Pre-Conference Workshop Day

Monday, March 29 | 10.00 AM - 3.00 PM

Workshop A - Hepatoxicity in the Context of Gene Therapies Developed for Blood Disorders

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

David Wilcox

David Wilcox
VP Research & Translational Medicine
Medical College of Wisconsin

Workshop B - Exploring Advances in Preconditioning for Ex Vivo Therapeutics

Moving beyond oncology applications, discussing the latest preclinical research to optimize the patient conditioning process for ex vivo gene therapies.

Conditioning is a critical step in ex vivo gene therapy approaches and is essential for unlocking the therapies potential. In autologous gene therapy the patient is taken through processes of mobilization followed by separation, transduction and harvesting before the patient is actually conditioned and the gene therapy administered.

In order for the harvested cells to find their way to the bone marrow, the marrow needs to be treated with a conditioning therapy, which essentially makes space for these cells to allow them to en-graft in high numbers. The process of conditioning itself can be intense for patients and thwart with side effects however.

This workshop will cover:

• Hear the latest advances in preclinical conditioning regimes using antibody-based techniques
• Discuss the suitability of the technique to sickle cell disease and beta-thalassemia
• Understand the risks this technique would mitigate for future gene therapy patients

Wendy Pang Headshot

Wendy Pang
VP Research & Translational Medicine
Jasper Therapeutics

Gabriella Denning

Gabriela Denning
VP Research & Development
Expression Therapeutics