Beyond One Foundation — Where existing medicine meets new hope

The medicine may already exist.We're building the fastest way to find it.

Beyond One Foundation uses AI and computational biology to screen thousands of FDA-approved drugs for the ones that can restore what a single broken gene took away. We start with STXBP1. We're built to go beyond it.

Fund the research Watch our story
2027 Goal: $700,000
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Nearly every dollar goes directly to research. Our transparency pledgeWe disclose every dollar spent and where it goes — updated every quarter and filed annually. All reports will be freely available right here on this page.

Our story

Two parents.
One diagnosis.
A different kind of foundation.

Jon and Chris share what it was like to hear the words "STXBP1," what they learned about how rare disease research actually moves, and why they decided to build something that could move faster.

Jon Watkins & Chris Pirozzi, PhD on Arco and the start of Beyond One

More about Jon and Chris: Chris has spent 16 years in brain tumor research at Duke's Preston Robert Tisch Brain Tumor Center, where his work focuses on the genetics of cancer. Jon owns The Teaching Studio, a broadcast-grade production company that produces live programs for Duke, UNC, and Fortune 500 clients. Together they bring the science and the storytelling that a foundation like this needs — and a very personal reason to move fast.

Our mission

Why "Beyond One"

Rare disease families are told to wait for a cure that is a decade and a billion dollars away. We don't think they should have to. Thousands of medicines already sit on pharmacy shelves with full safety data. Our job is to find the ones that were meant for more.

>1

Greater than one person

This started with Jon and Chris's son, Arco. But it will not end with him, and it will not end with his gene. This foundation is for every family facing rare disease.

>1

Greater than one disease

STXBP1 is the first disease in our pipeline, not the only one. We are building a repeatable workflow so the next rare genetic disease can plug in without starting from zero.

>1

Greater than one purpose

A drug approved for one condition can carry a second life. We look for existing medicines with a hidden ability to restore what a faulty gene took away.

Research
Firstour pledge to donors

We run lean by design, so nearly every dollar you give goes directly to the science: the computational biologist, the AI infrastructure, the advisory board's review, and the laboratory studies that turn a prediction into evidence.

And because trust should be earned, we keep open books: we disclose every dollar spent and where it went, updated every quarter and filed annually.

The problem we're built for

Rare disease is not rare.
Treatment is.

Most rare diseases are genetic, most have no approved treatment, and the traditional path to one is measured in decades. That math doesn't work for a child who is growing up right now.

0+
rare diseases identified worldwide
0%
of rare diseases have a genetic origin
0%
have no FDA-approved treatment
0M
people living with a rare disease globally

Developing a new drug

The traditional path
Time to patients10–15 years
Typical cost$1–2+ billion
  • Safety must be proven from scratch
  • Roughly 9 in 10 candidates fail in clinical trials
  • Rarely pursued for diseases affecting a few thousand people

Repurposing an approved drug

The Beyond One path
Time to patients3–6 years
Typical costA fraction of new-drug development
  • Human safety and dosing already established
  • Can move to trials on existing manufacturing and supply
  • Finally viable for small patient populations
Disease one

STXBP1

STXBP1 is a gene that builds a protein every neuron needs to release its chemical messengers. When one copy of the gene is broken, the brain gets roughly half the protein it needs. The result is a severe, lifelong neurological disorder, most often first noticed as seizures in infancy.

1 in
30,000

Estimated to affect about one in every 30,000 births, making it one of the more common causes of genetic epilepsy and developmental disorder.

2008

First linked to epileptic encephalopathy. Diagnosis has grown rapidly since as genetic testing became routine.

De novo

The vast majority of cases are not inherited. The mutation appears spontaneously, which means it can happen to any family.

0

Approved treatments that address the underlying cause. Today's medicines manage symptoms such as seizures. None restore the missing protein.

Why "turn up the good copy" works here

STXBP1 disorder is a haploinsufficiency: one gene copy is faulty, but the other is perfectly healthy. Instead of replacing the broken gene, we look for approved drugs that increase output from the working one. Raise the protein level enough and the biology has a chance to recover.

Healthy copy
Faulty copy
↓ an approved drug that upregulates the healthy copy ↓
Restored level

What families live with

STXBP1 disorder looks different in every child, but most families face some combination of:

  • SeizuresOften beginning in the first months of life and frequently hard to control with medication
  • Developmental delayNearly all children have intellectual disability, ranging from moderate to profound
  • Movement and toneTremor, unsteady movement, and low muscle tone are common
  • Speech and communicationMany children are non-verbal or have very limited speech

STXBP1 is where we start,
not where we stop.

Hundreds of rare genetic diseases share the same underlying problem: one healthy gene copy, one faulty one, not enough protein. The AI model, the review process, and the testing partnerships we build for STXBP1 are designed to be reused. When the workflow is proven, the next disease enters the pipeline without starting over.

STXBP1 — active pipeline
Next monogenic disease — workflow ready
Next monogenic disease — workflow ready
Nominate a disease → contact us
What makes us different

AI does the searching. Scientists do the deciding.

A human researcher can study a handful of drugs a year. A disease-specific AI model can evaluate thousands of approved compounds against the biology of STXBP1 in days, and rank the ones most likely to raise expression of the healthy gene. Then a panel of scientists takes over.

  • A model built for one disease at a time

    Our computational biologist trains an AI model on the specific gene, pathway, and cell biology of the disease, not a generic drug database.

  • Only medicines that are already approved

    Every candidate has cleared FDA safety review for some other use. That's what lets a promising result move toward patients in years instead of decades.

  • A scientific advisory board holds the gate

    No prediction becomes an experiment until five scientists have reviewed the evidence and recommended it. AI proposes; people approve.

  • Everything we learn is published

    Positive or negative, every result is released to the research community quarterly, so the next lab and the next family benefit.

The full workflow

From prediction to proof, in four stages

Each stage has a clear owner and a clear output.
Nothing moves forward without the stage before it.

1
Senior Scientist, Computational Biology & AI

Identify candidates

A full-time PhD computational biologist trains a disease-specific AI model, then uses it to analyze how thousands of approved drugs might upregulate the healthy gene copy.

Output: a ranked shortlist of drug candidates with supporting evidence
2
Scientific Advisory Board

Clear candidates

A five-member panel led by Chris Pirozzi, PhD, works alongside the scientist to challenge the model, refine the tests, and recommend which candidates advance to real-world testing.

Output: candidates recommended for laboratory testing
3
Foundation Board + testing partners

Fund and test

The Foundation Board approves funding. Studies run through university collaborations and leading independent research organizations. The advisory board then assesses each round of results and recommends the next stage of testing where warranted.

Output: real-world evidence on safety and effect
4
The scientific community

Share everything

All data and research are released to the scientific community and published quarterly. Findings feed back into the model, and the workflow is ready for the next disease.

Output: open data, faster science, a reusable pipeline

Two independent checks before any dollar is spent on testing

The Scientific Advisory Board decides what is worth testing. The Foundation Board decides what gets funded. Neither can skip the other, which keeps the science honest and your donation accountable.

AI model→ Advisory board→ Foundation board→ Laboratory studies→ Clinical evaluation
The people

Built by parents, scientists, and a board that believes there's hope in today's medicine.

Now hiring

Senior Scientist, Computational Biology & AI Drug Repurposing

Full-time · The first hire your donation funds

This scientist is the engine of the foundation. They will train the STXBP1 disease model, run the drug screening, work directly with the Scientific Advisory Board to design and refine AI tests, and prepare each candidate's evidence package for review. When STXBP1 is proven, they lead the onboarding of the next disease.

We are recruiting a PhD computational biologist with deep machine-learning expertise and a track record in drug repurposing. If that's you, or someone you know, we'd like to talk.

PhD, computational biologyAI / machine learningDrug repurposingRare disease

Scientific Advisory Board

Five scientists who review every candidate and decide what is worth testing. Chaired by Chris Pirozzi, PhD.

Chris Pirozzi, PhDChair · Researcher, Preston Robert Tisch Brain Tumor Center at Duke · 16 years in brain tumor research
To be announcedAdvisory board member
To be announcedAdvisory board member
To be announcedAdvisory board member
To be announcedAdvisory board member

Foundation Board

Approves funding for testing, safeguards our research-first pledge, and answers to donors.

Jon WatkinsCo-Chair · Co-founder · Owner, The Teaching Studio · Arco's dad
Chris Pirozzi, PhDCo-Chair · Co-founder · Duke researcher · Arco's dad
To be announcedBoard member
To be announcedBoard member
To be announcedBoard member
To be announcedBoard member

Testing partners: more details to come

We are in conversations with academic labs and independent research organizations for every stage of testing, from first laboratory studies through clinical trials. Partners will be announced here as agreements are finalized. If your lab or organization runs studies like these and wants to help, reach out.

DiscoveryLaboratory studiesClinical trials
Open access

Our data belongs to everyone

Rare disease research is slowed by silos. Labs repeat each other's work because negative results are never shared. We will publish all of our data and research to the scientific community every quarter: the model's predictions, the board's reasoning, and the testing results, whether they succeed or fail.

Q1STXBP1 disease model: methods and first screening resultsUpcoming
Q2Advisory board candidate reviews and recommendationsUpcoming
Q3Study protocols and interim testing dataUpcoming
Q4Annual results and workflow documentation for the next diseaseUpcoming
Get in touch

Help us go beyond one

Whether you're a family living with STXBP1, a scientist who wants to sit on the board, a lab that can run a study, or someone who simply wants to fund the work, we want to hear from you.

FamiliesTell us about your diagnosis and what you'd like to see studied next.
Scientists & labsJoin the advisory board, apply for the scientist role, or partner on testing.
Donors & partnersFund a stage of the pipeline or sponsor the next disease.

Ready to give?

Research comes first in everything we fund. Online giving is launching soon — until then, send us a note and we'll follow up personally within one business day.

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