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
I’m excited to share the latest news! Today we announced our sixth PerlQuest partnership. Our PerlQuest partner is the Multiple Sulfatase Deficiency Action Foundation (MSDAF), a research-focused charity based in Ireland whose mission is to work towards a cure for...
On March 1, 2018, Perlara PBC and Global Genes hosted an event in honor of World Rare Disease Day. The first of two fireside chats featured four Bay Area biotech CEOs working on rare diseases, who shared their stories of how they came to ‘care about rare’. This was...
Perlara PBC and Maggie’s Cure have started a PerlQuest for a glycosylation disorder called PMM-2 deficiency disease - one of the most common congenital disorders of glycosylation. Over the past year at Perlara, we have modeled the pmm-2 deficiency disorder in...