Use of CFTR Modulators for the Treatment of ADPKD

Case ID:
Disclosure Date:
Unmet Need
Autosomal dominant polycystic kidney disease (ADPKD) is characterized by progressive enlargement of multiple renal cysts that leads to hypertension, a decline in renal function, and renal failure. Currently no treatment exists that can prevent renal failure from ADPKD.

Technology Overview:
Johns Hopkins researchers have found that modulators of the Cystic fibrosis transmembrane conductance regulator (CFTR), a target currently used for the treatment of cystic fibrosis, also inhibits cyst growth. Proof of concept in relevant mouse models showed an 80% reduction in cyst growth in polycystin 1 null mice and in mouse derived, proximal tubule cells after compound administration. Other indices of ADPKD such as cAMP levels and forskolin-dependent stimulation of adenylyl cyclase were also lowered after treatment. The mechanism of action occurs via a reduction in heat shock factors that fuel cyst growth and redistribution of transporters to promote fluid absorption.

Stage of Development:
All studies were performed in an ADPKD mouse model and in mouse derived cell lines.

Principle Inventor:
Liudmila Cebotaru, William Guggino.
Patent Status:
PCT application filed 6/21/2018

Yanda et al. Journal of Biological Chemistry, (2018).
Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
New Strategy of Treating ADPKD with CFTR Derived Drug VX 809 and Similar Compounds PRO: Provisional United States 62/676,674 5/25/2018     Pending
Cystic Fibrosis Transmembrane Conductance Regulator Modulators for Treating Autosomal Dominant Polycystic Kidney Disease PCT: Patent Cooperation Treaty PCT PCT/US2018/038806   6/21/2018     Pending
For Information, Contact:
Vera Sampels
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