Status: Need is Closed

Novel drugs or targets to treat polycystic kidney disease

Request Number

Point of Contact
Need was closed on
Oct 16

GlaxoSmithKline (GSK) seeks a novel drug or target to treat polycystic kidney disease.


  • ADPKD affects ~500,000 patients in US and ~10 million world-wide with limited treatment options. ADPKD patients develop progressive cyst growth in kidney over decade, eventually leading to deterioration of kidney function combined with severe pain, hematuria and fibrosis/inflammation. Two mutations (Pkd1/pkd2) account for >95% of ADPKD cases, and the dominant nature and high penetration rate of this disease affects patient families in a profoundly negative way in both health care and social interactions. There is currently no FDA approved medicine for ADPKD in US, although vasopressin antagonist and somatostatin mimetics are in phase 3 studies.
  • GSK aims to deliver innovative medicine to treat ADPKD based on novel mechanism of action. We are particularly interested in agents or targets that function upstream of final cellular proliferative response and have solid genetic/pharmacological evidence supporting the role of such target/agents in human ADPKD. We are also interested in mechanisms/targets that can treat both liver and kidney cyst progression.
  • We are open to form a partnership or alliance with perspective investigators/sponsors to pursue such new target/agents upon review and approval.
Key success Criteria

The anticipated drug target molecule(s)/gene(s) should meet the following requirements:

  • A different mechanism of action from that of existing drugs including vasopressin antagonist or somatostatin mimetics.
  • Relevant validation data with the drug or target (or a viable plan to acquire) in a human in vitro disease model where the phenotype is driven by the predominant mutations of PC1/2 that drive human disease. Although not essential, evidence of efficacy in relevant PKD animal models would be desirable.



Possible Approaches

Any technology or approach that meets the requirements will be considered.


Approaches not of Interest
  • Target molecule(s) with the same mechanism of action as that of existing drugs (Tolvaptan) or investigational drugs under development (Octreotide, Lanreotide, Pasireotide)
  • Validation data derived from animal models only
  • Agents/targets that exclusively target cell proliferation response, e.g. repurposed oncology compounds.
  • Compounds with known toxicity in chronic setting.



Due Date
Oct 16
Items to be submitted


  • Applicant organizations are required to submit non-confidential proposals.
  • The scientists in GlaxoSmithKline will review proposals and select candidates for further discussions.. Once candidates are selected, GSK will execute confidential disclosure agreements (CDAs) to seek further information disclosure and discuss possible research approaches.
  • Once the best candidates have been determined, GSK will execute research collaborations, ,joint contract research and / or licensing agreements with partners.



  • A non-confidential proposal including the following information:
  • Overview of the drug target(s) (such as characteristics, superiority to existing drug targets)
  • Rationale and human in vitro modeling data indicating validity as a drug target (for example the data could include human genetic evidence, relevant human phenotypic readouts related to the mechanism of interest in human cell types, with appropriate controls)
  • Current research and development stage
  • Future research and development plans
  • An overview of your organisation and your experience with target validation
  • Any request concerning the structure of collaboration
  • Related intellectual property


Client Point of Contact

Martin Wallace
Gallery Moderator(s)
Stephen Clulow
Request Number
Preferred Collaboration Types
Area of Interest