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
