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Behind the Scenes at St. Jude

Here at Mirus Bio, we love to talk all things transfection. From identifying transfection solutions for a myriad of cell types and nucleic acids to troubleshooting complex experimental design with researchers developing gene and cell therapies, the team here at Mirus is comprised of passionate problem solvers.

Gaining an outside perspective about our transfection products keeps our team grounded and motivated to improve and innovate, especially when we hear about transfection challenges in the scientific community. In a recent podcast, Bryan Piras, Director of Manufacturing at St. Jude Children’s Research Hospital, describes such a transfection dilemma.

Piras’ team at St. Jude has been working on a gene therapy for Hemophilia B, a genetic disorder linked to the F9 gene which is located on the X chromosome. Production of the AAV8-based therapy was originally optimized in an adherent cell process, but to produce the quantities required for pre-clinical studies, the team scaled up from their adherent system to suspension cultures in stirred-tank bioreactors. Unexpectedly, when switching virus production platforms, the team discovered the AAV8 product had lost a significant amount of potency.

During the troubleshooting process, the team at St. Jude assessed many variables, including transfection reagent. Among other process changes, implementing the use of TransIT-VirusGEN® in their transfection process led to the required increase in titer and quality of virus required for treatment of a larger patient population.

Listen to the podcast to learn more about how the Process Development team at St. Jude worked through troubleshooting hurdles to deliver their Hemophilia B therapy.

Click Here to Listen!

Bryan’s story of encountering obstacles during the development of a large-scale manufacturing process is not uncommon. At Mirus Bio, a dedicated team of Transfection Experts are ready to support all of your transfection workflows, no matter how large or small. If you are in need of a transfection solution or support for your process, give us a call or send an email, and our Technical Support staff will connect with you.

Phone: 608.441.2852, ext. 2
Email: techsupport@mirusbio.com

Have a story about overcoming obstacles during process development? We’d love to hear it!

Explore Related Info & Links

  • Listen to the podcast ‘Overcoming Challenges with Viral Vector Optimization for Hemophilia Gene Therapy’ [15:38]
  • How is transfection critical to the success of a cell or gene therapy? Read more here.
  • Download the infographic ‘Large-scale Transfection for Virus Production’
  • Learn about transfection reagents for gene and cell therapy here

The TransMission
Feedback or questions? We’d love to hear from you. Email techsupport@mirusbio.com or call us at 888.530.0801.