Clinical trials & news

Current

Stanford trial underway

The first ever gene therapy trial for IPEX is currently underway at Stanford Medicine and actively recruiting!

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How we can help

  • Trip planning

    Experiences of past patients, travel and accommodation information, and more

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  • Travel support

    Discounted travel and accommodation for trial participants

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  • Financial support

    Direct support for trial-related costs and other financial hardship

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Recent articles and news

Jan 2026

'Hearts for Jax' brings IPEX cure one step closer

Thank you to the wonderful people of Zelienople, PA for your support as we raise $2M to fund the cure for IPEX

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JUL 2024

Hope for IPEX: Jax's Journey

Boy with IPEX treated with gene therapy at Stanford University

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JUL 2023

Innovator Spotlight: Dr. Bacchetta

Dr. Bacchetta of Stanford sits with The Scientist to discuss the future of gene therapy for IPEX

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FEB 2022

Stanford trial awarded $12M

The generous grant follows a previous $5.5M grant from CIRM to Dr. Bacchetta's team in 2019

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NOV 2019

Taylor speaks at CIRM

Young man shares his experience with IPEX, after Dr. Bacchetta's lab awarded $5.5M by CIRM to pursue trial

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SPRING 2019

A second chance for Ronnie & Levi

Young brothers are saved by a team with new approaches to a rare genetic disease

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2017

A rare diagnosis: Nick's Journey

STAT1 mutation discovery opens new treatment options for IPEX-like syndrome

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