Ryan Ponzi

1000 Cures Accelerating parent-powered cures for rare pediatric diseases one family, one program, one cure at a time. What is 1000 Cures? First accelerator purpose-built for rare disease family founders, the most motivated operators in biopharma. How does it work? Resourcing lean startups to reach FDA approval on capital efficient, expedited timelines compared to industry average. Why now? To end pediatric rare disease and secure priority review vouchers, which typically sell for 100M – 200M USD.

@PRVWatch Curious to know costs of law suit.

Here’s my previous thread explaining the history of the Sezaby approval, PRV denial and their success in overturning the decision.

@PerlaraPBC Dashboard incoming

Perlara 3.0 site launching soon

If true, it will be interesting to see if trad biotech still has the same incentive to sell the PRVs, rather than keep them, since they are still likely spending $150m+ development costs. This is exactly why we run lean at @1000cures. We help FAMCOs keep development costs low so even if the voucher price drops by 50% we can still deliver good returns and go after ultra-rare diseases with very small markets (e.g. n=10, n=20)

I’d theorise that the relatively high number of new PRVs issued this year and continued issuances of the Commissioner’s National Priority Vouchers are pushing PRV prices downwards.

.@1000cures team coining term “FAMCO”

The mission of @1000cures is clear: Accelerate family-founded companies (FAMCOs) toward parent-powered cures

Just wrapped up an inspiring few days at the 2026 CDG Scientific & Family Conference in Orlando with @eperlste Huge thanks to the entire CDG community — families, researchers, clinicians, and advocates for the powerful conversations, shared insights, and relentless drive to advance treatments and care for CDG patients. The energy and collaboration at this conference reinforce why @1000cures exists: to equip rare disease families with everything they need to go from idea to impact faster. If you’re a CDG parent, researcher, or advocate and we didn’t connect yet — reach out. The next 1000 cures come from communities like these.

@GeneInvesting @eperlste Send it

@eperlste $NTLA tomorrow is curesmaxxing

Who else is curesmaxxing?

And yet it seems like everyone is still sleeping on the PRV renewal 🤷 3.5 years is a rounding error for Pharma but it’s an eternity for entrepreneurial rare disease parents with clinical-stage assets already in hand 💪 I can tell you this: @1000cures is wide awake! LFG

7 rare pediatric disease priority review vouchers have been issued so far this year, already more than the 6 issued across all of last year.

Me to Claude: "Make no mistakes. DO NOT HALLUCINATE. YOU ARE AN EXPERT SOFTWARE ENGINEER WITH 15 YEARS OF EXPERIENCE."

@eperlste 1000cures.com

I've met so many entrepreneurial rare disease parents over the years that I’ve lost count. They have moved or are moving medical mountains to save their kids and others like them. But rare parent founders get no love from institutional players. Those days are done. Science, talent and compute are no longer limiting. Priority Review Voucher alpha makes the economics work. The missing spark is access to cure capital. That's why I’m incredibly pumped to relaunch @1000cures with my cofounder @ryan_1000cures! Ryan and I teamed up to create a “YC for Rare” that unites our biotech houses with complementary expertises, experiences and networks. 1000 Cures is an accelerator for lean startups led by parents on mission to cure pediatric rare diseases. We ride at dawn. Let’s go!

@eperlste Great! And much needed Signed, entrepreneurial rare disease parent