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
This post is dedicated to World Rare Disease Day (WRDD) 2017. It was at the WRRD four years earlier when the value proposition for Perlara crystalized in my mind. I went to NIH that frosty last day of February in order to meet with patient advocates and ask them...
I, along with Nina, performed our first high-throughput screen – well really medium-throughput but high-throughput for us – on Niemann-Pick Type C (NPC) patient-derived fibroblasts a couple of months back. We wanted to do a pilot screen with the Microsource...
Well it has been a little while since I wrote a post, and a lot has happened since then, which I am sure you read about in our blogs and tweets. We changed our company name, partnered with Novartis and started partnering with patient groups to engineer our...