Benevolent Platform Precision Medicine
Abl target hypothesis has been validated using
complex cell-based systems
The SITraN assay: a humanised cell model of
ALS
Working in collaboration with academic
experts at SITraN who have developed a
patient cell derived assay system
¡Astrocytes (via induced progenitors)
obtained from genetically-diverse ALS
patients cause motor neuron toxicity in a
co-culture assay
Can test biological hypotheses by
measuring the rescue of motor neuron
survival
Benevolent proprietary CNS penetrant
Abl inhibitors are neuroprotective in this
assay
Induced
Patient
Fibroblasts
Neuronal
Progenitor
Cell
Induced
Astrocytes
Motor
Neurons
uron survival
% motor neuron
-50
150-
100-
50-
Healthy donor
¡Astro/DMSO
C9orf72 Patient iAstro/DMSO
C9orf72 Patient iAstro/10uM Riluzole
C9orf72 Patient iAstro/10uM
Gefitinib
Non-CNS penetrant TK inhibitor
C9orf72 Patient iAstro/1uM BEN-A
BAI CNS penetrant Abl inhibitor
Benevolent 48View entire presentation