Overcoming anti-AAV pre-existing immunity to achieve safe and efficient gene transfer in clinical settings
Highlights From the ESGCT 28th Annual Congress

Overcoming Anti-AAV Pre-existing Immunity to Achieve Safe and Efficient Gene Transfer in Clinical Settings.

Giuseppe Ronzitti, PhD
Genethon, UMR_S951, Inserm, Univ Evry, Université Paris Saclay, EPHE

Key Data Points

Overcoming anti-AAV pre-existing immunity to achieve safe and efficient gene transfer in clinical settings

(A) The efficacy of transgene expression using AAV vectors can be reduced due to innate immunity. (B) In this proof of concept study involving non-human primates with anti-AAV antibodies, the endopeptidated imlifidase (IdeS) was used to determine if degradation of circulating IgG could improve AAV-mediated expression of hFIX.

Improved Efficacy of Gene Therapy in Seropositive Non-human Primate with IdeS

In non-human primates treated with FIX-containing AAV8, hFIX expression in plasma was significantly higher in an animal treated with IdeS compared with one without IdeS treatment. These results suggest that systemic IgG degradation may permit gene therapy with AAV vectors in sero-positive patients with hemophilia as well as readministration in previously treated patients with decreasing factor expression.

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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