Driving progress in Cell & Gene Therapy

Now with 19 member companies and 183 subject matter experts, the BioPhorum Cell & Gene Therapy (C&GT) Phorum has made great strides since its formation in 2018. The Phorum has a growing list of goals for the next 12–18 months and the team will use its combined strength to accomplish these.

The Phorum aims to support the faster delivery of C&GT products to market, by lowering business and supply chain risk, and accelerating improvements and the adoption of technical solutions. Do you want to be part of this drive and its impact on the industry?

At a meeting in April 2019 in Boston, US, the Phorum focused on two of the Phorum’s six workstreams: Raw Materials (Sourcing, Quality and Volume) and Validation. The meeting was a chance to reflect on the last six months and set a clear plan and direction for the future.

It was the first face-to-face meeting for many attendees, particularly those from companies new to the Phorum, and was a chance to build new relationships. It covered lots of subjects and helped accelerate the effort of the workstreams more than regular telephone calls.

The meeting was attended by 51 people over two days – a high level of attendance for such a new Phorum. The main goal was to focus the industry’s energies and mobilize the community to take advantage of new opportunities. Preparing sessions and case studies in advance helped keep engagement high and ensured knowledge and experience was shared.

The first day looked at the work of Raw Materials workstream, which discussed the key topics of Supplier Engagement, Material Performance and Risk Matrices.

The Supplier Engagement session generated a list of key suppliers with whom BioPhorum will engage, and a list of prioritized actions on how to improve supplier relationships. In particular, the attendees wanted to ensure a common understanding of the unique issues affecting the C&GT supply chain that differ from those of ‘standard’ biopharma manufacturing.

Material Performance looked at particulates, which is an area of increasing concern to the C&GT community. The emphasis was on actions to help understand the variety and extent of the source of particulates, and the risks they pose to the release of production batches.

Finally, the team reviewed its internal C&GT Risk Matrix, which covers the factors affecting the purchase and first-use of raw materials. It also discussed working with the Drug Substance Phorum, which has carried out work in raw material variance. While not focused on C&GT, its Risk Assessment Methodology document (awaiting publication), contains a lot of learning and practices that will be useful to the C&GT Phorum.

The second day concentrated on the Validation workstream and covered Contract Manufacturing Organization (CMO)/client interactions, C&GT-specific Validation Challenges, and Control Strategy and Process Validation.

The team reviewed the interfaces between CMOs and clients and documented the points where clear responsibilities and accountabilities are key. Clarifying and communicating these points will prove critical to building and maintaining successful relationships between biomanufacturers and CMOs.

Following the thought processes from the Raw Materials workstream sessions, the Validation team talked about the strategies and phased approach needed to cope with the limited variability of the raw materials available during development and manufacture. There are also unique considerations when using patient and donor cells, which may show both extreme variability and limited supply. Improved regulatory guidance and support for product validation will be driven by the stronger consortium voice of BioPhorum. Finally, the team discussed defining specifications using actual data, such as those for pre-clinical and process studies, and agreed on a broad approach to be taken.

Each day ended with the teams collating an action plan to help them achieve their goals.

The next meeting of the C&GT Phorum will take place in September 2019 and will focus on two other C&GT workstreams: Environmental Health & Safety/BioSafety, and Commercialization. The ‘working meeting’ format will be continued as the team looks to publish white papers and guidance notes, and collect survey data for these workstreams.

For more information about the Cell & Gene Therapy Phorum, and how to become a member, please contact [email protected]

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