anaconda-powered rapid drug discovery | anacondacon 2017
TRANSCRIPT
© 2017 Recursion Pharmaceuticals
DISRUPTING DRUG DISCOVERYWITH THE POWER OF IMAGES
Blake Borgeson Co-Founder & CTO [email protected]
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© 2017 Recursion Pharmaceuticals
PROBLEMSLOW PACE OF DRUG DISCOVERY
Maren. 38 months old. Her parents sent us this photo.
Thousands of rare genetic diseases exist, nearly all untreated.
Millions of people in the US alone are affected.
The traditional drug discovery model of one disease, one pathway would take hundreds of years to address them all.
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Monogenic diseases often present with specific structural phenotypes, as with the distinct facial
features of patients with Cornelia de Lange Syndrome. Similarly, specific structural signatures
for many monogenic diseases are accessible via cellular images.
Healthy child
Child with rare genetic disease
(Cornelia de Lange Syndrome)
Healthy cells
Genetic disease model cells
(Cornelia de Lange Syndrome)
© 2017 Recursion Pharmaceuticals
SOLUTIONTHE POWER OF IMAGES
\© 2017 Recursion Pharmaceuticals
TECHNOLOGYAUTOMATED & SCALABLE
Healthy Disease Disease + Drug?
Cell Painting images of cellular models of human diseases reveal hundreds of structural and
functional features unique to each disease model. These features encode disease specific
phenotypic profiles that can be extracted using computer vision methods.
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Indi
vidu
al F
eatu
res
0 IncreaseDecrease
Nuclear TextureNuclear RadiusER IntensityRNA IntensityMitochondrial Texture
© 2017 Recursion Pharmaceuticals
HTS biological tools enable us to build hundreds of disease models ‘per plate’
Computer vision and machine learning enable the efficient extraction and meaningful representation of the data
We use the latest biological tools to build hundreds of unique genetic disease models, imaging tens
of thousands of cells per model, and extracting nearly 1,000 structural features from every cell.
With a disease-specific cellular phenotype in hand, we can ask whether any drug rescues the
defects back to ‘health.’ This approach represents a scalable and generalizable drug
discovery assay.
TECHNOLOGYAUTOMATED & SCALABLE
\© 2017 Recursion Pharmaceuticals
DEVELOPMENTCASE STUDY: CEREBRAL CAVERNOUS MALFORMATION
Weakening of vessel walls
100,000+ symptomatic patients in U.S.
Stroke, headache, epilepsy
Adult-onset, progressive
No medical treatment
Patient Brain MRI
\© 2017 Recursion Pharmaceuticals
DEVELOPMENTCASE STUDY: CEREBRAL CAVERNOUS MALFORMATION
Weakening of vessel walls
100,000+ symptomatic patients in U.S.
Stroke, headache, epilepsy
Adult-onset, progressive
No medical treatment
Patient Brain MRI
Tested 2,000 compounds Selected 2
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DEVELOPMENTCASE STUDY: CEREBRAL CAVERNOUS MALFORMATION
Mouse brains showing lesions (healthy brains have none)
Mouse disease model
Tempol
Vitamin D
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a.com
Rec001 and Rec002 Reduce Lesion Burden in CCM
Mice
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w.RecursionPharm
a.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
www.RecursionPharma.com
Rec001 and Rec002 Reduce Lesion Burden in CCM Mice
www.RecursionPharma.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
www.RecursionPharma.com
Rec001 and Rec002 Reduce Lesion Burden in CCM Mice
www.RecursionPharma.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
www.RecursionPharma.com
Rec001 and Rec002 Reduce Lesion Burden in CCM Mice
www.RecursionPharma.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
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DEVELOPMENTCASE STUDY: CEREBRAL CAVERNOUS MALFORMATION
Mouse brains showing lesions (healthy brains have none)
Mouse disease model
Tempol
Vitamin D
ww
w.RecursionPharm
a.com
Rec001 and Rec002 Reduce Lesion Burden in CCM
Mice
ww
w.RecursionPharm
a.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
www.RecursionPharma.com
Rec001 and Rec002 Reduce Lesion Burden in CCM Mice
www.RecursionPharma.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
www.RecursionPharma.com
Rec001 and Rec002 Reduce Lesion Burden in CCM Mice
www.RecursionPharma.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
www.RecursionPharma.com
Rec001 and Rec002 Reduce Lesion Burden in CCM Mice
www.RecursionPharma.com
Gibson, et al., Circulation. In Press
Standard Diet REC001 Diet REC002 Diet
REC002 Diet
REC001 Diet
Standard Diet
11
Sunshine paper published Academic/gov’t funded trials
We’re moving towards clinical trials
Gibson, CG, 2015. Circulation.
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© 2017 Recursion Pharmaceuticals
DEVELOPMENTPROGRAMS
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NEUROFIBROMATOSIS TYPE 2
CORNELIA DE LANGE SYNDROME
ATAXIA-TELANGIECTASIA PROGRAM 1
DIAMOND BLACKFAN ANEMIA
SPINAL MUSCULAR ATROPHY (SMN) PROGRAM 1
PEUTZ-JEGHERS SYNDROME
CEREBRAL CAVERNOUS MALFORMATION PROGRAM 1
PRE-INDIN VITRO IN VIVO
SPINAL MUSCULAR ATROPHY (UBA1)
CEREBRAL CAVERNOUS MALFORMATION PROGRAM 2
USP7- UNDIAGNOSED DISEASE
METACHROMIC LEUKODYSTROPHY/ATYPICAL GAUCHER DISEASE
10+ INDICATIONS
with CANDIDATES in ACTIVE DEVELOPMENT
ATAXIA-TELANGIECTASIA PROGRAM 2
SPINAL MUSCULAR ATROPHY (SMN) PROGRAM 2
UNDISCLOSED PARTNER INDICATION 1
UNDISCLOSED PARTNER INDICATION 2
In less than 1 year of screening
\© 2017 Recursion Pharmaceuticals
FINDING NEW TREATMENTSINTERACTIVE DATA VISUALIZATION
Anaconda demo
\© 2017 Recursion Pharmaceuticals
Blake Borgeson Co-Founder & CTO [email protected]
Massively hiring:Data Scientists ML Researchers Software Engineers RecursionPharma.com/careers