Every child with TECPR2 deserves a cure.

Around the world, roughly forty children are living with TECPR2, also known as SPG49 or HSAN9 — a relentless, fatal childhood disease. We are racing to reach every one of them with a gene therapy cure, and to open the door for thousands of rare diseases behind it.

Fund the cure Meet the children

The cure exists. It works in the lab. Now it needs to reach the children.

~40

children worldwide known to be living with TECPR2

12,000+

rare diseases affecting 400 million people

95%

of rare diseases have no approved treatment

blueprint we're building for all of them

The children of TECPR2

This was never about one child. It's about all of them.

TECPR2 is so rare that most doctors will never see a single case. But behind that number are real children — in homes across the world — and families fighting the same fight, often completely alone.

A collage of children around the world living with TECPR2 — smiling, playing and being cared for at home and in hospital. — Every face here is a child living with TECPR2 — and a reason we refuse to slow down.

Founded by Dr. Michael Kaplan, David Ogman, Dr. Dov Tchernin and multiple families across the world with a single mission, to CURE this disease. It grew the moment we understood that a CURE could be possible for every child who carries this broken gene. Today our mission holds all of them: the children we've found, the ones still waiting for a diagnosis, and the families who deserve to hear the word hope for the first time.

Is your child affected?

If your family is facing TECPR2, you are not alone, and your child belongs here. Reach out to connect with the community, share your story, and be part of the trial ahead. Contact the foundation.

Why it's urgent

TECPR2 is a childhood disease that takes everything, quickly.

TECPR2 — also known as SPG49 (Spastic Paraplegia 49) or HSAN9 (Hereditary Sensory and Autonomic Neuropathy type 9) — is one of the rarest and most severe genetic diseases known. A single faulty gene sets off a progressive neurodegeneration that has been described as pediatric ALS: it attacks the whole body and worsens by the day.

Children face missed milestones, breathing and swallowing failure, recurrent pneumonias, surgeries, feeding tubes and ventilators. Until now, every family has been told the same three things: no treatment, no hope, no cure.

We refuse to accept that. The disease moves fast — which is exactly why the science can't wait.

The science

We didn't wait for a cure. We built one.

Working with the world's leading gene-therapy scientists, we have developed a breakthrough, first-of-its-kind AAV9 gene therapy: a harmless virus that delivers a healthy copy of the TECPR2 gene into the body, correcting the disease at its source. It has already achieved full rescue in the brain in animal models.

Stained sections of brain tissue from preclinical models, showing rescue of affected cells following the gene therapy. — Brain tissue from our preclinical studies — the therapy rescues the very cells that TECPR2 destroys.

Discover the cause

Pinpoint the exact genetic error behind TECPR2 and design a therapy to correct it.

Engineer the therapy

Build an AAV9 gene therapy that carries a healthy copy of the gene to where it's needed.

Prove it works

Demonstrate the cure across multiple animal models — including full rescue in the brain.

Manufacture for the trial

Produce a clinical-grade batch large enough for a human trial. This is the costly final push — and where your support goes directly to work.

We are here

Clinical trial & FDA approval

Bring the therapy safely to the children who need it, and secure the approval that makes it real.

Built with the best in the world

An unprecedented alliance for one gene.

To do the impossible, we broke the traditional model — assembling clinicians, geneticists, academia and biotech into a single team focused on curing these children.

Our partners: Boston Children's Hospital, Harvard Medical School, UT Southwestern Medical Center, Massachusetts General Hospital, University of Cambridge, Weizmann Institute of Science, Universität Leipzig, University of Florida, Scripps Research, Jem Therapeutics, Immuneering, Johnson & Johnson, and Forge Biologics.

Beyond TECPR2

Curing one disease is teaching us how to cure thousands.

Of the 12,000+ known rare diseases, 95% have no cure — and most strike young children. In solving TECPR2, we've created a network and a blueprint that can be reused, disease by disease, for the children who come next.

A repeatable model

The partnerships and process that cured TECPR2 become a path any small rare-disease community can follow.

The bespoke-medicine era

Gene therapy lets us correct disease at its root — treatment designed for the child, not the market.

Wider breakthroughs

The same discoveries point toward lysosomal storage disorders and neurodegeneration — including ALS, Alzheimer's and Parkinson's.

Ways to give

The pieces are in place. The last push is funding.

Every gift goes toward manufacturing the therapy and getting it into a clinical trial — the difference between a cure that exists and a cure that reaches these children.

Make a donation

Give by card, bank transfer or PayPal. Every dollar is 100% tax-deductible and goes straight to the cure.

Donate now

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Start a fundraiser

Turn a birthday, a run, or your community into momentum for the cure. We'll help you set it up.

Get started

Our signature evening

One Rare Night

An elegant evening for pediatric rare disease — live music, dinner, and silent & live auctions, with every dollar raised driving the cure forward. Come as a guest, reserve a table, or sponsor the night.

Visit One Rare Night