Perlstein Lab PBC the company was officially born in February 2014. What have we accomplished since? Here’s a recap of our first year (10 months), told in tweets.

Both the team and the lab itself coalesced over the course of the spring and summer. That meant assembling the infrastructure of the lab, and validating Niemann-Pick C/NPC mutant flies, worms and patient-derived cells:

By the fall, NPC mutant fly and worm phenotypic screens had been road tested on a 1,000+ compound collection of so-called bioactives, which includes FDA approved drugs and probe compounds used in preclinical research. We also got the gold standard NPC biochemical biomarker — filipin staining of intracellular cholesterol — working in multiple NPC patient-derived cell lines.

Below is a highlight reel of the optimization and industrialization processes:

After Thanksgiving, we commenced the NPC HTS campaign. Like all dry runs, we encountered unexpected snags and hiccups, but that only sharpened our protocols and methods. Just before Christmas we had our first screening positives thanks to yeast cells and 384-well plates, which can easily achieve a throughput rate of 10,000 compounds per 48 hours with a bit of teamwork:

We look forward to more screening positives from yeast, as well as screening positives from the NPC mutant fly and worm screens over the coming weeks and early months of 2015.

 

Image: (c) Can Stock Photo / Iscatel

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