Compositions and methods for treatment of spinal muscular atrophy

EP4758163A1Pending Publication Date: 2026-06-17THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
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Patent Information

Authority / Receiving Office
EP · EP
Patent Type
Applications
Current Assignee / Owner
THE TRUSTEES OF THE UNIV OF PENNSYLVANIA
Filing Date
2024-08-10
Publication Date
2026-06-17

AI Technical Summary

Technical Problem

Current treatments for spinal muscular atrophy (SMA) are costly and associated with toxic side effects, and existing gene therapies, such as Zolgensma, have safety concerns that limit their use in less severe SMA types.

Method used

A recombinant adeno-associated virus (rAAV) vector is developed, containing an expression cassette with a UbC promoter, a coding sequence for a functional human survival of motor neuron 1 (SMN1) protein, at least four miR182 target sequences, and a polyA sequence, designed for intrathecal delivery to treat SMA.

Benefits of technology

The rAAV vector effectively increases SMN1 protein expression, potentially reducing symptoms of SMA while minimizing toxic side effects, and can be administered via intrathecal routes such as intracerebroventricular (ICV) or intracisternal magna (ICM) injection.

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Abstract

Expression cassettes and recombinant AAV vectors comprising the same for delivery of a nucleotide sequence encoding hSMN1 are provided. Compositions provided are useful in methods for the treatment of spinal muscular atrophy (SMA) in a subject in need thereof.
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