Perlara embarks on journeys of discovery called PerlQuests™ with families and patient organizations to develop treatments for rare diseases.
There are about 7000 rare diseases affecting 350 million people worldwide, of which roughly half are inherited, i.e. relating to genes. Until now, the cost of discovering chemical compounds that might be safe and effective candidates for the development of an FDA approved drug therapy has been too high for “rare” diseases affecting small populations. At the current rate of new drug approvals, it will take another 450 years before drugs are approved for all rare diseases.
Our Perlara Drug Discovery Platform increases the speed and lowers the cost of cure discovery by rapidly screening thousands of potential drug candidates, providing new hope for millions of rare disease patients.
We have identified at least 250 single-gene diseases that fit our Perlara Platform. These diseases share some overlap with common diseases, allowing us to begin the quest for treatments for over 1000 diseases. Our first PerlQuests target Niemann-Pick C Disease (NPC) and Niemann-Pick A Disease (NPA), lysosomal storage disorders, NGLY1 Deficiency, a metabolic disease related to proteasome-mediated degradation, and PMM2-CDG, a congenital disorder of glycosylation. What we are learning from the NPC PerlQuest may lead to drug candidates for Alzheimer’s disease.
Let’s PerlQuest together
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Research updates from our Blog
What the NGLY1 community can learn from the humble fly On the first day of the new year, we submitted a new NGLY1 model preprint entitled “Defects in the neuroendocrine axis cause global developmental delay in a Drosophila model of NGLY1 Deficiency.” Follow on...
Comparative fly model studies of NGLY1 Deficiency Earlier this week I was alerted on Twitter (where else?) to a new NGLY1 paper from Dr. Clement Chow’s lab at the University of Utah entitled: “Transcriptome and functional analysis in a Drosophila model of NGLY1...
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...