How synbio must evolve to be successful
I’ve just got back to the office from a very interesting workshop looking to address the challenge of delivering rapid microbial testing in cell and gene products.
Cell and gene therapies – particularly advanced therapy medicinal products (ATMPs) – are big news right now. After two high-profile product launches late 2017 for CAR-T cell therapies (Kymriah™ from Novartis and Yescarta™ from Kite Pharma) there is growing excitement in their potential: and growing questions about how to provide them cost-effectively and safely at scale.
One critical requirement for these therapies is control of microbiological burden, ensuring the product is not contaminated with microorganisms. While this is a known problem for all therapeutics, it is thrown into sharp relief in an ATMP context. Existing techniques and methods take a long time to execute (days to weeks) and require a large amount of the product to be tested. When your ATMP has a shelf life of three days and a single autologous dose of tens of ml, existing methods struggle to cope.
The purpose of the workshop was to kick off a process to create a draft standard for application of rapid microbial test methods to cell and gene products. It was organised by the Standards Coordinating Body for Gene, Cell, and Regenerative Medicines and Cell-Based Drug Discovery (SCB) in conjunction with NIST, NIIMBL and BioFabUSA. There were about 70 attendees from numerous organisations working in cell and gene therapies. I was invited to give our perspective on developing rapid diagnostic technologies and managing complex manufacture processes containing biological materials.
Three key points hit home for me:
Clinical diagnostics has a lot to offer
Many of the user requirements in this application are similar to those in the clinical diagnostics community. There are good opportunities to build on what’s been learnt in usability, validation and data integration.
Integrating multiple technologies
There is no one technology that suits all and achieving the required speed will likely require integration of several methods throughout the manufacture process. Any standard needs to provide a framework where diverse technologies can be used as best suits the application and its risks. The focus needs to be on user requirements and robust validation approaches so developers can be confident how to demonstrate their technology is suitable and manufacturers can be confident in using them.
Learning over time
Our knowledge of microbiology is incomplete. We need to ensure standards expect an ongoing learning and improvement process (such as ISO13485 does) so we don’t hinder beneficial therapies unduly today but recognise we will learn more.
Many thanks to the organisers (Rod Rietze at Novartis, Spencer Hoover at CCRM and Richard McFarland at SCB) for organising a great workshop and giving us all plenty to think about. It was well worth another dose of jetlag for this attendee.