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Rizwana Rashid


DPhil Student

I am working on drug discovery for atypical lysosomal storage disease, Niemann-Pick type C disease (NPC) and NPC-related diseases. Very significantly, we have recently found that the NPC pathway is inhibited as a secondary consequence of the primary metabolic defect in multiple rare metabolic diseases and by a major human microbial pathogen, Mycobacterium tuberculosis. Importantly, the treatments we have developed for NPC disease are unexpectedly proving to be effective in treating NPC-related diseases.

We are now looking to develop a new class of NPC therapeutics to treat this wider family of NPC-related disorders, building upon our extensive drug discovery/development history (miglustat). We will exploit the broad range of cellular and animal models of these diseases established within our laboratory for screening and efficacy studies.


  1. Identify analogues of curcumin with a virtual screen using shape and electrostatics and predicted bioavailability.
  2. Develop and validate a high-content cellular screen for endolysosomal trafficking defects using a cell model of NPC.
  3. Determine the activity of known drugs (marketed and abandoned) in the high-content cellular screen.
  4. Determine the activity of the top-ranking compounds from the virtual screen in the high-content cellular screen.
  5. Determine the activity of compounds positive in the cellular screen in a proof-of-concept study in NPC mice.