NGLY1 NGLY1 PerlQuest 2700x880px NGLY1 PerlQuest

We identified tool compounds,
repurposable drugs as well as
novel chemotypes that rescue
worm and fly disease models.

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Niemann-Pick Type A Niemann-Pick Type A Perlquest Niemann-Pick
Type A
PerlQuest

We identified overlapping hits
between worm and patient fibroblast
drug repurposing screens.

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PMM2-CDG PMM2-CDG Perlquest 1200x675px PMM2- CDG PerlQuest

We identified natural products
and repurposable drugs that rescue
yeast and worm disease models.

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Multiple Sulfatase Deficiency Multiple
Sulfatase
Deficiency
PerlQuest

MSD mutant flies are in the lab
and phenotyping has begun!

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GNAOI GNAO1-PerlQuest-Hero-1200x600px GNAO1 PerlQuest

"Perlara’s work will help put
children with a GNAO1-related
neurodevelopmental disorder
further on the radar of researchers
and pharmaceutical companies.”

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Coffin-Lowry Syndrome Coffin-Lowry Syndrome PerlQuest1200x600px Coffin-Lowry
Syndrome
PerlQuest

"The collaborative drug
discovery effort gives hope
for new insights about CLS
and better lives for our children.”

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Pancreatitis Pancreatitis PerlQuest 2400x1600px Pancreatitis PerlQuest

We’re excited to break new ground
scientifically with a yeast-to-organoid
drug screening pipeline for pancreatitis
and cystic fibrosis.

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Cori Disease Cori Disease PerlQuest Glycogen illustration 1200x630px Cori Disease PerlQuest

Perlara’s expertise will accelerate
the identification of small molecules
as potential treatments for GSD patients.

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MPSIIIA Sanfilippo Syndrome MPS111A PerlQuest 800x800px MPSIIIA PerlQuest

With children losing ground to this disease
every day we have to we are hopeful that
our PerlQuests will bring us ever closer
to help for patients with Sanfilippo syndrome.

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MSIIIB Sanfilippo Syndrome MPSIIIB PerlQuest 1200x600px MPSIIIB PerlQuest

We are developing nematode and fly
disease models and screening compounds
for promising leads. Stay tuned.

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Leigh Syndrome Mitochondria 2000x1000px Leigh
Syndrome
PerlQuest

"A new treatment for Leigh syndrome
could have broader implications for
aging, sports medicine, traumatic
brain injury, neurodegeneration
and other indications."

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APOL1 kidney disease APOL1 Kidney Disease hero 2400x1200px APOL1
kidney disease
PerlQuest

"Genetic risk for APOL1 kidney
disease affects ~100 million
people worldwide"

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Perlara scientists are working with highly motivated families, patient organizations and researchers to develop cures for rare diseases and learn how genes work across diseases to map connections to common ailments. We call these exciting journeys of discovery PerlQuests™

Organic Intelligence (OI): more cures for more individuals and diseases

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