Wilson discloses fatal animal liver, neuron toxicities with AAVs

Gene therapy pioneer James Wilson reported fatal liver and neuronal toxicities in animal studies of high-dose systemic adeno-associated virus gene therapy vectors. The Human Gene Therapy publication comes on the heels of the Jan. 16 disclosure in a SEC filing that Wilson had resigned from the scientific advisory board of Solid Biosciences Inc. (NASDAQ:SLDB) citing “emerging” concerns with high systemic doses of AAVs (see BioCentury Extra, Jan. 17).

The paper concluded that the risk of the toxicities could be "general properties of intravenous delivery of AAV vectors at high doses irrespective of the capsid serotype or transgene."...