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: 1⃣ Of course the example I know best is @epalrestat which started out with PMM2-CDG yeast and worm avatars followed by patient fibroblast validation leading to a single-patient IND/pioneer study -- not to mention a novel clinical biomarker/primary endpoint onlinelibrary.wiley.com/doi/abs/10.100…

Here are the 5 common tactical errors I see made over and over again by recently diagnosed, emotionally overwhelmed families without monetary resources or connections who are nonetheless pursuing cure-focused research as quickly as humanly possible: 1) Funding follows the plan. Not the other way around!

You need at the outset a multi-modality, diversified portfolio approach and commercialization plan — aka a Cure Roadmap.

(That’s why I’m assembling a Cure Sherpa force at @PerlaraPBC so families start off on the right foot)

Thoughts on decentralized biotech 🧬📲

The era of DeBi is here 🧵👇 First off, I didn’t arrive here by accident. I’ve been thinking deeply about open science, community building, company building, and the long tail of disease for a while.

The mainstreaming of crypto and the pandemic have done the rest in terms of shaping my thinking.

On this day 25 years ago, Christopher Reeve sustained a spinal cord injury; today there is still no fix, no cure.

COVID19 abruptly cut short the SCI Roadmap project inspired by Reeve’s unrelenting cure vision.

People in chairs are still waiting for a superhero.. I spent the past year immersing myself in all aspects of the SCI community: people living with the injury, their caretakers and advocates, researchers and clinicians, foundations and other stakeholders.

I reached out to everyone in SCI who would share their feedback with me.

Here are the slides from my #ReeveSummit2020 keynote called “The Future of SCI (Spinal Cord Injury) Research” We often begin our presentations with a remembrance of Christopher Reeve, and this quote of his in particular has reverberated through the decades.

In fact, this year marks the 25th anniversary of Christopher’s injury. I was a sophomore in high school at the time.

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: I believe a kiss of death for @PerlaraPBC was its ahead-of-its-time status as the first biotech Public Benefit Company. While that excited at least one investor (@mcuban) it turned off 99% of VCs who either thought their upside would be capped or didn’t want the biz model risk.

VCs spend a lot of time on Twitter rhapsodizing A+ founders.

Equally important is what makes an A+ investor.

Here are my top criteria: A+ investors unfailingly engage regardless of the size of their investment.

Engagement can take the form of replies to email updates, 👍/likes/favs/RTs, phone calls, and of course face time.