A Cure for George: Help Advance CMT4C Gene Therapy to Clinical Trials

The Illingworth Family is raising money for Charcot-Marie-Tooth Association
Project Foresee is a CMTA sponsored project to advance the most promising CMT4C Gene Therapy cure through known steps to ultimately cure the first handful of patients in the next 3 years.

Story

In February 2025, we were devastated to learn our four year old George was born with a neurodegenerative disease that is gradually deteriorating his mobility, use of his hands, and potentially hearing, chewing, and breathing. We were told all we could do was attempt to slow the progression with bracing and physical therapy, but that most children with CMT4C lose their ability to walk independently in their teens.

Still in the shock of heartbreaking diagnosis, we learned that a geneticist in Cypress had already developed a gene therapy that would stop the progression of George's condition in its tracks. CMT4C is rare, and this felt like a miracle.

That six-year study successfully demonstrated the feasibility of an AAV-mediated gene therapy in animal models. Now, funding is needed to advance the gene therapy towards FDA approval and human clinical trials.

Project Foresee is accelerating this development.

Your contributions will fund drug manufacturing and formal preclinical toxicology studies, both critical components of the IND (Investigational New Drug) submission process with the FDA.

Every contribution brings us closer to stopping the progression of this cruel disease for George and the 8,000 other Americans with CMT4C, many of whom are young children. The sooner George has access to this drug, the more function will be preserved for the rest of his life.

Help The Illingworth Family

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About the campaign

Project Foresee is a CMTA sponsored project to advance the most promising CMT4C Gene Therapy cure through known steps to ultimately cure the first handful of patients in the next 3 years.

About the charity

The Charcot-Marie-Tooth Association (CMTA) is a community-led nonprofit focused on advancing treatments for CMT and improving patients' lives. As the leading global funder of CMT research, CMTA unites the community with clinicians and experts to accelerate treatments and, ultimately, find a cure.

Donation summary

Total
US$14,570.00
Online
US$14,570.00
Offline
US$0.00

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