Research programme

Repair-HD objectives will be achieved through the completion of 6 interlinked research and innovation work packages (WP):on  

Optimisation of differentiation protocols in vitro and in vivo 

  • WP1 will compare systematically three existing striatal differentiation protocols for their capacity to yield GMP-ready MSN precursors from GMP-grade hESCs/iPSCs
  • WP2 will perform a functional comparison of cell sources in rodent models using cell preparation protocols derived from WP1
  • WP3 will focus on PS-MSN functionality in allo- and xeno-graft in non-human primates as well as on the development of motor/cognitive tests and imaging tools to evaluate for differentiation, proliferation, potential immune reaction and efficacy.
     

 Characterisation and validation of GMP protocols for target cell generation 

  • By the beginning of year 3, we will select the optimal hPSC product and 'lock-down' the differentiation protocol, which will then be scaled up and validated at GMP grade (WP4).
      

Towards clinical application

At last, WP5 and WP6 will cooperate to deliver a full clinical trial protocol and regulatory package by the end of the programme for hESCs, which will equally inform subsequent applications involving hiPSCs:

  • WP5 will focus on establishing an updated assessment protocol to characterise the slow longitudinal progression of HD disease using the most sensitive and stable neurological, neuropsychological, neuropsychiatric and imaging measures available
  • WP6 will consider the regulatory, ethical and trial design issues required to integrate the data collected in WP5 into an effective clinical programme.

  

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