Meet the Inaugural Gene Therapy Candidates
The two gene therapy clinical trials for Wilson disease (WD) launched by Ultragenyx and Vivet Therapeutics are underway! Here, we introduce the WD community to two people who are trailblazers in these studies that will show us if gene therapy works as a treatment for the rare genetic liver disease. Emily signed up for the Ultragenyx trial, and Warren volunteered for the Vivet trial.
They are both in their 40’s. Both are married with a young child, and they both live east of the Mississippi and north of the Mason-Dixon Line. They’re both Caucasian, brunette and wear glasses. They’re the only ones in their respective families with WD. And both were diagnosed from the copper deposits found in their corneas, called Kayser-Fleischer rings.
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Why They Said ‘Yes’ to Gene Therapy
We continue the story of two trailblazers who are among the first Wilson disease (WD) patients to volunteer for one of the two gene therapy clinical trials now underway for our rare genetic liver disorder. Emily signed up for the Ultragenyx trial and Warren volunteered for the Vivet trial. In our Winter 2023 newsletter we introduced them to the WD community. Emily received her diagnosis five years before on her 35th birthday, while Warren was diagnosed in 1980 at age seven. Emily is a wife and mom to a four-year-old son and works as a school secretary. Warren is a husband and dad to an 11-year-old daughter and had to give up his job as a preacher due to WD complications.
The story of their patient experience left off with Emily and Warren facing the decision of whether or not to participate in a trial designed to determine if gene therapy in WD patients is safe, and works.
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Gene Therapy
Gene therapy is a technique designed to treat or prevent disease. When a gene mutation causes a protein to be missing or faulty, gene therapy may be able to restore the normal function of that protein. In the case of Wilson disease, patients have a faulty copy of the ATP7B gene, which is responsible for transporting excess copper out of the liver.
To learn more about how gene therapy might help people with Wilson disease, view this video produced by The American Society of Gene & Cell Therapy (ASGCT):
Two gene therapy studies are underway for patients with Wilson disease. While both studies are designed to deliver a one-time infusion of a normal, working copy of the ATP7B gene into the liver, details for the clinical trials differ. Study locations, enrollment criteria and study design continue to evolve. For the most up-to-date information about the gene therapy studies being conducted by Ultragenyx and Vivet Therapeutics visit https://clinicaltrials.gov/ and enter “Wilson disease” in the search bar.
You can find additional information from each pharmaceutical company’s trial here:
The chief medical officers of Vivet Therapeutics and Ultragenyx provided these presentations at the WDA’s 2023 Virtual Conference:
