We identified tool compounds,
repurposable drugs as well as
novel chemotypes that rescue
worm and fly disease models.
We identified overlapping hits
between worm and patient fibroblast
drug repurposing screens.
We identified natural products
and repurposable drugs that rescue
yeast and worm disease models.
MSD mutant flies are in the lab
and phenotyping has begun!
"Perlara’s work will help put
children with a GNAO1-related
further on the radar of researchers
and pharmaceutical companies.”
"The collaborative drug
discovery effort gives hope
for new insights about CLS
and better lives for our children.”
We’re excited to break new ground
scientifically with a yeast-to-organoid
drug screening pipeline for pancreatitis
and cystic fibrosis.
Perlara’s expertise will accelerate
the identification of small molecules
as potential treatments for GSD patients.
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.
We are developing nematode and fly
disease models and screening compounds
for promising leads. Stay tuned.
"A new treatment for Leigh syndrome
could have broader implications for
aging, sports medicine, traumatic
brain injury, neurodegeneration
and other indications."
"Genetic risk for APOL1 kidney
disease affects ~100 million