetic and RNA-based approaches emerging across the ADPKD pipeline