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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

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.
Deeper Perlara Science

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

CE Aging 2018 Conference Recap

by | July 17, 2018 | Science | 0 Comments

Last week, our blog featured a recap of the first of two C. elegans topic meetings - CE Neuro 2018 - that some members of Perlara's worm team attended last month. In this post, Hillary and I share our experiences at the CE Aging 2018 conference. Also held at the...
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Developing High Throughput Enzymatic Assays

by | July 10, 2018 | Science | 0 Comments

At Perlara, we work to find therapeutics for rare genetic diseases. We develop and use immunoassays, phase contrast imaging, as well as enzymatic assays, in order to screen thousands of compounds and obtain hits from cell readouts to complement our model organism...
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CE Neuro 2018 Conference Recap

by | July 9, 2018 | Science | 0 Comments

Perlara’s worm team recently attended two C. elegans neuroscience conferences - CE Neuro 2018 & CE Aging 2018 - both held at the University of Wisconsin, Madison. This post recaps Zach and my time at the CE Neuro 2018 conference, which focused on neuronal...
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OI-driven drug discovery

by | June 4, 2018 | Organic Intelligence, Science | 0 Comments

What is the role of OI in drug discovery? Organic Intelligence (OI) is the collective brainpower and high emotional quotient of scientists and rare disease advocates teaming up to use the PerlArk™ Platform of model organisms, lab automation and computation for a new...
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Completing an MLIV drug repurposing screen in cells

by | May 23, 2018 | Science | 0 Comments

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 disorders. For background on MLIV, and to learn more about our work in optimizing a MLIV drug...
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Modeling Gaucher disease in worms

by | May 17, 2018 | Science | 0 Comments

In 2016, Perlara and Wylder Nation launched a PerlQuest to find a cure for Niemann-Pick Type A (NPA). We have also been developing simple organism disease models for Niemann-Pick Type C (NPC) as a part of our research collaboration with Novartis. An interesting...
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59th Drosophila Research Conference Recap

by | April 30, 2018 | Science | 0 Comments

My colleague, Tamy Portillo Rodriguez, and I attended the 59th Annual Drosophila Research Conference - a fantastic meeting of scientists exploring many different fields of biology using the fly - in Philadelphia earlier this month. The conference (in some circles...
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Finding the right marker for Mucolipidosis Screen

by | December 11, 2017 | Perlara science | 0 Comments

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 that...
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Hit to lead, lead to partnership

by | December 6, 2017 | Perlara science | 0 Comments

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...
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From Dead to Alive with a Small Molecule

by | November 1, 2017 | Perlara science | 0 Comments

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 and...
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