P05

Let's standardize our AAV biodistribution procedures for CNS gene therapy

J Schock Vaiani(1) J Mittler(1) K Fransquin(1) R ALONSO(1) F PIGUET(1) S Jacquot(1)

1:GENOV - Paris Brain Institute, 47 boulevard de l’hôpital 75013 Paris

Here, we propose a standardized method for assessing the AAV biodistribution of after intravenous or intracerebral delivery, to easily compare several preclinical studies.

It would allow to compare same AAV serotype carrying several payloads or several AAVs carrying the same payload to assess efficiently biodistribution even with different routes of administration.

It is a crucial step to fill regulatory agencies requirements before these gene therapy products can be used in humans based on the consideration that any new construct must undergo a full evaluation, including a biodistribution study in several animal models.

Our new method will lead to robust preclinical data evaluation that would build a library of AAV biodistribution to which we can compare independently of the Study Sponsor.