Mapping the Genetisphere
All life on earth has a shared genetic heritage that we call the Genetisphere
Our Perlara Drug Discovery Platform is based on creating disease models using simple animals that share genetic similarity with humans, allowing us to screen massive numbers of disease models and drug candidates quickly and at low cost.
How the Perlara Platform works
1. Build an Ark
We use gene editing technology to model specific human diseases in simple animals like yeasts, worms, flies and fish. Think Noah’s Ark of Drug Discovery.
2. Try out a boat load of compounds
We screen compounds on our disease models, and gather massive amounts of chemical and physiological response data.
3. Predict the best candidates
Data will fuel our predictive engine to identify high-potential drug candidates for specific patient mutations.
4. Precision treatments
We collaborate with BioPharma and partner with patients to turn Perlara drug candidates into precision treatments for rare and common diseases.
Disease modifiers for genetically similar rare and common diseases
Most of the approximately 4,000 single-gene diseases fit the Perlara Platform. Many of these diseases are caused by an ancient gene that also contributes to common disease risk. Our first PerlQuest™, Niemann-Pick Type C (NPC) disease, could lead to therapies for common neurodegenerative diseases. Learn more about PerlQuests on our FAQs page.
Does it really work?
Science updates from our Blog
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...
Perlara's high throughput screen progress in Mucolipidosis IV (MLIV) flies… In this blog post, I’ll tell you about Perlara's high throughput screen progress in MLIV flies. Last year, Tom Hartl reviewed some details of MLIV disease biology and described how worms...