Show Notes:We began the interview by discussing the tremendous promise that gene therapies have for changing the healthcare paradigm, and also the challenges in delivering on that promise. I asked Ratish if he could explain what he thinks the biggest challenges in gene therapy production are. He explained that he believes companies are already delivering on the promise of the technology and this can be seen in the steady increase in approved gene therapy therapeutics. That said, challenges remain in manufacturing and he categorized the challenges into three primary areas.
- Unique – Unlike monoclonal antibody production, viral vector processes have no recognized templates for process development, manufacturing capabilities or capacities. In addition, there are multiple AAV serotypes which have to be considered in process development and different dosing requirements based on the target indication. Lastly, there is an overall lack of established experience in viral vector production.
- Urgent – This addresses supply and demand issues. Because these therapeutics are intended to cure rare diseases, the timelines for process development and manufacturing are compressed. The race to market encompasses not only process and assay development, but also technology transfer and manufacturing time.
- Uncertainty – Due to the lack of historical data and shorter history compared to monoclonal antibody counterparts, there is a focus on the evolving regulatory situation. Guidances are being drafted to accommodate these novel therapies, while still keeping patient safety in mind.