Genethon gearing up for Wiskott-Aldrich gene therapy trial

Genethon, a French drug developer created and funded by the French  Association against Myopathies, has produced, controlled and released a  batch of lentiviral vectors derived from the human immunodeficiency  virus for a gene therapy trial in humans in a rare immune deficiency.

The gene and cell therapy unit at Genethon says it has now become the  first organization in Europe to have produced this type of vector in  accordance with Good Manufacturing Practice standards.

According to the firm, the release of this first batch of HIV-derived  vectors is a crucial step in the preparation of the clinical trial  authorization application to be submitted to the Afssaps (the French  drug safety authority) during the course of March. Following approval by  regulators, this batch will be used in a gene therapy trial for  Wiskott-Aldrich syndrome, a rare immune deficiency. Genethon will be the  sponsor of this trial, which is expected to be launched during the last  quarter of 2009.