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

Compound library screening

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.

find rare disease drug candidates to become precision cures

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?

Read about our lead compound for NPC (PERL101) and PerlQuest updates on our Blog.

Science updates from our Blog

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