NGLY1 Deficiency PerlQuest

“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

We are screening compounds and have discovered some promising leads. Stay tuned!

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NGLY1 PerlQuest updates from our Blog

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...

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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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