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1000 Cures is the first accelerator built for FAMCOs:
family-founded biotech companies led by rare disease parents
family-founded biotech companies led by rare disease parents
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1000 Cures, Inc retweeted
Rare disease parent founders starting FAMCOs are the OG Pipeline-in-a-Person
This is amazing and love to see this kind of ingenuity rewarded. Pipeline-in-a-Person @Prof_Oak_
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Rare disease parent founders starting FAMCOs are the OG Pipeline-in-a-Person
This is amazing and love to see this kind of ingenuity rewarded. Pipeline-in-a-Person @Prof_Oak_
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1000 Cures, Inc retweeted
Founder-Mode Medicine: The Antidote to Rare Diseases
Every waking moment, a bolt of genetic lightning strikes an unsuspecting family. It strikes indiscriminately, without regard for background, income, or geography. A long tail of more than 10,000 rare diseases afflicts roughly 400 million people worldwide. Most families, understandably, are overwhelmed and unequipped to respond to a devastating diagnosis in time to matter.
Traditional pharmaceutical companies and life science investors have little appetite for small, fragmented markets when global blockbusters beckon. Most rare diseases are ultra-rare, affecting fewer than 1,000 patients worldwide. Some have patient populations you can count on both hands. But to the industry, the long tail is invisible.
Market failures fester. The rare disease community yearns for a pharma white knight but settles for Sir Ghost-a-Lot. It’s time to introduce the long tail to the invisible hand. Turns out the matchmakers—dare I say kingmakers—have been flying under the radar all along.
Sometimes fate messes with the wrong person.
A subset of rare disease parents are natural-born entrepreneurs: constitutionally incapable of taking no for an answer and unintimidated by uncertainty. Few have biotech operating experience or scientific training. By conventional wisdom, they’re the last people for the job.
The industry ignores them at its peril. These parents are relentless autodidacts, reading papers past midnight, cold-emailing researchers, organizing patient communities and, of course, fundraising. They somehow do it all with a capital efficiency and speed that would make Chinese biotech blush.
They look nothing like standard-issue biopharma management teams. But they share the rare, much-coveted traits—high-agency chief among them—of the most successful founders in any industry. The résumé is unconventional, but the founder DNA is not.
Investors prize founder-led companies because the founder is the beating heart of the organization and the embodiment of its mission. @paulg, co-founder of @ycombinator, the world’s most successful start-up accelerator, calls this hands-on style “founder mode.” Inspired by YC’s model for backing overlooked, undervalued founders, I’d extend Graham’s concept to medicine. After all, who embodies a mission more completely than a parent trying to save their child?
After more than a decade operating in rare disease, I’ve had the honor of working alongside dozens of parents going founder mode on their kid’s disease. Faced with a ticking clock and limited resources, they have spearheaded N-of-1 medicines time and again. In 2018, Julia Vitarello (@stopbatten) and @timyu MD, PhD, created milasen—the first drug custom-built for a single patient, Julia’s daughter Mila—in less than a year.
That was nearly nine years ago! Many parents have achieved medical firsts since, but the market discounts them as one-offs. A few, most notably John Crowley (@IAmBiotech), have turned a parent’s determination into commercial success. Yet the chasm between the Julia Vitarellos and the John Crowleys still yawns. How do we close it?
All the necessary conditions are in place. The science is no longer limiting. The talent most certainly isn’t. There’s even a market incentive: the Priority Review Voucher (PRV), created by Congress in 2012 to spur rare pediatric disease drug development.
Companies that win approval for a rare pediatric disease drug earn a voucher that entitles its holder to a priority review—shortening the standard 10-month @FDA review clock to six months—for any future drug application. Because four months can be worth hundreds of millions to billions in revenue—think GLP-1s—the vouchers can be sold to bigger drugmakers racing blockbusters to market.
According to @PRVWatch, recent sales have topped $200 million. The very appetite for blockbusters that left rare disease behind can now help bankroll the long tail at essentially zero cost to taxpayers.
The rate-limiting step for too many promising rare disease programs is access to cure capital. We should back parents as founders, not just applaud them as advocates. It should not fall to families to make medicines for their children. But when they step forward, we should meet them with the resources to create treatments and cures at scale.
The founders are locked in. It’s time for the capital to catch up.
Every waking moment, a bolt of genetic lightning strikes an unsuspecting family. It strikes indiscriminately, without regard for background, income, or geography. A long tail of more than 10,000 rare diseases afflicts roughly 400 million people worldwide. Most families, understandably, are overwhelmed and unequipped to respond to a devastating diagnosis in time to matter.
Traditional pharmaceutical companies and life science investors have little appetite for small, fragmented markets when global blockbusters beckon. Most rare diseases are ultra-rare, affecting fewer than 1,000 patients worldwide. Some have patient populations you can count on both hands. But to the industry, the long tail is invisible.
Market failures fester. The rare disease community yearns for a pharma white knight but settles for Sir Ghost-a-Lot. It’s time to introduce the long tail to the invisible hand. Turns out the matchmakers—dare I say kingmakers—have been flying under the radar all along.
Sometimes fate messes with the wrong person.
A subset of rare disease parents are natural-born entrepreneurs: constitutionally incapable of taking no for an answer and unintimidated by uncertainty. Few have biotech operating experience or scientific training. By conventional wisdom, they’re the last people for the job.
The industry ignores them at its peril. These parents are relentless autodidacts, reading papers past midnight, cold-emailing researchers, organizing patient communities and, of course, fundraising. They somehow do it all with a capital efficiency and speed that would make Chinese biotech blush.
They look nothing like standard-issue biopharma management teams. But they share the rare, much-coveted traits—high-agency chief among them—of the most successful founders in any industry. The résumé is unconventional, but the founder DNA is not.
Investors prize founder-led companies because the founder is the beating heart of the organization and the embodiment of its mission. @paulg, co-founder of @ycombinator, the world’s most successful start-up accelerator, calls this hands-on style “founder mode.” Inspired by YC’s model for backing overlooked, undervalued founders, I’d extend Graham’s concept to medicine. After all, who embodies a mission more completely than a parent trying to save their child?
After more than a decade operating in rare disease, I’ve had the honor of working alongside dozens of parents going founder mode on their kid’s disease. Faced with a ticking clock and limited resources, they have spearheaded N-of-1 medicines time and again. In 2018, Julia Vitarello (@stopbatten) and @timyu MD, PhD, created milasen—the first drug custom-built for a single patient, Julia’s daughter Mila—in less than a year.
That was nearly nine years ago! Many parents have achieved medical firsts since, but the market discounts them as one-offs. A few, most notably John Crowley (@IAmBiotech), have turned a parent’s determination into commercial success. Yet the chasm between the Julia Vitarellos and the John Crowleys still yawns. How do we close it?
All the necessary conditions are in place. The science is no longer limiting. The talent most certainly isn’t. There’s even a market incentive: the Priority Review Voucher (PRV), created by Congress in 2012 to spur rare pediatric disease drug development.
Companies that win approval for a rare pediatric disease drug earn a voucher that entitles its holder to a priority review—shortening the standard 10-month @FDA review clock to six months—for any future drug application. Because four months can be worth hundreds of millions to billions in revenue—think GLP-1s—the vouchers can be sold to bigger drugmakers racing blockbusters to market.
According to @PRVWatch, recent sales have topped $200 million. The very appetite for blockbusters that left rare disease behind can now help bankroll the long tail at essentially zero cost to taxpayers.
The rate-limiting step for too many promising rare disease programs is access to cure capital. We should back parents as founders, not just applaud them as advocates. It should not fall to families to make medicines for their children. But when they step forward, we should meet them with the resources to create treatments and cures at scale.
The founders are locked in. It’s time for the capital to catch up.
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We're proud that our CEO, @eperlste , contributed to the Milken Institute's Power Of Ideas series. Read his essay on backing rare disease parents as founders, "Founder-Mode Medicine: The Antidote to Rare Diseases," ahead of MIHealthSummit.
Read the full essay here: milkeninstitute.org/content-hub/po…
Read the full essay here: milkeninstitute.org/content-hub/po…
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1000 Cures, Inc retweeted
Mark Cuban wants to crack open the market for rare disease treatments statnews.com/2026/10/21/mar… via @statnews
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1000 Cures, Inc retweeted
Founder-mode medicine is the antidote to all diseases
People who cured / are curing their own cancer on X:
1/ @sytses - bone cancer, billionaire founder of Gitlab, developed 10+ personalized therapies for himself.
2/ @paul_conyngham - shrank his dog Rosie's mast cell cancer tumors after chemo and surgery weren't enough. Now backed by YC and Founders Fund
3/ @miriamgonp - stage 4 breast cancer, tumor sequenced, searching for vaccine manufacturer now
4/ @kevinace - stage 4 lung cancer, angel investor in 300+ companies, first drug stopped working after 18 months. Vaccine designed, now paying $300k out of pocket for the vaccine
5/ @masonbgrant: going founder mode on his his 32-year-old wife's rare liver cancer, sequencing her tumor with AI agents
6/ @ArunRLverma: former product leader at Atoms and Shopify, brain tumor, built a vaccine from his tumor. Now building an AI health agent startup based on what he went through
We're going to make it
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We have a new look but our mission is the same.
The new 1000cures.com explains how we back family-founded biotechs (FAMCOs), the parents building cures for their own children's ultra-rare diseases.
Our model is simple. When a rare pediatric drug is approved, it can earn an FDA Priority Review Voucher, and selling that voucher funds the next family's program. Cures fund more cures.
Our first FAMCO partnership will be announced this quarter! A family, a child, and a cure that's already in progress. Stay tuned. 👀
The new 1000cures.com explains how we back family-founded biotechs (FAMCOs), the parents building cures for their own children's ultra-rare diseases.
Our model is simple. When a rare pediatric drug is approved, it can earn an FDA Priority Review Voucher, and selling that voucher funds the next family's program. Cures fund more cures.
Our first FAMCO partnership will be announced this quarter! A family, a child, and a cure that's already in progress. Stay tuned. 👀
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1000 Cures, Inc retweeted
We've been quietly fundraising and evaluating FAMCOs the past few months -- big announcements dropping soon..
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We've been quietly fundraising and evaluating FAMCOs the past few months -- big announcements dropping soon..
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This is why 1000 Cures exists 👇
@eperlste @draparente @fidjissimo Imagine what could be accomplished if so many folks didn't have to expend this much of their own (limited!) energy and capital trying to solve their own medical mysteries! I do sometimes feel like I've got my own startup, but focus is managing my own personal immune system. 😅
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1000 Cures, Inc retweeted
I have to say I have tremendous admiration for Ethan and Ryan's @1000cures. They setup a biotech launchpad for parents of rare disease children working on cures. Arran is orders of magnitude too complex for a typical cure/drug otherwise I'd be banging on their door daily. They give me a lot of hope for an industry that all too often thinks about the same few major diseases (the bottom line) and forgets about the millions of suffering families (like mine) that don't fit the mold.
hey @DarioAmodei if you want to cure all the diseases in 5-10 years you’ll have to start with the rarest of the rare.
there are literally 1000s of rare genetic diseases to chose from, like PKU which was featured in the Claude Science demo last month.
the thing is intelligence won’t be the rate-limiting step in your bold vision for saturating cure coverage. and god knows there’s no shortage of patients.
1 in 10 people are struck by genetic lightning. no family is spared. in the limit we will all be patients.
even as more patients gain access to frontier intelligence the rate-limiting step in cure creation is access to capital for drug development and clincial trials.
patients with the financial resources can go founder mode/N-of-1 on their disease, but what about everyone else?
it would be amazing if @AnthropicAI committed capital and compute for cures for rare genetic diseases.
let’s set a clear target:
1000 cures for 1000 rare genetic diseases in 10 years for $10B
crazy ambitious but achievable if the superabundance being promised actually materializes
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1000 Cures, Inc retweeted
Q2 had the highest no. of Priority Review Voucher sales ever in a quarter. The wide price range $180M–$220M shows just how unstandardised PRV pricing remains.
If you're a small biotech, you'd be kicking yourself if you sold for $180M only to see another voucher fetch $220M
If you're a small biotech, you'd be kicking yourself if you sold for $180M only to see another voucher fetch $220M
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1000 Cures, Inc retweeted
Why aren’t more people are talking about the Priority Review Voucher (PRV) program?
Folks act like it’s some kind of state secret.
It’s just a market exclusivity fee on global blockbusters that is paid by Big Pharma.
More accurately, it’s a consensual and democraticizing g Pharma to Baby Biotech aka FAMCOs.
One easy way to restore people’s trust in Pharma is for there to be more FAMCOs, or family-founded biotech companies, in the ecosystem.
So why aren’t we shouting PRV from the rooftops?
Are folks worried about an oversupply of PRVs that would crash the price?
@1000cures is taking the other side of that bet.
We believe that more PRVs will stimulate innovation by incentivizing competition for market share of global blockbuster medicines.
Big Pharma is vigorously competing to score approval on the next Ozempic and will happily fork over $200M+ for a chance at billions in sales.
It’s like buying a dollar for a dime.
And with longevity medicines on the horizon, the demand for market exclusivity on global blockbuster medicines will keep increasing.
Folks act like it’s some kind of state secret.
It’s just a market exclusivity fee on global blockbusters that is paid by Big Pharma.
More accurately, it’s a consensual and democraticizing g Pharma to Baby Biotech aka FAMCOs.
One easy way to restore people’s trust in Pharma is for there to be more FAMCOs, or family-founded biotech companies, in the ecosystem.
So why aren’t we shouting PRV from the rooftops?
Are folks worried about an oversupply of PRVs that would crash the price?
@1000cures is taking the other side of that bet.
We believe that more PRVs will stimulate innovation by incentivizing competition for market share of global blockbuster medicines.
Big Pharma is vigorously competing to score approval on the next Ozempic and will happily fork over $200M+ for a chance at billions in sales.
It’s like buying a dollar for a dime.
And with longevity medicines on the horizon, the demand for market exclusivity on global blockbuster medicines will keep increasing.
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1000 Cures, Inc retweeted
Recently launched the new PRV Watch website tracking the world of FDA Priority Review Vouchers. Check it out: prvwatch.com
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