Interested in participating? Learn about which studies might be right for you.

Eligibility criteria may vary from country to country, based on a study site’s current approved version of the protocol. Please talk to your physician for more information.

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STUDY 1| Clinical Interventional

Cyprus2+™ Gene Therapy Phase 1/2/3 Study


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Are 18 years of age or older

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Have been diagnosed with Wilson Disease

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Have been on copper chelator and/or zinc therapy for at least 6 months prior to participating in this study, with no changes in dose or medication type.

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Limit your intake of high-copper-containing foods

Why are we doing this study?

An investigational gene therapy called UX701 is being tested for the treatment of Wilson Disease. “Investigational” means the study drug is not approved by the Health Authority and the safety and efficacy have not been established.

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Additional travel support may be available

Ultragenyx recognizes that your participation in clinical research has an impact on your (and your loved ones’) time. We may provide compensation and travel support for participation in our clinical trials—the details may vary program to program. You will learn more during the consent process.

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UX701, an investigational gene therapy

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Support is provided for travel costs to clinic visits

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Your participation can shape the future of Wilson Disease treatment

What you can expect

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One-time infusion of study medication into your arm

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Lab tests (urine, stool, blood, saliva)

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Medical exams, questionnaires, and interviews

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Heart tests

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Brain imaging

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Eye exams

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Liver tests

What is involved?

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~1 year icon
One intravenous infusion of UX701 followed by oral corticosteroids for ~2 months


Follow-up visits only
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11 visits over 4 years (6 can be home-health visits)

Additional Visit Information

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The screening period will consist of two screening visits, one of which will be completed after your eligibility is confirmed.
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Once screening is completed, there will be 2 clinic visits on consecutive days (Baseline and Dosing Day)
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After Dosing, 19 follow-up visits over 1 year (14 can be home-health visits)
Interested in participating or have questions? Get started

Understanding Gene Therapy

Gene therapy is the introduction, removal, or change of genetic material in the cells of a person. For certain rare genetic diseases, gene therapy offers the potential for treatment that could have a significant impact on the lives of patients. Find out more details, including how gene therapies are designed to work.

Dig Into the Science

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This study is approved by the Ethics Committee (EC), an established organization that protects the rights and welfare of patients participating in research activities.

Get Started

To learn about current and future studies, you only need to fill out the form once.
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Why are we asking this?

As with most clinical trials, certain criteria such as age must be met to determine who can participate.

The date of birth entered should be that of the person who would be enrolled in the trial.
If you are under the age of 18, a parent, care partner, or guardian must fill out this form for you.
Why are we asking this?

Letting us know the Patient's zip code can help us locate clinical trials that are closest to you or your loved one. Many clinical trials are held in hospitals and clinics and may require you to visit a facility in person.

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By submitting this form, you confirm that a diagnosis of Wilson Disease has been made by a medical professional.

If you would like to know more about your privacy and the data you submit and data we collect while you are on this website, please see below or visit our privacy policy.

What happens after you submit your information?

Our Patient Enrollment Liaison (PEL) team will contact you by email or phone within one business day to discuss your study eligibility and answer questions.

If you choose to be contacted by email, be sure to check your spam folder for an email from [email protected], if it’s not in your regular inbox.

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Got Questions?

Email our PEL team about current and future medical research opportunities.

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Patient portrayal.

Wilson Disease is a rare genetic disorder caused by an inability to remove excess copper from the body. Too much copper can lead to issues like liver disease and neurological complications.

If you are thinking about joining a study, our Patient Enrollment Liaison (PEL) team is here to help. Every member of our PEL team has a robust professional background and clinical expertise and experience to help answer your questions. Your PEL team member will assist in exploring Ultragenyx clinical and non-interventional clinical trials and related sites where you could be considered for enrollment. Your PEL is here to make the process as easy as possible.

To speak with a PEL about current and future medical research opportunities, please email our PEL team at [email protected].

When you voluntarily submit your personal data, Ultragenyx stores and processes this information in an electronic database consistent with our Privacy Policy. Ultragenyx will use this information to contact you to fulfill your request regarding Ultragenyx research opportunities.

Clinical and nonclinical studies provide invaluable information to treat, diagnose, or discover new breakthroughs in treating rare and ultra-rare diseases. By choosing to participate in research, you may help advance treatments, provide crucial insights, and ultimately improve the quality of life for others living with a rare or ultra-rare disease. You might also benefit from learning more about your condition, gain access to specific care, or achieve faster access to treatment. All efforts of participation, big and small, can make a difference.

Ultragenyx is leading the future of rare disease medicine. We leverage our experience, insight, and commitment to help move the rare disease community forward. Our primary goal is to provide medicines to those with limited options, and to help patients face rare disease head on with courage and confidence.

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