Seeking Novel Targeted Approaches for Genetic Kidney Disease

Our client, a world leader in developing treatments for rare genetic diseases, is seeking novel therapeutic approaches to treat the underlying causes of genetic kidney disease. Specifically, our client is interested in solutions to treat autosomal dominant polycystic kidney disease (ADPKD) and Alport syndrome, as well as cystinuria and UMOD-associated chronic kidney disease (CKD). 

Approaches to treat other rare genetic kidney diseases may also be of interest provided there is high unmet need, and the solution targets the underlying mechanisms of disease. 

Approaches of Interest:

• Novel therapeutics to treat autosomal dominant polycystic kidney disease (ADPKD) 
• Alport syndrome approaches that will either treat X-linked Alport or all subtypes of Alport with the same therapeutic 
• Approaches for cystinuria (any genetic subtype) 
• Approaches to treat UMOD-associated chronic kidney disease (CKD) 
• Approaches to treat autosomal dominant tubulointerstitial kidney disease (ADTKD-UMOD) alongside UMOD-associated chronic kidney disease are also of interest 

All modalities are of interest, with a preference for small molecules, oligonucleotides, peptides, AAV/gene therapy and biologics. Unique therapeutic modalities are encouraged. Approaches should address the core mechanisms of disease (E.g., correcting the genetic cause, or the central pathway of disease), not the associated symptoms. 

Out of Scope:

• Non-disease-modifying drugs, i.e. those only targeting sequelae 

Developmental Stages of Interest:

• Research with disease modifying potential at any developmental stage is of interest (early studies through to Phase III and registration) 
• Opportunities with in vivo validation and proof-of-concept data are of high interest, although opportunities with strong in vitro or ex vivo validation are also within scope 

Submission Information and Opportunity for Collaboration

Submission of one-page, 200–300-word briefs is encouraged, along with any optional supplementary information e.g. relevant publications. The team encourages including the proposed next steps in developing the research towards commercialization. In submitting to this campaign, you confirm that your submission contains only non-confidential information. 

Our client is open to a range of collaboration opportunities, with the most appropriate outcome being decided on a case-by-case basis. Example outcomes include funded research collaborations and agreements, or licencing of assets.