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Treating more patients with gene therapies

A Genethon team has successfully inhibited immune response to adeno-associated virus vectors, and in so doing, opened potential paths for treating more patients with gene therapies.
Press release of Genethon >

In an article published 01 June 2020 in Nature Medicine, researchers from Genethon and their partners from CNRS/Inserm and the biotech Spark Therapeutics announced their use of the enzyme imlifidase (IdeS) to successfully suppress immune response against adeno-associated virus (AAV) vectors. The antibodies mediating that immune response may be naturally present or consecutive to gene therapy.

In both cases, the immune action they provoke against the vector carrying the gene therapy poses a risk to that treatment, notably rendering it ineffective. In their work, the multi-institution team used IdeS to cleave those antibodies, effectively suppressing the immune response against the AAV vector. The team’s results open new therapeutic perspectives and the possibility of increasing the number of people able to be treated by gene therapies.



IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies. Nature Medicine, 2020.

With the support from
Région île de France