Hi, my name is Ethan. I’m the Founder and CEO of Perlstein Lab. Perlstein Lab is a biotech startup and public benefit corporation based in San Francisco. Our mission is precision (personalized) orphan disease drug discovery. In this inaugural post, I will briefly recount the genesis of Perlstein Lab, the science behind our evolutionary approach, and why Perlstein Lab takes online outreach seriously.

18 months ago I faced the most important decision of my professional life: continue to try to break into academia, or leave for greener pastures. Like so many other members of Generation Postdocalypse, I could not ignore the prospects of endless competition for publications, grants and jobs . Was there a way to blend curiosity-driven research with tangible therapeutic outcomes? Of all things, Twitter became my global learning and support group, and I started to investigate options beyond the ivory tower. Very quickly I discovered communities of patient advocates, including inspiring parents of kids with orphan diseases, who convinced me that solving orphan disease puzzles would be the ideal path forward.

When I began putting together a formal business plan and slide deck last Fall, I was most confident about the science underlying our evolutionary drug screening platform, which is based on genome-edited primordial disease models: yeast, worms, flies and fish. When I was a grad student in Professor Stuart Schreiber’s lab at Harvard between 2002 and 2006 I validated yeast as a model for pharmacology. I then advanced these ideas as an independent fellow at Princeton from 2007 to 2012 by studying the pharmacology of antidepressants in yeast cells.

I knew I was onto something when I attended Rare Disease Day 2013 at NIH and learned that very few (if any) orphan disease researchers leverage primordial disease models, even though ancestral forms (aka homologs) of many orphan disease genes are evolutionarily conserved in simpler organisms. Instead, the vast majority of orphan disease research relies on mouse models of disease, and has relied on mouse models for the last 30+ years.

Most biotech startups don’t have a blog or Twitter presence. Perlstein Lab will be different from the typical biotech startup with respect to online outreach, especially patient engagement. What initially drew me into the orphan disease space was the openness, zeal and grace of patient advocates on Twitter and the blogosphere, so it’s only fair that Perlstein Lab returns the favor. As a for-profit company, we obviously have to be mindful of proprietary concerns. Wherever possible we will strive to engage patients, advocates, scientists or the public on our science, and share the week-to-week victories and tribulations of a 5-person biotech startup.

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