Mapping the Genetisphere
All life on earth has a shared genetic heritage that we call the Genetisphere
Our PerlArk™ 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 PerlArk™ 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
One of the rare genetic disorders that we at Perlara have been working on is Mucolipidosis type IV (MLIV) – one of the Mucolipidosis group of inherited lysosomal storage...