Hope for Jojo

Our roadmap to a treatment for Jojo

For the first time, a therapy for Jojo and kids like her is within reach, thanks to several recent breakthroughs.

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Why now

The science is ready

A proven roadmap

A parent-led therapy in 2.5 years

Terry Pirovolakis showed what one parent can do: he assembled a research team, raised the funds, and developed a gene replacement therapy for a neurodevelopmental disease in just 2.5 years.

Nature Medicine, 2024

Gene silencing

ASOs are already FDA-approved

The FDA has approved several antisense oligonucleotides (ASOs) for neurological diseases, including one for a genetic form of ALS in 2023.

Gene editing

A personalized CRISPR treatment in 6 months

Researchers at CHOP, Penn, and the Innovative Genomics Institute treated the first patient with a personalized gene-editing treatment, in the liver, developed in just 6 months.

NEJM, 2025 · New York Times

Jojo's therapy is the next step: taking an approach that has worked in the liver and in mice to the next frontier, the human brain. This is more than one therapy. It's a proof of concept for an approach that can be adapted to treat an entire class of devastating diseases.

The plan

Six steps to treatment

Some steps run in parallel. Timelines and costs are estimates.

Part 1: Lab research

Steps 1 to 3
  1. 1

    Make the gene therapy

    We're partnering with the Innovative Genomics Institute (IGI), founded by Nobel laureate Jennifer Doudna, to lead this step. IGI has a pioneering track record: it helped develop the world's first personalized CRISPR treatment, for Baby KJ, who was born with a rare genetic disease.

    Scientists design gene therapy candidates and a guide RNA that acts as a GPS, steering the editor to the mutation in Jojo's SCN8A gene. Advanced sequencing checks for unwanted off-target effects.

    1 to 2 months $50,000
  2. 2

    Prove it works in human cells

    With the support of Stanford University, Jojo's blood cells are reprogrammed into stem cells (iPSCs) and grown into neurons. Scientists then test the gene therapy on these human neurons to see whether it corrects the SCN8A mutation.

    They also run an initial off-target analysis to make sure the editor isn't making unwanted changes to other genes before moving to animals.

    4 to 6 months, in parallel with the next steps $250,000
  3. 3

    Prove it works in an animal model

    A humanized mouse model is created with Jojo's SCN8A variant. Scientists package the gene therapy into a delivery vehicle that can travel to the brain, then give it to the mice to confirm it can be delivered, correct the mutation, and treat the disease.

    This step is being run by The Jackson Laboratory (JAX), a leading biomedical research institution known for its mouse models and more than 90 years of genetics research.

    9 to 12 months, in parallel with the prior steps $700,000

Part 2: Manufacturing and FDA review

Steps 4 to 6
  1. 4

    Manufacture the gene therapy

    To get through the bottlenecks of gene therapy manufacturing, we'll partner with a specialized contract development and manufacturing organization (CDMO). The therapy will be made in an FDA-compliant facility under Good Manufacturing Practice (GMP), so it meets FDA standards for purity, potency, and safety.

    1 to 3 months, depending on manufacturing slots $2M to $2.5M
  2. 5

    Complete safety testing

    Formal, large-scale safety (toxicology) studies are done to Good Laboratory Practice (GLP) standards. This step confirms the therapeutic window and checks that no dangerous toxicity appears in a living system before a child is treated.

    3 months $1.5M to $2M
  3. 6

    Get FDA clearance and treat Jojo

    Data from every earlier step is compiled into an Investigational New Drug (IND) application and submitted to the FDA. Once the FDA clears it, Jojo can be treated.

    About 1 month (the FDA's standard 30-day IND review) $500,000 to $1M

Fundraising progress

$1.44 million raised

Step 4a is funded. Next up: the full production run.

$1,441,687
raised of $5,000,000
29% of goal

Includes cash, grants, and committed in-kind partnerships. As of August 23, 2026.

  • Step 1: Make the gene therapyDesign gene therapy candidates and a guide RNA to target the SCN8A mutation. Secured: $50k in-kind (IGI).
    Funded
  • Step 2: Prove it works in human cellsTest the therapy on Jojo's cells to confirm it corrects the mutation safely. Secured: $35k in-kind (Stanford), plus donors like you.
    Funded
  • Step 3: Prove it works in animalsEngineer a mouse model with SCN8A to confirm the therapy reaches the brain. Secured: $120k (JAX, IGI, UK MRC), plus donors like you.
    Funded
  • Step 4a: Manufacturing prep and depositStart the manufacturing contract (process development).
    Funded
  • Step 4b: Full production run Large-scale production of the gene therapy for clinical use. Raising now: $500k to set up the first batch. $191,687 raised (38%).
    $1.75M goal
  • Step 5: Safety testingFinal toxicology and safety studies required before treatment.
    $1.5M goal
  • Step 6: FDA clearanceSubmit the IND application so Jojo can be treated.
    $500k goal

Your gift

What your gift does

$50

Funds one hour of a bioinformatician's time.

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$150

Buys lab supplies for making a guide RNA, the molecule that steers the editor to the SCN8A mutation.

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$500

Helps cover the cost of making the gene-editing protein.

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Medical research is a complex journey with inherent risks and no guaranteed outcomes. The costs and timelines listed are estimates based on current data and may change as we work through regulatory and manufacturing requirements. We're committed to transparency and will share regular updates on our progress.