Gene therapy for the treatment of Wilson's disease

ES3072955T3Undetermined Publication Date: 2026-07-07THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA (100 00)
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Patent Information

Authority / Receiving Office
ES · ES
Patent Type
Patents
Current Assignee / Owner
THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA (100 00)
Filing Date
2017-12-29
Publication Date
2026-07-07

AI Technical Summary

Technical Problem

Current treatments for Wilson's disease, such as chelating agents and liver transplantation, require lifelong medication and can cause side effects, and do not restore normal copper metabolism, while liver transplantation necessitates immune suppression.

Method used

A replication-deficient adeno-associated virus (AAV) vector is used to deliver a codon-optimized human copper-transporting ATPase 2 (ATP7B) gene to liver cells, utilizing liver-specific expression control elements for long-term correction of Wilson's disease, reducing circulating copper levels by about 25% or more.

Benefits of technology

The AAV-mediated gene therapy effectively lowers circulating copper levels and prevents liver disease progression, offering a potentially lifelong correction of Wilson's disease without the need for continuous medication and immune suppression.

✦ Generated by Eureka AI based on patent content.

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Abstract

Compositions and regimens useful for the treatment of Wilson's disease are provided. These compositions include a recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and a promoter that directs human ATP7B expression.
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