Investor Presentaiton
Target
protein
Molecular
glue
molecule
E3
ligase
Targeting the previously undruggable: A novel CELMOD
for Sickle Cell Disease
Transformational potential
Oral small molecule that increases fetal hemoglobin to
functionally cure sickle cell anemia (e.g., eliminate pain
crisis, prevent long term organ damage).
Causal human biology
Genetically validated targets that lead to persistence of
fetal hemoglobin (HbF) are associated with improved
clinical outcomes in patients with sickle cell anemia.
Beta-globin locus
5 4 3 2 1
ε
Gy Ay
δ Β
HbS
Matching modality to mechanism
Through our CELMOD
proteomics initiative, we
have identified CELMODs
that degrade HbF genetic
targets and increase HbF
in pre-clinical models.
HbF expression
8000-
wwww
6000-
Vehicle control
CELMOD (high)
CELMOD (low)
4000-
2000-
TH
Path to clinical proof-of-concept
α
B
disease
10%
HbF
20%
30%
Hydroxyurea
a
LCR HSS Embryonic
Fetal
Adult
3'HS1
HbF
Ill Bristol Myers Squibb™
healthy
Reduced Reduced
mortality recurring events
Asymptomatic
presentation
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