Ethan Perlstein

@eperlste

Founder+CEO , W16, orphan drug discoverer, evolutionary pharmacologist, threenage negotiator

Oakland, CA
perlara.com
Joined January 2011
631 Photos and videos Photos and videos

Tweets

You blocked @eperlste

Are you sure you want to view these Tweets? Viewing Tweets won't unblock @eperlste

  1. Pinned Tweet
    Mar 13

    Rare and especially ultra-rare disease communities do the impossible not despite but because of their small numbers. I call it Economies of Rare

    1 reply 29 retweets 68 likes
    Undo
  2. 12 hours ago

    BOS ✈️ SFO after very productive meetings. TGIF!

    17 likes
    Undo
  3. Retweeted
    Apr 19

    On April 25th, we're hosting at in convo w/ entrepreneurs from University of California about building a successful bio venture and launch of YC Bio. Seats are going very quickly, RSVP here:

    Image
    12 retweets 21 likes
    Undo
  4. Retweeted
    Apr 19

    let me write that in 21st century for u: czi=good oligarch calico=bad oligarch

    A study in contrasts: CZI = open science Calico = closed science
    2 retweets 9 likes
    Undo
  5. Apr 19

    A study in contrasts: CZI = open science Calico = closed science

    Image
    Today we announced funding for 85 one-year projects that will focus on developing an open set of computational tools, algorithms, visualization methods and benchmark datasets for scientists working on the . Learn more about the projects:
    1 reply 2 retweets 5 likes
    Undo
  6. Retweeted
    Apr 19

    If a PAG partners with Perlara to create yeast, worm or fly models, these models are freely available for any and all non-commercial use, and we will license for commercial use. Bottom line is disease models are made to be shared, not hoarded.

    2 replies 5 retweets 18 likes
    Show this thread
    Image
    Show this thread
    Undo
  7. Apr 19

    On this topic Bruce and I are in wildly violent agreement. If a company whips out their premoney valuation ($2B!) in the first sentence of a press release, it’s a good sign there’s no there there. Please prove us wrong!

    Wow. Peak bio-AI? Skeptical at $21M, much less $2.1B... then again, they are “disrupting pharma by lowering costs, decreasing failure, and increasing the speed“... CADD has never made those claims before, or has it? Hope they prove me wrong but remember: biology is hard.
    Show this thread
    1 reply 10 likes
    Undo
  8. Retweeted
    Apr 19

    A publicly funded SAB (with matching Pharma $) that assesses R&D opportunities on behalf of patient advocacy groups is an excellent idea!

    Replying to @andy_utoronto @dgmacarthur and 4 others
    it’d be very cool to put together a publicly funded sab to advise all pags with $
    1 reply 4 retweets 10 likes
    Undo
  9. Apr 19

    If a PAG partners with Perlara to create yeast, worm or fly models, these models are freely available for any and all non-commercial use, and we will license for commercial use. Bottom line is disease models are made to be shared, not hoarded.

    2 replies 5 retweets 18 likes
    Show this thread
    Image
    Show this thread
    Undo
  10. Apr 19

    Learned about a huge pain point for patient advocacy groups that sponsor research, namely mouse models. Academic lab gets PAG $, publishes mouse but won’t share it others, or just shares with PIs they know. Not cool. If you take PAG $, you’re committing yourself to open access.

    6 replies 5 retweets 17 likes
    Show this thread
    Image
    Show this thread
    Undo
  11. Retweeted
    Apr 19

    FDA ADCOMM VOTE: Is the benefit-risk profile of favorable for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome in patients 2 years of age and older? 13-Yes 0-No 0-Abstain PDUFA goal date is June 27

    1 reply 18 retweets 13 likes
    Show this thread
    Image
    Show this thread
    Undo
  12. Retweeted
    Apr 19
    Replying to

    The costs of awareness/education vs funding research are quite different. Some orgs start with the former because it's simply feasible.

    1 retweet 3 likes
    Undo
  13. Apr 19

    A publicly funded SAB (with matching Pharma $) that assesses R&D opportunities on behalf of patient advocacy groups is an excellent idea!

    Replying to @andy_utoronto @dgmacarthur and 4 others
    it’d be very cool to put together a publicly funded sab to advise all pags with $
    1 reply 4 retweets 10 likes
    Undo
  14. Retweeted
    Apr 19
    Replying to and

    Yes, I think this is exactly what's needed. Families starting advocacy groups need to be able to tap into unbiased scientific guidance on where to direct their effort and $.

    2 replies 3 retweets 6 likes
    Undo
  15. Retweeted
    Apr 19
    Replying to

    JJB was 'new school' driving the research to the brink of a trial. But now we have gone back to the old ways. Raising awareness for MPSIII- to find patients- to get pharma to fund our trial. Advocating to keep federal dollars flowing to the NIH and FDA so we can support research.

    1 reply 1 retweet 3 likes
    Undo
  16. Retweeted
    Apr 18

    Let’s put it this way: gene therapy better work well for a mechanistically straightforward blood-based monogenic deficiency otherwise there’s not no hope for the other 99% of diseases

    Image
    Image
    Gene therapy prevails again: transfusion-dependent β-thalassemia achieving cures in place of allogenic donor transplant by and colleagues
    Show this thread
    2 replies 16 retweets 56 likes
    Undo
  17. Apr 18

    Let’s put it this way: gene therapy better work well for a mechanistically straightforward blood-based monogenic deficiency otherwise there’s not no hope for the other 99% of diseases

    Image
    Image
    Gene therapy prevails again: transfusion-dependent β-thalassemia achieving cures in place of allogenic donor transplant by and colleagues
    Show this thread
    2 replies 16 retweets 56 likes
    Undo
  18. Retweeted
    Apr 18

    I am excited to report that my first 1st author manuscript has been accepted for publication in the G3 Journal. This is the first journal publication of and there will be many more to come in the near future!

    5 replies 9 retweets 70 likes
    Undo
  19. Apr 18

    Incumbent PAGs tend to be national orgs driven by Boards with a grayer leadership that doesn’t have a ticking clock mentality. OTOH newer PAGs are driven by one or more families racing against the clock while also caregiving, and they’re not afraid to use the c word - cure

    2 retweets 6 likes
    Show this thread
    Image
    Show this thread
    Undo
  20. Apr 18

    You can tell which patient advocacy groups are “old school” because they focus on policy and awareness. PAGs that are “new school” focus on research and commercialization.

    6 replies 2 retweets 17 likes
    Show this thread
    Image
    Show this thread
    Undo
  21. Apr 18

    SFO ✈️ BOS for meetings with our collaborators at and

    3 retweets 13 likes
    Undo

@eperlste hasn't Tweeted yet.

Loading seems to be taking a while.

Twitter may be over capacity or experiencing a momentary hiccup. Try again or visit Twitter Status for more information.

    You may also like

    ·