Viral vectors for treating bladder diseases

The use of defective HSV-1 vectors to selectively silence bladder afferent nerves addresses the limitations of current treatments for OAB and IC by providing a targeted and stable gene expression, effectively reducing symptoms with minimal side effects.

WO2025003764A1PCT designated stage expired Publication Date: 2025-01-02EG 427
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Patent Information

Application Number
PCT/IB2024/000361
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2023-06-30
Filing Date
2024-06-28
Publication Date
2025-01-02

AI Technical Summary

Technical Problem

Current treatments for overactive bladder (OAB) and interstitial cystitis (IC) are largely ineffective and have significant side effects, with limited medical options available, particularly due to the nonspecific nature of existing pharmacological approaches that often result in systemic effects and complications such as urinary infections.

Method used

A viral expression vector that selectively modulates or silences the afferent nerves of the bladder using therapeutic transgenes, specifically targeting and inhibiting neurotransmission in bladder afferent neurons, thereby reducing urgency symptoms and pain sensation, achieved through the use of defective HSV-1 vectors that express botulinum toxins or other therapeutic proteins under afferent neuron-specific promoters for long-term expression.

Benefits of technology

This approach provides stable and selective gene expression in bladder afferent neurons, effectively reducing symptoms of OAB and IC without off-target denervation, preserving bladder function and sensation, and offering a more targeted and effective treatment compared to existing methods.

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Abstract

The present invention provides a method and a pharmaceutical composition for the treatment of overactive bladder (OAB) and / or bladder pain syndrome (BPS) comprising the viral expression vector carrying a transcription cassette that harbors transgene(s) inhibiting / silencing neurotransmission or synaptic transmission of afferent neurons.
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Citation Information

Patent Citations

  • Peripherally delivered glutamic acid decarboxylase gene therapy for spinal cord injury pain

    WO2006050211A2

  • Non-toxic HSV vectors for efficient gene delivery applications and complementing cells for their production

    WO2015009952A1

  • Viral vectors for treating neurogenic detrusor overactivity

    WO2017220800A1