Unmet Need
- Autosomal dominant polycystic kidney disease (ADPKD) is a common genetic disorder characterized by progressive kidney cyst formation, frequently leading to end-stage renal failure.
- Current therapies are limited, with Tolvaptan offering only modest efficacy and being associated with liver toxicity and poor tolerability.
- Existing approaches do not directly target key metabolic and inflammatory pathways driving disease progression.
- There is a clear need for novel, mechanism-based and better-tolerated therapies for ADPKD patients.
Our Innovation
- This invention introduces a novel therapeutic strategy for ADPKD based on modulation of a naturally occurring endogenous biological signaling pathway that has not previously been targeted in this disease context.
- We have identified that key components of this endogenous pathway are dysregulated in ADPKD, with pathway activity increasing alongside disease severity, directly linking it to cyst growth and inflammatory processes.
- Based on these findings, we propose the use of peripherally acting modulators of this endogenous pathway to slow or prevent disease progression.
- By selectively targeting pathway activity outside the central nervous system, the approach aims to reduce kidney pathology while minimizing the risk of neurological side effects.
- Alterations in pathway-related markers may also support the development of biomarkers for disease monitoring and assessment of treatment response.
Advantages
- First-in-class approach targeting this endogenous pathway in ADPKD
- Mechanism-driven intervention, addressing inflammation, cell proliferation, and metabolic dysregulation
- Peripherally restricted strategy, designed to improve safety and tolerability
- Biomarker-enabled personalization, allowing patient stratification and response monitoring
- Supports both therapeutic and diagnostic applications within a unified biological framework
Commercial Opportunity
- We are seeking collaboration with pharmaceutical and biotech companies developing therapies for kidney or metabolic diseases, as well as diagnostic companies interested in precision medicine solutions based on endogenous biological pathways.
Contact in Yissum: Ariela Markel
