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):
One gene therapy study is underway for patients with Wilson disease. The study, being conducted by Ultragenyx, is designed to deliver a one-time infusion of a normal, working copy of the ATP7B gene into the liver called UX701. Study locations, enrollment criteria and study design continue to evolve. For the most up-to-date information about the UX701 gene therapy study being conducted by Ultragenyx visit https://clinicaltrials.gov/ and enter “NCT04884815” in the search bar.
You can find additional information about the Ultragenyx trial here:
The chief medical officer of Ultragenyx provided this presentation at the WDA’s 2023 Virtual Conference:
Prime Medicine is studying gene editing as a possible treatment for Wilson disease and presented potentially promising data on its preclinical gene editing studies at scientific meetings in late 2024 and 2025. In the laboratory, or preclinically, scientists were able to correct the faulty ATP7B gene that causes WD with its gene editing technology.
To learn how prime editing works you can watch a video here.
Prime Medicine is conducting a research study called Prime-0211 for adults living with Wilson disease (WD). This is a pre-screening study, which means no treatment is being given and no investigational therapy is being tested. The purpose of this study is to collect information that may help guide future research in Wilson disease, including possible studies of gene-editing approaches. Details on how to participate can be found here and for the most up-to-date information visit https://clinicaltrials.gov/ under NCT07226622.
Vivet Therapeutics made the difficult decision in October 2024 to terminate its VTX-801 gene therapy trial after infusing four patients at two different dosing levels. Data gathered at that time from the two doses that were tested showed that while the gene therapy was safe, it did not show a significant enough effect. Bumping up to the next dosing level would require additional funding.
