A blog about our discoveries and community
World Rare Disease Day 2017
This post is dedicated to World Rare Disease Day (WRDD) 2017. It was at the WRRD four years earlier when the value proposition for Perlara crystalized in my mind. I went to NIH that frosty last day of February in order to meet with patient advocates and ask them one...
read moreOptimizing my first High Throughput Screen
I, along with Nina, performed our first high-throughput screen – well really medium-throughput but high-throughput for us – on Niemann-Pick Type C (NPC) patient-derived fibroblasts a couple of months back. We wanted to do a pilot screen with the Microsource bioactives...
read moreNPC1 KO mouse brain study
Well it has been a little while since I wrote a post, and a lot has happened since then, which I am sure you read about in our blogs and tweets. We changed our company name, partnered with Novartis and started partnering with patient groups to engineer our organisms...
read more2016 in review
"Last Month in Tweets" was on again, off again in 2016. Apologies, we'll be more consistent with monthly updates across the board in 2017. For now, let's relive the best moments of 2016 at Perlara! Although I wasn't able to announce it publicly for several months, we...
read moreFrom Pitch to Partnership – 3 years of Perlara spending
As 2016 draws to a close, we look back on two and half years of Perlara spending. Let’s begin with burn rate. This figure shows our monthly burn since first cash inflows in April 2014: In 2014, we completed model validation and assay development for our lead discovery...
read morePerlara’s first preprint
Two weeks ago we posted Perlara's first preprint to bioRxiv, along with all the raw data on GitHub: https://twitter.com/biorxivpreprint/status/796019478999998465 This effort was led entirely by a very talented undergrad named Maria Teresa Chavez, who reached out to...
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