Systems and methods for improving safety of split intein aav mediated gene therapy

EP4762183A1Pending Publication Date: 2026-06-24SEATTLE CHILDRENS HOSPITAL (DBA SEATTLE CHILDRENS RES INST)
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Patent Information

Authority / Receiving Office
EP · EP
Patent Type
Applications
Current Assignee / Owner
SEATTLE CHILDRENS HOSPITAL (DBA SEATTLE CHILDRENS RES INST)
Filing Date
2024-08-16
Publication Date
2026-06-24

AI Technical Summary

Technical Problem

Current AAV intein vectors for gene therapy face challenges due to immune or toxic responses and regulatory concerns, particularly when encoding nonmammalian components, which limits their safety and clinical translation.

Method used

The system employs split intein-mediated protein trans-splicing to reconstitute large proteins, using AAV vectors to deliver N- and C-fragments of split inteins along with the sodium channel alpha subunit, and incorporates a degradation signal (degron) to remove byproducts, enhancing safety and reducing immunogenicity.

Benefits of technology

This approach effectively improves the safety of split intein-mediated gene therapy by minimizing immune responses and regulatory concerns, enabling the safe delivery of coding sequences for sodium channel proteins to treat conditions like Dravet Syndrome.

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Abstract

Systems and methods are provided for split intein-mediated AAV mediated delivery and expression of large protein molecules with improved safety features. Specifically degron sequences as degradation signals are included at specific terminus of N- or C- split intein fragments that are each coupled to a portion of the coding sequence of voltage-gated sodium channel alpha subunit (e.g., alpha subunit 1, Nav1.1), and upon expression and trans-splicing of split intein fragments, Nav1.1 is reconstituted whereas trans-spliced intein is digested along with the coupled degron via degron degradation pathways.
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