Ethan Perlstein @eperlste Twitter profile

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Ethan Perlstein

2 years

PMM2-CDG pioneer Maggie has been on epalrestat for almost two years as part of an ongoing single-patient IND study. Her first dose was January 2020. See the results with your own eyes 👇

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Ethan Perlstein

@eperlste

10 months

@RyanFMandelbaum @JacquelynGill If libraries were smart they’d all have popup cafes and support a popup ecosystem! Why does it have to be one (legacy choice) or the other?

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Ethan Perlstein

@eperlste

5 years

It is with a broken heart but defiant spirit that I announce the wind down of @PerlaraPBC . After 5 unforgettable years this ambitious, hopeful, necessary experiment is over. We lived as the first biotech public benefit company and we die as the first biotech PBC.

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Ethan Perlstein

@eperlste

5 years

So excited to announce my next patient-inspired adventure as Chief Scientific Officer @ReeveFoundation !! It’s time to retire the wheelchair. I can’t wait to help translate basic science into new treatments and ultimately a cure for spinal cord injury!

Christopher & Dana Reeve Foundation appoints Ethan Perlstein, Ph.D., as Chief Scientific Officer

/PRNewswire/ -- The Christopher & Dana Reeve Foundation announced today the appointment of Ethan O. Perlstein, Ph.D., as the Reeve Foundation's first Chief...

www.prnewswire.com

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Ethan Perlstein

@eperlste

4 years

It’s only n-of-1 (as of now) but I still can’t totally wrap my head around the concept that we discovered a drug that’s working in an actual human being! And she’s the first child with her rare disease to ever take this drug. I just want to do it again, and again, and again..

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Ethan Perlstein

@eperlste

5 years

We welcomed baby Luca to the world at 10:55am PT this morning!!! 6 lbs 7 ounces Scheduled C section went perfectly. Thanks Kaiser Walnut Creek docs and nurses! Mom is recovering. And Kai is excited to finally be a big brother.

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Ethan Perlstein

@eperlste

2 years

Are there any scientists left in academia after today’s announcement?

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Ethan Perlstein

@eperlste

2 years

How it started (2014): How it's going (2022):

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Ethan Perlstein

@eperlste

1 year

Someday grad students will realize that being a postdoc sucks unless you’re academia or bust. The market will reward PhDs with great salaries and great science. There’s no reason to do a postdoc unless you want to be a professor.

Leslie Vosshall PhD

@leslievosshall

1 year

🧵📢 We need to retain talented postdocs in academic science. I'm happy to say that @HHMINEWS has taken a big step forward this week. $70,000 That's the new starting minimum salary for all postdocs employed in HHMI labs (1/4)

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Ethan Perlstein

@eperlste

2 years

STUDY MAY PROCEED!!!

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Ethan Perlstein

@eperlste

5 years

As one of our dying acts, I am open-sourcing all data, discoveries from the #NGLY1 PerlQuest which was denied a commercial home, starting with this preprint describing 3 therapeutic approaches: NRF2, catecholamine and anti-inflammatory pathway activation

Drug screens of NGLY1 Deficiency worm and fly models reveal catecholamine, NRF2 and anti-inflamma...

N -glycanase 1/ NGLY1 Deficiency is an ultra-rare and complex monogenic glycosylation disorder that affects fewer than 40 patients globally. NGLY1 Deficiency has been studied in model organisms such...

www.biorxiv.org

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Ethan Perlstein

@eperlste

6 years

Hey @bloomberg what kind of bullshit sexism is this? You list me as Dr but my PhD colleagues @NinaDiPrimio and @IyerSangeetha are listed as Ms? (h/t @jessplao )

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Ethan Perlstein

@eperlste

4 years

A year ago we were fundraising for @PerlaraPBC ’s Series B. I failed my team and our partners. I couldn’t tell a story that excited VCs. Now that I’m firmly settled and over the moon in my new role — and I’ve had a year to reflect — here’s the deck, with lessons learned:

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Ethan Perlstein

@eperlste

2 years

Yesterday FDA said the three magic words: Study May Proceed Here’s how we went from a hit in yeast and worm drug repurposing screens to a single-patient IND study to the start of a pivotal Phase 3 in less three years..

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Ethan Perlstein

@eperlste

3 years

Thoughts on decentralized biotech 🧬📲 The era of DeBi is here 🧵👇

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Ethan Perlstein

@eperlste

7 years

With this round (finally!) behind us, a few choice learnings from pitching VCs. Listen up pre-A BioPharma companies, this thread is for you:

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Ethan Perlstein

@eperlste

1 year

Not sure which cracks me up more about academic conferences: Assistant professor who acts like they - let's be honest, HE - has already won the Nobel Prize. OR Professor emeritus who's an actual Nobelist but looks like they came in off the street for the free coffee.

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Ethan Perlstein

@eperlste

4 years

Let’s just call masks “cloth vaccines” in blue states and “freedom shields” in red states.

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Ethan Perlstein

@eperlste

3 years

It's hard to describe the feeling of having achieved clinical efficacy and safety in humans. You feel differently about yourself as a scientist making real-world impact, even if limited to a few patients at first. You start to believe the impossible is possible.

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Ethan Perlstein

@eperlste

3 years

Just got a text from the parent of a child who started taking epalrestat 3 months ago, and watched the video of his son walking his first 3 unassisted steps. 🤯😂🥰

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Ethan Perlstein

@eperlste

3 years

I've been waiting for years to announce news like this on a #RareDiseaseDay . Maggie's been epalrestat for 14 months now and the evidence is in: the drug is working better (and more safely) than we ever imagined! 🧵👇

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Ethan Perlstein

@eperlste

7 months

Mike Eisen just got canceled for dissent by his own people. While I don’t agree 100% with his politics and we’ve had our fair share of Twitter tussles over the years, his rock-solid commitment to open access and open science has endeared him to me and countless scientists around…

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Ethan Perlstein

@eperlste

6 years

Here’s a crazy idea for #PostdocAppreciationWeek : starting now, every NIH-funded PI must pay their postdocs at least 75K per year, and at least 95K per year in places like Boston and the Bay Area.

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Ethan Perlstein

@eperlste

2 years

$2M SBIR Direct to Phase 2 grant just got final funding approval for the @epalrestat trial — woohoo!!

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Ethan Perlstein

@eperlste

6 years

Your annual reminder that Martin Shkreli (in jail awaiting sentencing) and Elizabeth Holmes (bankrupt) were once #30under30 !

Matthew Herper

@matthewherper

6 years

Here's a link to the #30Under30 in healthcare for 2017!

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Ethan Perlstein

@eperlste

2 years

Last week was a scientific first for drug repurposing! Monday AM: receive yeast hit list Monday PM: yeast hits independently confirmed in patient derived iPSC astrocyte/neuron Tuesday: family orders top hit OTC, doses child Wednesday: first babbling since it was lost at 8 months

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Ethan Perlstein

@eperlste

5 years

First one in, last one out. Goodbye 6000 Shoreline Court. It was fun while it lasted.

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Ethan Perlstein

@eperlste

1 year

My biggest and proudest professional achievement just happened today: “First patient first dose” for the @epalrestat Phase 3 trial for kiddos with PMM2-CDG — let’s do this!

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Ethan Perlstein

@eperlste

5 years

My Mom died in 2002. Shoutout to all those who lost their mothers too soon. #HappyMothersDay

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Ethan Perlstein

@eperlste

5 years

I can’t possibly convey the depth and breadth of gratitude I feel toward everyone who rooted for us over the years. Thank you. A million times thank you. I’ve livetweeted the odyssey this far so I’m not going to stop now. Onward!

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Ethan Perlstein

@eperlste

5 years

Just had coffee with a friend and biotech CEO. They’re moving operations out of the Bay Area and California entirely, decamping for sites back East, eg RTP. It’s gotten to the point where you have to be a Pharma or enjoy setting cash on fire to operate a lab in the Bay Area.

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Ethan Perlstein

@eperlste

2 years

What if rare disease families/foundations pooled their $$$, rented lab space, built common lab infrastructure, hired scientists to do assay dev and standardized drug screening at a fraction of CRO costs and many times faster than academic labs? Pirate pop up labs for cures!

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Ethan Perlstein

@eperlste

5 years

💵 Cashflow positive again this month PerlQuest revenues: 2016 - $60,000 2017 - $190,000 2018 - $1,275,000 to date

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Ethan Perlstein

@eperlste

3 years

I lost my Mom in 2002 to a decades-long struggle with addiction and mental illness. Today she would have turned 70. I hope I’ve done you proud!

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Ethan Perlstein

@eperlste

1 year

We have access to our SVB funds!

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Ethan Perlstein

@eperlste

5 years

My antidote to imposter syndrome is simple: partner with rare disease families. Nothing clarifies my mind and focuses my heart like knowing my higher purpose is to save a life when everyone says it’s impossible to do.

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Ethan Perlstein

@eperlste

6 years

A year ago, our Series A fundraise took flight. At the time I was (naively) optimistic. 9 bruising months and 2 pitch pivots later, we closed a skinny A last October. I thought it would be useful to #sharethedeck , slide by slide with commentary

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Ethan Perlstein

@eperlste

2 years

Too many doctors who see rare disease patients seem to believe that drug repurposing rarely works. I think that is a perception based in risk aversion not a conclusion supported by the science. Here are 5 not-talked-about-enough examples of rare-purposing working:

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Ethan Perlstein

@eperlste

1 year

All the techbio VCs tripping over themselves with their new AI platform portcos do realize that the ultimate bottleneck is clinical trial recruitment, right? It doesn’t matter how kickass your tech is. If you can’t recruit study participants your platform will be a museum piece.

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Ethan Perlstein

@eperlste

6 years

“Congratulations, your manuscript has been accepted!” Hereby ending my 6 year publication slump. More preprints and papers on the way..

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Ethan Perlstein

@eperlste

1 year

An over-the-counter drug sitting in practically every medicine cabinet in the world improves symptoms of autism in kiddos with an ultra-rare genetic disease. Oh, and we used a fly model of the disease to discover the drug. This is why drug repurposing/multipurposing is going 📈

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Ethan Perlstein

@eperlste

3 years

Interesting see the bimodal reactions of scientists to the Altos Labs announcement: 1) Boo selfish billionaires who want to watch to the earth burn and inequality grow wider! 2) Wow a massive injection of unexpected funds to work on biology’s biggest problem!

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Ethan Perlstein

@eperlste

3 years

The biotech founder narrative is finally changing: Open networks, free sharing of knowhow, operators backing operators, no academic or investor gatekeepers, no credentialism. Love it!!

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Ethan Perlstein

@eperlste

3 years

You thought @PerlaraPBC died a quiet death 2 years ago... Well turns out genetic diseases didn’t get the pandemic memo. A new family is introduced to me every week. So, Perlara is coming back to life! I just need to find lab space to meet the surging demand for new PerlQuests.

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Ethan Perlstein

@eperlste

4 years

Noticing some interesting new trends among @ycombinator S20 bio companies that speak to a new kind of biotech founder and a new kind of biotech investor.🧵👇

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Ethan Perlstein

@eperlste

3 years

Date mRNA, marry small molecule

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Ethan Perlstein

@eperlste

5 years

Today was my first onsite visit @ReeveFoundation . Pictured below is Christopher Reeve’s chair. I will be thinking of it each day moving forward, a daily reminder of my new mission.

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Ethan Perlstein

@eperlste

7 years

Brief modern history of biotech trends: 2010-11: Repurposing 2012-13: Big Data 2014-15: Cloud Labs 2016: CRISPR 2017: AI the shit out of it

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Ethan Perlstein

@eperlste

2 years

The first AI-powered drug platforms arrived on the scene a decade ago promising “faster, better, cheaper” — and yet none of them has gotten a novel chemical entity hitting a novel target across the approval finish line.

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Ethan Perlstein

@eperlste

5 years

If the rare disease community will have me, I want a second act. I don’t know what other scientific problem is worthy of my passion and attention than the unmet medical needs of rare disease communities around the world. I know there are others out there like me.

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Ethan Perlstein

@eperlste

3 months

Want to see what a direct-to-patient biotech pipeline looks like? 22 programs funded by pioneer families, 12 in various stage of 1-to-N clinical studies, and half of hits are clinically actionable nutraceuticals. This is how you start to do drug repurposing at scale.

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Ethan Perlstein

@eperlste

4 years

Contact tracer will be 2020's Job of the Year.

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Ethan Perlstein

@eperlste

4 years

Last day of my 30s....Started the decade single, childless and still an academic trainee. The last ten years have been a helluva ride!

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Ethan Perlstein

@eperlste

5 years

Single patient IND submitted. The CDG embers of Perlara burn on.. I should mention again that we made this repurposing discovery in less than 6 months using yeast, worms and patient fibroblasts. At a cost of ~$200K. No AI or $100Ms of capital required. And it’s truly scalable.

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Ethan Perlstein

@eperlste

11 years

We used jargon instead of plain English to prove that a decade of grad school and postdoc made us smart. #overlyhonestmethods

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Ethan Perlstein

@eperlste

3 years

As a therapeutic modality, small molecules get no respect. Quietly doing yeoman’s work, even the heavy lifting of other modalities, but without fanfare or glitz. Small molecules need a rebrand — the word “small” needs to go. Any ideas?

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Ethan Perlstein

@eperlste

2 years

Listening to @ycombinator President @gralston (my group partner in W16!) giving the State of YC address. His last before @garrytan takes the reins. 3500+ startups over 17 years 9000+ founders 300+ companies > $150M 80+ YC alum valued at over $1B Total YC Co’s valued > $600B

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Ethan Perlstein

@eperlste

6 years

A founder friend is raising her Series A and just asked me for a list of the VCs I pitched last year, so I thought I'd just tweet out my investor rejections and save everyone the trouble of asking! First up: tech VCs/Sand Hill Road

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Ethan Perlstein

@eperlste

5 years

Next adventure update: I’m interviewing for a once-in-a-lifetime opportunity. Hope to be able to say more about it soon. I’ve been consulting for a few families as a CSO for hire on their n-of-1 cure odysseys and will share more in the weeks ahead.

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Ethan Perlstein

@eperlste

1 year

Aren’t billionaires tired of having their names plastered on some university building? So 20th century.. If they crave real legacy and being remembered by posterity, I have an idea for them: a “1000 Cures Fund” by the ultra-rich for the ultra-rare.

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Ethan Perlstein

@eperlste

8 years

Academia is now a cult of productivity. But the # of papers one publishes is not a measure of knowledge and/or utility created for humanity.

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Ethan Perlstein

@eperlste

5 years

Tip to VCs: don’t ghost on founders, especially after multiple warm intros and double especially after the first pitch call/meeting. If you don’t have the capacity to reply to or send emails with professionalism and timeliness, you’re doing it wrong

Uriri "Ulili" Onovakpuri

@unolil

5 years

Tip to Founders: Be nice to ALL investment team members regardless of their role. Today's Associates are tomorrow's Partners.

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Ethan Perlstein

@eperlste

6 years

Remember the ALS Ice Bucket Challenge that netted @alsassociation $120M? They spent most of that massive war chest on research which is now culminating in clinical trials, right? WRONG. They’re sitting on 80% of the windfall. How many ALS patients have died waiting for action?

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Ethan Perlstein

@eperlste

3 years

Waiting for the DAO that will collaborate and fundraise to extend the healthy lifespan of the most studied eukaryote on Earth by 10-100X: a yeast cell. It’s a science project that has no obvious business case today but if it worked it would have multiplicative enabling effects.

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Ethan Perlstein

@eperlste

6 years

It’s endlessly amusing to see tech VCs appoint themselves philosopher kings with 7-minute-read Medium posts about “radical empiricism” and other such malarky when 10-15 years ago they would have been anonymous traders on Wall St or engagement managers at McKinsey.

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Ethan Perlstein

@eperlste

1 year

To all the people casting aspersions on biotech startup founders for not managing multiple bank accounts while trying to make new medicines — like @VivekGRamaswamy who knows better — I have a simple message: FUCK YOU

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Ethan Perlstein

@eperlste

5 years

The outpouring of love for @PerlaraPBC has touched me to the core. 🙏 I wish VCs had loved the company as much as everyone else. Sad that an unabashedly rare disease discovery engine — even one with clinical-stage assets — wasn’t seen as compatible with venture scale returns.

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Ethan Perlstein

@eperlste

9 years

I'm a Dad!!!

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Ethan Perlstein

@eperlste

6 years

I know I’m doing my job right when rare disease parents tell me: “you think like us.” Not a day goes by that I don’t imagine myself in their shoes.

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Ethan Perlstein

@eperlste

4 years

If all the “we use AI to discover drugs!” companies have been identifying new lead compounds in the blink of an eye and shortening the time to IND and making all the other claims they make in press releases — for years now — then where all the FDA approved AI discovered drugs??

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Ethan Perlstein

@eperlste

2 years

Going to spend most of #JPM22 week preparing for our pivotal Phase 3 trial at Mayo Clinic. No press releases, no presentations, no fundraising announcements. Just nose to grindstone: bringing a new medicine to patients with unmet need. That’s what it’s all about it, right?

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Ethan Perlstein

@eperlste

9 years

Not to be outdone by @nature , @sciencemagazine announces new #OpenAccess partnership w/ @Snapchat : you have 30 seconds to read any paper.

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Ethan Perlstein

@eperlste

6 years

I get asked a lot about Perlara’s fundraising history and my decision-making process. So, here it is in one thread:

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Ethan Perlstein

@eperlste

1 year

@oneunderscore__ @stephaniemlee @JoyAnnReid Dude, who cares expect journos who lost their precious blue checks? You still have your 400K+ followers. All those people still like you. But keep dunking on the platform that gives you a massive audience FOR FREE 🙄

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Ethan Perlstein

@eperlste

6 months

~40 drugs are approved by FDA each year. That number has been essentially flat for decades. One day in the future, individual companies/teams — and one day even individual operators — will develop 40 medicines each year. We’ll look back on today like the Stone Age.

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Ethan Perlstein

@eperlste

10 months

@RyanFMandelbaum @JacquelynGill I would love to see those options in Oakland. If they exist in my back yard and I don’t know another them, someone please me!

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Ethan Perlstein

@eperlste

8 years

I repeat this at every conference I go to: all grad students and postdocs need to be on Twitter. #ASHG16

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Ethan Perlstein

@eperlste

8 years

Super thrilled to finally announce that @PerlsteinLab is a YC company!

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Ethan Perlstein

@eperlste

6 years

Best conference poster title EVER 😂👍🙌

SaraH Zanders 🎃💀

@ZandersLab

6 years

María Angélica Bravo Nuñéz presenting ‘wtf causes aneuploidy’ at FASEB Yeast chromosome biology and cell cycle meeting

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Ethan Perlstein

@eperlste

4 years

If you’re a professor and you’ve got the entrepreneurial spirit plus an idea (or ideas!) that you’ve been incubating for years, now is actually the perfect time to leave academia to start a biotech startup.

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Ethan Perlstein

@eperlste

5 years

I announced the wind down and virtualization of @PerlaraPBC on Rare Disease Day. 6 months later, two of our PerlQuests are entering the clinic — and the papers describing how yeast, worms and flies made this first-of-its-kind drug repurposing possible will be published soon.

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Ethan Perlstein

@eperlste

2 years

Why did it take an outsider like @mcuban to shake up drug pricing? Where was that leader from Pharma?? Seems like successful bio CEOs go on to become VCs or sit on a bunch of Boards.. If today’s expensive innovations aren’t tomorrow’s cheap generics, the system is broken.

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Ethan Perlstein

@eperlste

3 years

@WillManidis If you have revenue Newtonian accounting rules apply. If you don’t have revenue quantum accounting rules apply. Say hello to Schrödinger's startup

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Ethan Perlstein

@eperlste

5 years

So glad to hear Perlara people are landing on their feet at amazing jobs or are interviewing at amazing places. All their employers should consider themselves supremely lucky. I’ve never worked with a better group of humans. 🙌 ❤️

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Ethan Perlstein

@eperlste

1 year

For fuck's sake: micromolar is µ not u!!! You can't be a real scientist and keep getting this wrong. Option + M, people, Option + M....jfc 🤦‍♂️

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Ethan Perlstein

@eperlste

4 years

Drug repurposing is cool again thanks to #COVID19 .

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Ethan Perlstein

@eperlste

3 years

Founder-led Bio movement, welcome to San Francisco!

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Ethan Perlstein

@eperlste

3 years

Paper accepted for publication!!

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Ethan Perlstein

@eperlste

8 years

There is hope after the Postdocalypse. Assemble an exceptional team and anything is possible

Y Combinator

@ycombinator

8 years

Perlara (YC W16) signs rare disease pact with Novartis

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Ethan Perlstein

@eperlste

6 years

We just signed our 11th PerlQuest contract — the most ambitious one yet! Our PerlQuest portfolio is 100% bootstrapped and poised for impact. Official announcement soon..

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Ethan Perlstein

@eperlste

5 years

Orphan Drug Designation/ODD application has been submitted. If granted, it will be the first time worm and fly data were the centerpiece of the animal/in vivo data package. #NGLY1 #DrugRepurposing

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Ethan Perlstein

@eperlste

2 months

Biotech startups in the future will be increasingly ultra-lean, direct-to-consumer, cash-flow positive, fully remote, and distributed first. Seems crazy to incumbents, but it’s happening whether they want it or not.

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Ethan Perlstein

@eperlste

1 year

Allow me to make the contrarian case for remote biotech 🧵👇

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Ethan Perlstein

@eperlste

7 years

I would NOT have been able to start a startup without Obamacare. #truefact

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Ethan Perlstein

@eperlste

4 years

@blekhman Hey kids! Let’s play a game called Study Section

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Ethan Perlstein

@eperlste

2 years

The “ultra rare problem” isn’t go away — in fact, it’s growing. If you’re a BioPharma with an orphan drug candidate that doesn’t have the resources or economic justification to advance toward the clinic, here’s why you need to partner your asset with a family or foundation 🧵👇

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Ethan Perlstein

@eperlste

5 years

Are you a CSO at a biotech or nonprofit foundation? Interested in forming a CSO community/network with me so we can swap notes on everything from science to venture philanthropy and everything in between?

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Ethan Perlstein

@eperlste

4 years

True to the original PerlQuest vision, we’ve created a jointly owned NewCo that will commercialize epalrestat if it’s approved for the treatment of PMM2-CDG. But first things first: we’re expanding the successful n-of-1 trial beyond Maggie. Onward!

Holly Carmichael

@CarmichaelHolly

4 years

n=1 trial for the treatment of PMM2CDG 6-mo Update Disclaimer: I’m mom to Maggie, age 6. This post is based on my interpretation of Maggie’s progress and should not be taken as medical advice. You should consult with your Dr about any treatment options or available drug trials.

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Ethan Perlstein

@eperlste

1 year

Imagine the world's first biotech investment fund that gives capital to rare genetic disease families racing to save their kids, lets them decide which modalities to work on, and then empowers them to execute like crazy faster and cheaper than anyone else in the world. If these…

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