WP 4 : Screening of potential therapeutic molecules​

The objective of this WP is to:

  • Screen FDA approved, Lopac and ENYOpharma proprietary libraries with SMA MyoScreen,
  • Validate the hits in secondary (in vitro muscle fibers) and tertiary (organoids) assays,
  • Optimize non-developable compounds of interest by EDELRIS,
  • Evaluate developable candidate molecules in vivo in SMA mouse models,
  • Characterize the mode of action of chosen leads.

WP 4.1: In vitro screening

Cytoo, Gache, Chazaux, ENYOPharma

WP 4.2. In vivo efficacy

Courchet, Chazaud, Freyssenet
Jacquemond, Schaeffer, Charbonnier

WP 4.3: Lead optimization for non developable leads

ENYOpharma, EDELRIS, Cytoo, Schaeffer

WP 4.4: Study of the chosen lead mechanism of action

Courchet, Chazaud, Freyssenet, Jacquemond, Schaeffer, Gache, Charbonnier