Every child deservesa chance.

We give family-founded biotechs — FAMCOs — the capital, regulatory strategy and operations to take their child’s cure to approval.

For families

You are not alone.

Capital and hands-on support, built around your child’s program.

Start a conversation

For investors & partners

Back the most determined founders in drug development.

Explore the model

10K+

rare diseases — fewer than 10% have a treatment

400M+

people affected, most of them children

3 yrs

family-led path to a pivotal trial, vs. 7 on average

In the press

Founder-mode medicine.

Ethan Perlstein on why rare disease parents should be backed as founders.

Milken Institute · Power of Ideas · Oct 26, 2026

Founder-Mode Medicine: The Antidote to Rare Diseases

In this Power of Ideas essay, 1000 Cures CEO Ethan Perlstein argues that rare disease parents who go “founder mode” share the high-agency traits of the most successful founders in any industry, and should be backed as founders, not just applauded as advocates. He points to the FDA Rare Pediatric Disease Priority Review Voucher as a market incentive that can help bankroll the long tail of rare diseases, and says access to capital is now the rate-limiting step.

Ethan also speaks at the Milken Institute Future of Health Summit 2026 on the panel “Patients and Parents as PIs: The Rise of Family-Led Science,” Friday, Nov 13, 9:00–10:00 AM ET.

Read the essay

The model

A different way to fund cures.

Traditional drug development relies on big markets. Ultra-rare cures rely on the PRV.

Traditional drug development

1000 Cures · PRV-driven model

Cost to approval

TraditionalVery highBillions across a broad program.

1000 CuresLeanA fraction of the cost, family-led.

Trials

TraditionalLong and largeYears of multi-site enrollment.

1000 CuresFaster and smallerFocused studies in a handful of patients.

Indication

TraditionalBroadBuilt for the widest possible market.

1000 CuresNarrowOne ultra-rare pediatric disease.

Patients

TraditionalLarge populationsThousands to millions.

1000 CuresVery fewSometimes only a handful worldwide.

ROI driver

TraditionalDrug salesReturn depends on years of commercial revenue.

1000 CuresApproval → PRVNDA approval earns a voucher; its sale is the return.

Who pays

TraditionalPatients and payersCosts flow down to consumers.

1000 CuresThe PRV buyerBig pharma buys the voucher: a defined liquidity event.

How it works

Cures fund more cures.

Each approval can earn an FDA Priority Review Voucher (PRV). Its sale funds the next family’s program. The PRV model

Cures fund more cures.The PRV flywheel

Stage 01 / 05

Discover

~1 week

from first call to signed NDA (non-disclosure)

A parent founds a FAMCO, a family-founded biotech, and brings us their child’s program.

Stage 02 / 05

Select

60+

programs screened

Designation-first, M&A-style diligence: science, RPDD and PRV path, IP and freedom-to-operate, team.

Stage 03 / 05

Deploy

$500K–$3M

per asset

Staged milestone capital plus our operating team: CRO and CDMO management, regulatory strategy, IP.

Stage 04 / 05

Approve

2–3 yrs

from RPDD to NDA submission

Focused studies in a handful of patients. FDA approval of the rare pediatric drug earns a Priority Review Voucher.

Stage 05 / 05

Reinvest

$210M

most recent disclosed PRV sale (PRV Watch, Oct 2026)

Pharma buys the voucher. Proceeds are shared with the family-founders and fund the next FAMCOs.

Click any stage · cures fund more cures

PRV sale price: most recent disclosed sale, PRV Watch, as of Oct 2026.

Leadership

Two scientists. Two decades of expertise.

Meet the team

Co-founder & CEO

Ethan Perlstein, Ph.D. on LinkedIn (opens in a new tab)

Ethan received his Ph.D. in Molecular and Cell Biology from Harvard University (training under Professor Stuart Schreiber) and completed an independent postdoctoral fellowship at Princeton. During his academic training, he developed an evolutionary approach to drug discovery in model organisms. This methodology anchored his subsequent career in translational science and patient-centered therapeutic development. He went on to found Perlara PBC, the world’s first biotech public benefit corporation and Y Combinator alum (W16) and later served as Chief Scientific Officer of the Christopher & Dana Reeve Foundation. As an investor in ~100 companies (top performer: Gilgamesh Pharma), Ethan brings translational science, patient-centered development, and builder expertise.

Harvard Ph.D.Princeton postdocYC W16

Co-founder & COO

Ryan Ponzi, Ph.D. on LinkedIn (opens in a new tab)

Ryan received his B.S. in Chemistry from the University of New England and Ph.D. in Organic Chemistry from Boston College (under Professor Amir H. Hoveyda). He transitioned to intellectual property law, becoming a USPTO-registered Patent Agent at a Vault Top 5 firm. Ryan supported high-value rare disease portfolios generating billions in annual revenue and evaluated assets for multibillion-dollar acquisitions. Ryan made a deliberate transition from bench science to intellectual property law, motivated by a conviction that the most impactful contributions to drug development happened not only in the laboratory, but in the legal and business decisions that determine which science reaches patients.

Boston College Ph.D.Patent AgentIP strategy

Join the mission

Help us reach the next family.

Alone, each child is rare. Together, they’re unstoppable.

I’m a parent-founder I’m an investor or partner