Aav vectors for retinal and CNS gene therapy

LT3913061TActive Publication Date: 2026-07-27GENZYME CORP
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Patent Information

Application Number
LT21159585T
Authority / Receiving Office
LT · LT
Patent Type
Patents
Current Assignee / Owner
Priority Date
2015-02-10
Filing Date
2015-05-02
Publication Date
2026-07-27
Estimated Expiration
2035-05-02

AI Technical Summary

Technical Problem

Current methods for delivering adeno-associated virus (AAV) vectors to the eye and central nervous system (CNS) face challenges, particularly in achieving effective transduction and minimizing immune responses, which hinders the treatment of retinal degenerative diseases and neurologic disorders.

Method used

Development of recombinant AAV particles with specific amino acid substitutions in the capsid proteins that interact with heparan sulfate proteoglycan, reducing binding and enhancing transduction efficiency in retinal and CNS cells, using AAV serotypes like AAV2, AAVrh8R, and others, to improve gene therapy delivery.

Benefits of technology

The modified AAV particles demonstrate increased transduction efficiency and reduced immune response, leading to improved gene expression and treatment outcomes for ocular and CNS disorders, such as retinitis pigmentosa and neurologic conditions.

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Abstract

Provided herein are improved rAAV (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of ocular disorders or CNS disorders wherein the rAAV comprise one or more substitutions of amino acids that interact with heparan sulfate proteoglycan. The invention provides methods for improved transduction of retinal cells and methods for treating ocular diseases with improved compositions of rAAV particles. Further provided herein are improved recombinant adeno-associated virus (rAAV) (e.g., rAAV2, rAAVrh8R, etc.) for enhanced gene therapy of disorders of the CNS. The invention provides methods for delivering the rAAV to the CNS, methods for treating disorders of the CNS with improved compositions of rAAV particles, and kits for delivering the rAAV to the CNS and / or treating a CNS disorder.
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