Posted on: 18th January 2019

The BioPhorum Cell & Gene Therapy (C&GT) Phorum has made significant progress since its creation in 2018 – from building networks and sharing knowledge to endorsing workstream plans and agreeing on the need for best practices – and will start implementing and publishing its work in 2019 and beyond.

However, the Phorum still has lots to accomplish. Do you want to be part of the conversation about shaping the future of the Phorum and its impact on the industry?

The recent November 2018 meeting in Houston, USA focused on High-Level Analytics and Regulatory Strategy, which are two of the Phorum’s six workstreams. It showed that high levels of collaboration between experts will be crucial to addressing industry challenges.

For many of the participants, this was their first face-to-face meeting and was a chance to make connections and forge new working relationships. It covered a lot of ground and fast-tracked the work of the groups much more than monthly telephone conferences.

The meeting was attended by 53 participants from 18 member companies. This level of attendance was very high for such a new Phorum and demonstrated its importance to the industry.

The primary goal of the meeting was to endorse the focus of the two workstreams. To enable this, a pre-meeting survey was conducted to highlight areas of interest and themes, and the teams prepared case studies and working sessions in advance. This drove a high level of active engagement from participants at the meeting and is a model that will be enhanced for future events.

The High-Level Analytics session included a site tour hosted by FujiFilm Diosynth Biotechnologies. There was much discussion around the implementation of Next Generation Sequencing and how to drive its pragmatic and consistent adoption within the CGT industry. The outputs of these discussions will be integrated into the workstream plans in the coming months.

Potency assays were also discussed and the team agreed that aligning methodologies, standardizing data reporting and approving general terminology (e.g. the standardization of units) should all be driven by the workstream.

The second day focused on the Regulatory Strategy workstream and included presentations from the National Institute of Standards and Technology (NIST) and the European Directorate for the Quality of Medicines (EDQM). The latter gave an overview of the European Pharmacopeia about cell and gene therapy, and the Official Medicines Control Laboratories’ Gene Therapy working group, which aims to establish standard analytical methods and reference standards for gene therapy products.

This was the first interaction of the Phorum team with NIST and EDQM and all agreed to continue and develop the relationship to share information and ensure approaches are aligned.

The theme of multiproduct facilities was addressed in separate break-out sessions where three scenarios were discussed in detail, with a consensus forming around risk-based approaches in all cases. The Regulatory Strategy workstream will expand on these approaches in future meetings, adding detail to the draft risk management plans and highlighting areas of interest for new members.

The remainder of the meeting focused on the guidelines for Chemistry, Manufacturing, and Control Information for Human Gene Therapy Investigational New Drug Applications released by the FDA’s Center for Biologics Evaluation and Research. This generated a lot of discussion as the manufacturing processes are new and there are many knowledge areas that need to be developed. The Phorum will play a crucial role in helping companies to understand what gaps exist and how to fill them. The Regulatory Strategy workstream will create a strategy to address the points raised.

The next meeting of the C&GT team in April 2019 will focus on Validation and Raw Materials (Sourcing, Quality and Volume), while Environmental Health and Safety and BioSafety, and Commercialization will be discussed in September 2019. For more information about the Cell & Gene Therapy Phorum and how to become a member, please contact Christelle@BioPhorum.com

 

 

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