Scalable drug discovery platform
Perlara uses a platform of genetically engineered animals (yeast, nematodes, fruit flies and zebrafish) in phenotypic screens to identify orphan drug candidates that reverse disease much faster and cheaper than current approaches. Our proof-of-concept diseases are Niemann-Pick type C (NPC), a lysosomal storage disorder first described nearly a century ago, and NGLY1 Deficiency, a metabolic disease related to proteasome-mediated degradation.
Perlara Platform Science
1. What does Perlara do?
Perlara develops precision drug discovery programs for rare genetic diseases. We engineer model organisms to express patient mutations for use in industrial-scale, organism-based phenotypic screens,
2. Shared genes
All animals sit on the same branch of the evolutionary tree and share thousands of genes. Many of these ancient genes are unchanged at the DNA level despite a billion years of evolution.
3. Single gene diseases
Single gene diseases are caused by a spectrum of mutations and result in cellular defects. Missense mutations prevent proteins from folding correctly, which causes partial loss of gene function. Nonsense mutations prevent protein production, which a causes complete loss of gene function.
4. Gene editing
Gene editing technology enables us to generate patient-mutation-matched Perlara disease models that leave no mutation behind.
5. Automated drug screening
The resulting disease models manifest cellular defects that can be reversed by novel chemical entities in drug screens. The first version of the Perlara Platform leverages multi-well plate technology and automation.
6. Bio-Pharma cure pipeline
We partner with BioPharma and Patient advocacy Groups to advance precision drug candidates to the clinic. Our pipeline begins with childhood single-gene disease involving loss of an ancient cellular process: lipid storage diseases, congenital disorders of glycosylations, peroxisomal biogenesis disorders, mitochondrial diseases, ciliopathies and laminopathies.
Science updates from our Blog
Earlier this week I was alerted on Twitter (where else?) to a new NGLY1 paper from Dr. Clement Chow’s lab at the University of Utah entitled: “Transcriptome and functional analysis in a Drosophila model of NGLY1 deficiency provides insight into therapeutic...
The Condition: Mucolipidosis Type IV Mucolipidosis is a group of inherited lysosomal storage disorders characterized by aberrant storage and accumulation of lipid vesicles in patient cells. Mucolipidosis type IV (MLIV) is a rare genetic lysosomal storage disorder...
This is the fourth post in an open science blog series about PERL101 (P101), a small molecule with big hopes. Demystifying drug discovery The default setting of biotech startups and pharma giants alike is stealth mode. At Perlara the default is set to open. As the...