With $2M Seed, Perlstein Lab Tests Unorthodox Rare-Disease Plan

Xconomy San Francisco — 

DIYBio is a hot term these days, as more so-called citizen scientists gain access to biotechnology tools and “biohacker” lab spaces. But how about DIY drug discovery? Independent scientist Ethan Perlstein is trying to prove it’s possible.

After a year bootstrapping an ambitious project to find treatments for diseases too rare to attract drug industry attention, he just raised a $2 million seed round for his Perlstein Lab, with a chunk of the cash coming from publicly traded rare disease firm Retrophin (NASDAQ: RTRX).

In 2013, Perlstein had his doctorate from Stuart Schreiber’s Harvard University lab and five years of post-doctoral work at Princeton University under his belt, but he couldn’t land an academic position. He didn’t want to work for a drug company, so armed with the networking tools of social media, he moved to the San Francisco Bay Area to build his own lab, unaffiliated with a school, company, or other institution.

He now has five full time employees, and two consultants who bring decades of industry experience: former medicinal chemist John Alan Tucker and current Cleave Biosciences CEO Laura Shawver.

“Ethan’s enterprising style may be indicative of the new generation of entrepreneurs,” Shawver told Xconomy. “Twitter, crowdsourcing, more innovative financings than what is traditional. He cold-called me via e-mail, which normally would not even be opened, so you can see the level of engagement that he has.”

His startup has space at [email protected], a San Francisco incubator affiliated with the University of California and sponsored by Janssen Labs, an arm of health care giant Johnson & Johnson.

Of the $2 million seed money, New York-based Retrophin and the Wilsey family foundation in San Francisco have combined to contribute about one-third. The rest has come from angel funders in increments of $10,000 to $400,000, Perlstein says.

Perlstein first caught Retrophin CEO Martin Shkreli’s attention on Twitter, and their exchange led to a meeting at the J.P. Morgan conference in San Francisco.

The Wilsey family, legendary in San Francisco society circles and the subject of a best-selling memoir, is also keen on cures for rare diseases. A great-granddaughter, Grace, has an extremely rare disorder caused by a mutation in the NGLY1 gene; her parents have created a rare-disease foundation with an A-list team of scientists, advisors and board members including Gilead Sciences (NASDAQ: GILD) chief scientific officer Norbert Bischofberger, Nobel laureate Shinya Yamanaka, 23andMe CEO Anne Wojcicki, Amicus Therapeutics (NASDAQ: FOLD) CEO John Crowley, and LinkedIn CEO Jeff Weiner. (The San Francisco Chronicle documented the Wilsey story in April, and The New Yorker had a feature about NGLY1 last week that caught national attention.)

Perlstein has made the NGLY1 disorder and Niemann-Pick type C disease his lab’s first priorities. Both are lysosomal storage disorders, in which patients don’t properly produce the enzymes that break down fatty materials in their cells, but they are far rarer than the ones such as Gaucher’s and Fabry disease that fueled the work of companies such as Genzyme and BioMarin Pharmaceutical (NASDAQ: BMRN).

Perlstein’s heavy Twitter networking and angel crowdfunding match the unorthodoxy of his scientific vision, a blend of old and new concepts. His drug discovery platform is based on what he has dubbed “evolutionary pharmacology.” He believes disease-causing genes can be found across species, and those similarities can make low-cost organisms—yeast, fruit flies, nematodes, and zebra fish—great ways to test drugs.

So Perlstein is building his lab around those four “lower” organisms, has hired full-time scientists with … Next Page »

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