Over the summer we completed the hit-to-lead phase of our NPC discovery program, and in the fall we commenced mouse validation studies with PERL101, our most promising hit from the nematode and fly primary screens.

The big question we had going into our first mouse PK experiments was would PERL101 be orally bioavailable. Turns out it is! (I’ll blog more about that next week).

It was a blast to share PLab’s story as an invited speaker by the grad students at Dalhousie University in Halifax, Nova Scotia. I met with Jason Berman’s lab, which works on zebrafish, and met with other researchers who work on NPC, like Barbara Karten. There’s an amazing community of rare disease researchers at Dal and I look forward to returning one day.

Tom blogged about the fat and flies. Like many cellular processes, there’s more conservation than you think.

There was no PLab presence at the American Society for Human Genetics this year, but we’ll there next year when the conference returns to the West Coast. Drosophilista Ross Cagan received a shout-out. His lab’s work on using flies as a screenable model for thyroid cancer was an inspiration for us when we set out on the model org drug discovery journey.

Open Science means not just sharing results in real-time, but also lifting the curtain on the budget behind the data. We know we’re pioneers here but we think every biotech startup can and should increase transparency.

We continued to assess PERL101 on a battery of preclinical assays, including stability in human hepatocytes.

Our original landing page designs came from 99designs. In fact, our logo/Twitter avatar was designed by them. But at a certain point we need design concepts from someone with deeper scientific knowledge. I put out the call on Twitter and got connected to Betsy Skrip, an amazingly talented scientific illustrator and animator. We look forward to working with Betsy on future designs!

Nina blogged about NPC biomarkers, what’s known and what’s left to discover.

A reminder that the genes responsible for most Mendelian diseases are more ancient than the average gene, meaning model organisms are a really good fit for orphan drug discovery.

See you next month!

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