Gene therapy trials working group report

In 2019, given the growing number of gene therapy clinical trials at GOSH/ICH, we approached all the PIs involved in ongoing and upcoming clinical trials as well as PIs with relevant expertise to create a joint UCL/GOSH working group. The group met to discuss ongoing safety, emerging toxicity of AAV gene therapy trials, pathogenesis of adverse events, monitoring options and therapeutic options to manage these adverse events. The following documents are a record of the discussion and thoughts.

GOSH BRC AAV SOP v1 (717.59 KB)

NM GT follow up_v1 (269.71 KB)