Pediatric Rare Disease

Every child
deserves
a chance.

Over 400 million people live with a rare disease — most are children, and over 90% have no approved treatment. We exist to change that. 1000 Cures is the first accelerator built for the families and scientists racing to develop cures the system has overlooked.

Our Mission
10K+
rare diseases worldwide, fewer than 10% with an approved treatment
400M+
people affected globally, the majority of whom are children
3 years
for a family-led program to reach pivotal clinical trial — vs. 7-year industry average

Ending pediatric rare disease — one family, one program, one cure at a time.

Somewhere right now, a parent is learning their child has a disease affecting fewer than a thousand people in the world. No pharmaceutical company is working on a cure. No clinical trials. No approved treatment options. The only path forward is to build it themselves.

1000 Cures was founded to change that reality. We bring capital, scientific expertise, and operational support to the most motivated drug developers in the world: the parents and scientists who refuse to accept "there is nothing we can do."

What We Do

Lean. Capital-efficient.
Built for family founders.

We help family-founded biotech companies secure FDA designation and proceed through clinical studies — providing capital, contract research management, and regulatory strategy to approval. Our team has spent the last two decades doing this work in academic labs, biotech startups, and the largest rare disease IP portfolios in the industry.

We Resource

Capital, contract research management, regulatory strategy, and clinical operations oversight — at a fraction of the time and cost of traditional pharmaceutical development.

Families Lead

Parent-founders drive the science with single-purpose urgency no corporate incentive can replicate. We support, advise, and execute alongside them — but the mission is theirs.

Cures Reach Patients

Our work is measured in lives changed. Every program we accelerate is a chance for a child to grow up — and the foundation for a future where rare disease is no longer a sentence.

Get Involved

There is a place for you in
this mission.

Whether you are a family fighting for your child, a scientist with relevant expertise, an institution looking to partner, or anyone moved to support our work — we want to hear from you.

For Families

You are not alone.

If your family has been affected by a rare pediatric disease and you are exploring options for therapeutic development, we want to talk. We work with parent-founders at every stage — from early diagnosis to active clinical programs.

Contact our team →
For Scientists

Bring your expertise.

We collaborate with researchers across academic medical centers, contract research organizations, and biotech to advance therapeutic candidates. If you have relevant expertise in rare disease biology, drug repurposing, or clinical development, we'd like to connect.

Explore collaboration →
For Supporters

Help us reach more children.

Our work is funded by people and organizations who believe no child should be left behind because their disease is too rare to attract commercial interest. There are many ways to contribute — from amplifying our work to direct support.

Get in touch →
The Team

Two decades of rare disease experience.

1000 Cures was founded by two scientists who have spent their careers at the intersection of drug discovery and rare disease — one as a biotech entrepreneur, the other inside the largest rare disease IP portfolios in the industry.

Ethan O. Perlstein
Ethan O. Perlstein, Ph.D.
Co-Founder & CEO

Founder and former CEO of Perlara, the world's first biotech public benefit corporation. Y Combinator alum. Has spent over a decade working directly with rare disease families on therapeutic development.

Harvard Ph.D. Princeton YC W16
Ryan P. Ponzi
Ryan P. Ponzi, Ph.D.
Co-Founder & COO

USPTO Patent Agent and former patent agent at a Vault Top 5 IP law firm. Managed global IP portfolios for rare disease programs and evaluated key assets in multibillion-dollar biopharma acquisitions.

Boston College Ph.D. USPTO 10+ yrs life sciences
Join the Mission

Help us reach the next family.

Every conversation begins the same way — someone refused to accept that nothing could be done. Whoever you are, however you can help, that is where it starts.

Contact Us

We read every message personally.

Whether you are reaching out about your family, a research collaboration, press inquiry, or general support — we will get back to you.

Get in Touch

For all inquiries — families, researchers, partners, press, and supporters — this is the right starting point. We read every message personally and will respond within 2 business days.

Send us a message

Thank you. Your message has been received — we'll respond within 2 business days.

We typically respond within 2 business days. For urgent matters, please email info@1000cures.com directly.