Adeno-associated virus gene therapy products and methods

JP2025524636A5Pending Publication Date: 2026-07-21RES INST AT NATIONWIDE CHILDRENS HOSPITAL
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Patent Information

Authority / Receiving Office
JP · JP
Patent Type
Applications
Current Assignee / Owner
RES INST AT NATIONWIDE CHILDRENS HOSPITAL
Filing Date
2023-07-11
Publication Date
2026-07-21

AI Technical Summary

Technical Problem

Current treatments for neurodegenerative disorders, such as ALS, fail to effectively address the neuroinflammatory components, despite targeting individual cell types like microglia, astrocytes, and neurons, leading to limited therapeutic benefits and disease progression.

Method used

A combination treatment approach using AAV gene therapy vectors that express therapeutic proteins or RNAs to correct gene deficiencies in neurons and astrocytes, while secreting anti-inflammatory proteins or peptides to modulate microglial activation, including the use of galectin-1 to maintain microglia in a non-inflammatory state.

Benefits of technology

Enhances therapeutic efficacy by reducing neuroinflammation and improving survival and motor function in ALS models, with potential benefits for a range of neurodegenerative disorders.

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Abstract

Adeno-associated virus (AAV) gene therapy vectors express therapeutic proteins or RNAs that treat gene deficiencies in cells. This disclosure provides AAV gene therapy vectors that further express anti-inflammatory proteins or peptides. When the provided AAV gene therapy vectors are used, for example, in methods of treating neurodegenerative diseases, the therapeutic protein / RNA treats the gene deficiency in cells directly transduced by the AAV vector, while the anti-inflammatory protein / peptide is secreted by the transduced cells into the intercellular environment to treat microglial activation associated with the neuroinflammatory component of the neurodegenerative disease. The therapeutic protein and the anti-inflammatory protein / peptide can be expressed as a fusion protein in which the two are separated by a self-cleaving peptide in the fusion protein. Accordingly, the provided AAV gene therapy vectors and methods are useful for treating neurological and neurodegenerative disorders.
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