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PM577a · 1 trial · 3 indications
| Arm | Type | Description |
|---|---|---|
| PM577a | EXPERIMENTAL | PM577a is a sterile suspension of lipid nanoparticle (LNP)-formulated Prime Editors (PEs) intended for single dose intravenous (IV) infusion for the treatment of Wilson disease (WD). |
| Name | Type | Description |
|---|---|---|
| PM577a | DRUG | PM577a is being evaluated in participants with Wilson disease caused by biallelic pathogenic, likely pathogenic, or suspected pathogenic ATP7B variants, including at least one p.H1069Q allele. |
Inclusion Criteria: * Confirmed Wilson Disease (WD) diagnosis as determined by medical history consistent with WD * Historical genetic analysis demonstrating biallelic pathogenic, likely pathogenic, or suspected pathogenic ATP7B variants, including at least one p.H1069Q allele. * Treated and stable...
PM577a is an investigational small molecule developed by Prime Medicine, Inc. for Wilson Disease, a rare inherited disorder of copper metabolism. It is being studied in a Phase 1 clinical trial in participants aged 12 years and older in the United States and New Zealand. It has received a Rare Pediatric Disease designation from the FDA.
PM577a is being developed as a treatment for Wilson Disease, a rare genetic condition that causes copper to accumulate in the body. The Phase 1 study is evaluating its safety and efficacy in participants with Wilson Disease. It is investigational and has not been approved for any indication.
PM577a targets ATP7B, the copper-transporting ATPase that is defective in Wilson Disease. By acting on this molecular target, the small molecule is intended to address the underlying copper metabolism defect. The target is central to the disease biology, as ATP7B mutations cause impaired copper excretion.
PM577a is developed by Prime Medicine, Inc., a biotechnology company traded under the ticker PRME. Prime Medicine is the sponsor of the ongoing Phase 1 trial of PM577a in Wilson Disease. The company focuses on gene editing and related therapeutic approaches.
PM577a is in Phase 1 clinical development. The single ongoing trial is a Phase 1 study that is currently recruiting participants. It is an investigational therapy and has not been approved by the FDA for Wilson Disease or any other condition.
PM577a is being studied in one clinical trial, NCT07748403, titled "A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease (WD)." This Phase 1 trial is recruiting 42 participants aged 12 years and older in the United States and New Zealand. It is an uncontrolled, non-randomized study.