The High-Level Analytics Workstream has been running since September 2018, with representatives from 21 member companies.

The workstream is now progressing several deliverables:

• Using initial survey data to elucidate the different approaches being taken to measure potency of cell and gene therapies (CGTs), the Potency sub-team, are actively working on a publication that promotes and discusses the adoption and acceptance of a matrix approach within the industry to address challenges for potency development for CGTs. There are several definitions applied to the matrix approach currently, and a variety of viewpoints about the scope and mode of application. This paper will seek to establish alignment across the industry and provide high level guidance on how to utilize a matrix approach.

• Identifying the common current methods, understanding common method challenges and collaborating and aligning Industry around opportunities for method improvements is the goal of the Approaches to Improve Methods sub-team. The team formed at the start of Q1 2020 and is currently working to create an initial benchmarking survey focusing on methods to measure viral vector titer and empty / full capsid ratio (AAV) that will be distributed to members of High-Level Analytics in Q2 2020. Responses to this survey will support the team to elaborate content for their stimulus / opinion article on approaches to analytical testing for measuring CQAs of viral vector titer & empty/ full capsids.

• The Phase Appropriate Approach to Validation sub-team was formed at the start of Q1 2020 and is comprised of SMEs from High- Level Analytics, Regulatory Strategy and Validation Workstreams. The team’s goal is to co-develop a best practise guidance article that will address areas of particular challenge arising from current lack of Industry-alignment on approach to planning what is a phase appropriate validation strategy for CGTs for early clinical phases. The team have agreed that a survey could be a good place to start and in the short-term will focus on establishing relevant survey questions designed to improve understanding of current Industry approach / limitations of phase appropriate validation as a precursor to developing a best practise guidance article.