NGLY1

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

PerlQuest Follow NGLY1 PerlQuest NGLY1 NGLY1 PerlQuest 2700x880px
Niemann-Pick Type A

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

PerlQuest Follow NPA PerlQuest Niemann-Pick
Type A
Niemann-Pick Type A Perlquest
PMM2-CDG

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

PerlQuest Follow PMM2-CDG PerlQuest PMM2- CDG PMM2-CDG Perlquest 1200x675px
Multiple Sulfatase Deficiency

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

PerlQuest Follow MSD PerlQuest Multiple
Sulfatase
Deficiency
GNAOI

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

PerlQuest Follow GNAO1 PerlQuest GNAO1
Coffin-Lowry Syndrome Coffin-Lowry Syndrome PerlQuest1200x600px

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

PerlQuest Follow CLS PerlQuest Coffin-Lowry
Syndrome
Pancreatitis

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

PerlQuest Follow Pancreatitis PerlQuest Pancreatitis Pancreatitis PerlQuest 2400x1600px
Cori Disease

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

PerlQuest Follow GSD III PerlQuest Cori Disease Cori Disease PerlQuest Glycogen illustration 1200x630px
MPSIIIA

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.

PerlQuest Follow MPSIIIA PerlQuest MPSIIIA Sanfilippo Syndrome MPS111A PerlQuest 800x800px
MSIIIB

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

PerlQuest Follow MPSIIIB PerlQuest MPSIIIB Sanfilippo Syndrome MPSIIIB PerlQuest 1200x600px
Leigh Syndrome

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

PerlQuest Follow Leigh Syndrome PerlQuest Leigh
Syndrome
Mitochondria 2000x1000px
APOL1 kidney disease

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

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

Welcome to the archival website of Perlara 1.

Take a look around at the foundations of Perlara’s exciting new Guided Cures™ Journey.

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

Translate »