Scalable drug discovery platform
Perlara uses a platform of CRISPR-engineered animals (yeast, nematodes, fruit flies and zebrafish) in phenotypic screens to identify orphan drug candidates that reverse disease much faster and cheaper than current approaches. Our proof-of-concept diseases are Niemann-Pick type C (NPC), a lysosomal storage disorder first described nearly a century ago, and NGLY1 Deficiency, a metabolic disease related to proteasome-mediated degradation.
Perlara Platform Science
1. What does Perlara do?
Perlara develops precision drug discovery programs for rare genetic diseases. We engineer model organisms to express patient mutations for use in industrial-scale, organism-based phenotypic screens,
2. Shared genes
All animals sit on the same branch of the evolutionary tree and share thousands of genes. Many of these ancient genes are unchanged at the DNA level despite a billion years of evolution.
3. Single gene diseases
Single gene diseases are caused by a spectrum of mutations and result in cellular defects. Missense mutations prevent proteins from folding correctly, which causes partial loss of gene function. Nonsense mutations prevent protein production, which a causes complete loss of gene function.
4. CRISPR gene editing
CRISPR/Cas gene editing technology enables us to generate patient-mutation-matched Perlara disease models that leave no mutation behind.
5. Automated drug screening
The resulting disease models manifest cellular defects that can be reversed by novel chemical entities in drug screens. The first version of the Perlara Platform leverages multi-well plate technology and automation.
6. Bio-Pharma cure pipeline
We partner with BioPharma and Patient advocacy Groups to advance precision drug candidates to the clinic. Our pipeline begins with childhood single-gene disease involving loss of an ancient cellular process: lipid storage diseases, congenital disorders of glycosylations, peroxisomal biogenesis disorders, mitochondrial diseases, ciliopathies and laminopathies.
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 one...read more
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 bioactives...read more
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 organisms...read more