A patient portal and drug discovery platform for families affected by rare disease
Ethan Perlstein
Friday, May 19, 2017
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Chapters
Introduction
PerlQuest
PerlQuest: democratizing drug development
Perlara's drug discovery platform
From simpler disease models to novel lead compounds
Lead program partnered with Pharma
Nematode model of NPC
PERL 101 rescues developmental delay
PERL 101 has an unexpected MOA
PERL 101 & related chemotypes induce "bypass" vesicles
Bypass vesicles = lipid droplets
PERL 101 induces lipophagy?
PERL 101 is metabolically stable
PERL 101 is orally bioavailable
PERL 101 is well-tolerated
From primary screen in worms
NGLY1 PerlQuest partnered with Grace Science Foundation
NGLY1 larval size assay ready for HTS
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