ADPKD Research Research watch

ADPKD cure research, clinical trials, and new evidence

Track real movement without overselling early signals.

Last reviewed: June 1, 2026

Cure research

  • Gene editing/therapy correction of causal variants (PKD1/PKD2)
  • RNA-targeted correction/silencing strategies
  • Cell/organ regenerative replacement concepts
  • Multi-pathway disease interception (beyond single-target)

Latest meaningful movement

  • Key new evidence this run , GLPG2737 / CFTR inhibition failed in the phase 2a MANGROVE study: it did not reduce htTKV growth or slow eGFR decline versus placebo in rapidly progressive ADPKD (PMID42201338).
  • Four-track review outcome
  • What strengthened , The negative CFTR result cleaned up a weak hypothesis.