Cystic Fibrosis Transmembrane Conductance Regulator Modulators for Treating Autosomal Resessive Polycystic Kidney Disease

Case ID:
C16640
Disclosure Date:
11/6/2020

Unmet Need / Invention Novelty: Autosomal recessive polycystic kidney disease (ARPKD) patients that survive beyond neonatal age face various comorbidities that progress into chronic liver disease and end stage kidney disease. There is no cure for ARPKD and current treatments are limited to supportive treatments that manage symptoms and preserve kidney and liver function. There therefore exists an unmet need to develop a treatment for ARPKD.

 

Technical Details: Johns Hopkins researchers have discovered a potential strategy for the novel treatment of ARPKD involving repurposing modulators currently used to treat cystic fibrosis. in vitro studies in a liver model of ARPKD demonstrated the ability of the modulators to restore several cellular processes malfunctioning in ARPKD, resulting in reduced cyst growth. The modulators would improve function of the liver, kidneys, and lungs in ARPKD.

 

Value Proposition:

  • Identification of a novel target and strategy to treat ARPKD
  • The modulators offer a potentially curative treatment for ARPKD
  • Suitable as mono- or combination therapy to optimize treatment of ARPKD

Looking for Partners to: Develop & commercialize the technology as a novel treatment for ARPKD.


Stage of Development: Pre-Clinical

           

Data Availability: in vitro 

 

Inventors: Liudmila Cebotaru 

 

Patent Status: Provisional patent application filed.

 

Publication(s): N/A

Patent Information:
Title App Type Country Serial No. Patent No. File Date Issued Date Expire Date Patent Status
CYSTIC FIBROSIS TRANSMEMBRANE CONDUCTANCE REGULATOR MODULATORS FOR TREATING AUTOSOMAL RESESSIVE POLYCYSTIC KIDNEY DISEASE ORD: Ordinary Utility United States 17/674,090   2/17/2022     Pending
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For Information, Contact:
Vera Sampels
vsampel2@jhu.edu
410-614-0300
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