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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 therapy being developed for Wilson Disease, a rare genetic disorder of copper metabolism. It is designed to address the underlying genetic cause of the condition. The drug is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
PM577a targets the ATP7B gene, which is mutated in Wilson Disease. By targeting this gene, the therapy aims to correct the underlying genetic defect responsible for impaired copper transport in the body. This approach is intended to address the root cause of the disease rather than just managing symptoms.
PM577a is being developed by Prime Medicine, Inc., a biotechnology company publicly traded under the ticker symbol PRME. The company is conducting clinical research to evaluate the safety and efficacy of this investigational therapy for Wilson Disease.
PM577a is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still undergoing clinical trials to assess its safety and efficacy in patients with Wilson Disease.
PM577a is being studied in a Phase 1 clinical trial registered as NCT07748403. This open-label, uncontrolled study is evaluating the safety and efficacy of PM577 in participants with Wilson Disease. The trial is recruiting up to 42 participants aged 12 years and older in the United States and New Zealand.
PM577a is closely related to PM577, as both names appear in the context of the same clinical development program. The Phase 1 trial NCT07748403 is titled 'A Study of the Safety and Efficacy of Prime Editing (PM577) in Participants With Wilson Disease,' indicating that PM577a and PM577 refer to the same investigational therapy.