The maturing of Cell and Gene Therapy products provides an opportunity to serve patients with options for treatment where none have previously existed. In the past few years, several cell and gene therapy products have gained regulatory approval in the US and EU. The number of products in clinical development in the US continues to increase and many of these products have the potential for accelerated regulatory pathways. Manufacturers of cell and gene therapy products must tackle technological challenges under the pressure of short timelines resulting from streamlined clinical development. This has been further compounded by demands for capacity, human resources, and manufacturing materials resulting from the global COVID pandemic. This session will focus on innovations that focus on the implementation platform processes and technology that will enable rapid product development and in turn aid in Regulatory review and approval.

The goal of platform-based process technology is to bring products to market sooner and to ease the Regulatory burden for sponsors and Regulators.

• What aspects of the platform process approach do you see as the first to gain Regulatory acceptance?
• Where do you see the biggest challenges for the Regulatory relief?
• What technology breakthroughs are on the horizon to overcome the challenges?

The public/private partnership described by Steven Hoffman for the AAV platform is a great opportunity, but even taking the existence of DMF in the US into account (which is not an option in the EU) is this approach a valid one within the current regulatory framework? Does a framework need to be developed.

• To what extent will Regulators accept inter-company platform knowledge, given that it will need to be implemented locally?
• Is this the same as an MF?

What do you see as the main challenges for automated platform manufacturing of cell-based products? (starting with someone other than Qasim)

What is the interplay between platform technologies and GMP?

• Will it make compliance easier, or more difficult? For cell-based products there is a widespread lack of understanding what is required of closed systems.
• Should the approach be tightened based on increased knowledge and scale-up possibilities? (Currently, the EU GMP for ATMPs is seen by many as less strict than standard GMP)

Rob McCombie

Orca Bio

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