NGLY1 Deficiency PerlQuest

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“We’ve worked closely with the Perlara team since the company’s formation. We are proud to be a launch partner of PerlQuests and are excited about the drug leads that will emerge. Perlara’s nimble platform allows it to quickly iterate and in turn cut down the time when NGLY1 patients can receive a life-saving treatment.”
Matt Wilsey
President of Grace Science Foundation

Our Progress

Stages 1-3 are complete. We’re currently in licensing discussions to advance repurposable drugs toward clinical development.

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PerlQuest updates from our Blog

Grace Science Foundation’s 2018 NGLY1 Conference recap

This past summer was a rare disease conference-palooza. In July, I attended the 2018 NGLY1 Conference, hosted by Grace Science Foundation (GSF) in Palo Alto. It was the 5th gathering of families and scientists from around the world, united behind one goal: to find a...

Developing an NGLY1 worm screen

In 2016, Perlara and Grace Science Foundation launched a PerlQuest for NGLY1 Deficiency (also known as NGLY1-CDDG). As a part of this collaboration, we have embarked on developing an NGLY1 worm screen. This post will detail the worm team's latest progress toward that...

New NGLY1 preprint

On the first day of the new year, we submitted a new NGLY1 model preprint titled “Defects in the neuroendocrine axis cause global developmental delay in a Drosophila model of NGLY1 Deficiency.” Follow on Twitter. Here I will explain in plain English what the NGLY1...

Fly models 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 titled: “Transcriptome and functional analysis in a Drosophila model of NGLY1 deficiency provides insight into therapeutic approaches,”...

Our talk for the 2016 Grace Science Foundation Meeting

This past weekend was the 2016 Grace Science Foundation Meeting in Palo Alto.  It was a great success!  Here are the slides from my talk, with a few blurbs about them and what's to come. Curious about this?  Check out my first blog on making NGLY1 flies.  Prior to...

Fruit Flies and DMSO Sensitivity

On my last blog I told you guys about a good friend of small drugs, DMSO who has wonderful characteristics that make it such an irresistible small drug solvent. Although is a great friend of small drugs, some other of its characteristics make DMSO a toxic molecule to...

CRISPR genome editing to make an NGLY1 fly

N-glycanase 1 deficiency, or “NGLY1,” is a rare genetic disorder arising from mutations in the ngly1 gene.  The disease was recently diagnosed in what was a remarkable partnering of patient advocates and scientists who used DNA sequencing to trace the disease-causing...
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