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UX701 · 1 trial · 1 indication
| Arm | Type | Description |
|---|---|---|
| Stage 1: UX701 Dose Level 1 | EXPERIMENTAL | Participants receive a single, peripheral intravenous (IV) infusion of UX701 at dose level 1. |
| Stage 1: UX701 Dose Level 2 | EXPERIMENTAL | Participants receive a single, peripheral IV infusion of UX701 at dose level 2. |
| Stage 1: UX701 Dose Level 3 | EXPERIMENTAL | Participants receive a single, peripheral IV infusion of UX701 at dose level 3. |
| Stage 1: UX701 Dose Level 4 | EXPERIMENTAL | Participants receive a single, peripheral IV infusion of UX701 at dose level 4. |
| Stage 2: UX701 at Selected Dose | EXPERIMENTAL | Participants randomized to UX701 receive a single, peripheral IV infusion of UX701 at the selected dose. |
| Stage 2: Standard of Care (SOC) to UX701 | EXPERIMENTAL | Participants randomized to SOC will continue their baseline SOC medications for 52 weeks, followed by a single, peripheral IV infusion of UX701 at the selected dose. Following UX701 administration, participants will be evaluated for modification of their SOC medications. |
| Name | Type | Description |
|---|---|---|
| UX701 | GENETIC | Nonreplicating, recombinant gene transfer vector |
| Standard of Care (SOC) | DRUG | SOC treatment (i.e., copper chelators and/or zinc) administered according to standard regimens. |
Key Inclusion Criteria: * Confirmed diagnosis of Wilson disease based on genetic confirmation of heterozygous or homozygous biallelic ATP7B mutation. * Stable Wilson disease as evidenced by ongoing copper chelator (ie, penicillamine, trientine) and/or zinc therapy for at least 2 months at screening...
UX701 is an investigational gene therapy being developed for the treatment of Wilson Disease, a rare inherited disorder that causes copper to accumulate in the body. It is designed to address the underlying genetic cause of the condition. The therapy is currently in clinical development and has not been approved by regulatory authorities.
UX701 is a gene therapy intended to deliver a functional copy of the gene that is defective in Wilson Disease. By providing this gene, the therapy aims to restore the body's ability to properly process and eliminate copper, potentially addressing the root cause of the disease. This mechanism is based on the therapy's design as a gene replacement approach.
UX701 is being developed by Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on rare diseases. The company's stock is traded under the ticker symbol RARE. Ultragenyx is conducting clinical trials to evaluate the safety and efficacy of UX701 in patients with Wilson Disease.
UX701 is currently in Phase 1 of clinical development. The ongoing trial is a Phase 1/2/3 study, meaning it is designed to evaluate the therapy across multiple phases in a single trial. The study is active but not recruiting participants, and the therapy remains investigational.
UX701 is being studied in a Phase 1/2/3 clinical trial with the identifier NCT04884815. This trial is enrolling adults with Wilson Disease and is being conducted in the United States, Portugal, Spain, and the United Kingdom. The study has a planned enrollment of 82 participants and is currently active but not recruiting.
UX701 is a specific gene therapy developed by Ultragenyx for Wilson Disease. It is not known to be the same as other gene therapies for this condition. The therapy is being evaluated in its own clinical trial and has a distinct mechanism of delivering a functional gene to address the disease.