Liver toxicity associated with dosing of gene therapy products is an area of priority when it comes to better understanding safety challenges for patients in trials.

Recent high profile cases including death and HCC have highlighted associated risks of delivering high doses of gene therapy products into patients and thus there is much work to be done in terms of better understanding the risks of hepatoxicity alongside potential mitigation strategies. Whilst most cases of hepatoxicity observed in blood disorder trials pose more of an efficacy issue, the risk of liver failure still exists and is of concern to both drug developers and patients.

This workshop will cover:

• Should observed liver toxicity in Gtx Blood Disorder trials merit stricter entry criteria for trials? If so what are the ramifications for BioPharma and patients
• How low can we go? How viable and effective are lower doses as a preventative measure against liver toxicity
• What long-term animal studies currently exist to map and examine liver toxicity to Gtx delivered to the liver?
• Is total vector genome dose per kg or factoring in some measure of body mass index for patients a preferable and effective methodology to use?
• Advances of modelling liver toxicity in non-human primates