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An industry first – aligning release standards for transfection reagents

1 December 2023
Advanced Therapy Medicinal Products
Supply Partner
Victoria Mwanza
Senior Global Change Facilitator

Cell and gene therapies (CGT) represents a novel and growing class of innovative products that often have complex manufacturing processes. In many cases, genetic material is introduced into eukaryotic cells via transient transfection, which is a crucial manufacturing step.

However, due to inherent complexity and criticality, there is an acute need for a standardized approach for certifications of analysis of chemical transfection reagents used in good manufacturing processes.

This is why we have published A proposal to align release standards for transfection reagents.

A quality-by-design approach was used to derive manufacturing process- and product-impactful raw material and/or ancillary material attributes. Although polyethyleneimine, a cationic polymer, is explored as an exemplar chemical transfection reagent, this approach also applies to other transfection reagents used in CGT manufacturing processes. The analysis and learnings discussed may be extrapolated to other transfection materials, providing a platform for discussion with suppliers and, ideally, enabling uniform and meaningful material attributes to be reported on these certifications of analysis.

“The standardization of release criteria for raw material, including transfection reagents, aims to accelerate the development timelines and reduce the overall costs of development for cell and gene therapy products,” said Nic Prayet, Associate Director, Gene Therapy CMC Development at UCB. “This is one step in the right direction to make these innovative therapies more sustainable and accessible.”

An agreed framework for release testing polymer-based transfection reagents has many benefits. It will give you confidence that your actions are aligned with those of your peers and alignment will bring reliability and consistency to the manufacturing process as everyone can meet the same standard for a particular material.

It will also mean that manufacturers, suppliers, and clients can use the same language and refer to an agreed reference table and testing. Importantly, when you file with regulatory authorities, you will have a data pack covering what they expect to see and demonstrating that you are managing and controlling that material appropriately.

There is a need to better define and harmonize the polymer-based transfection reagent release testing needed for CGT processes, so the BioPhorum approach is an industry first. With the explosive growth of the CGT industry, the need for these release specifications is loud and clear.

Readers are invited to comment on the specific standards, tests, and the overall proposed approach. Feedback can be provided by completing this form. For more information, download the paper here and contact Victoria Mwanza, Senior Global Change Facilitator, at victoria.mwanza@biophorum.com

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Advanced Therapy Medicinal Products

Advanced Therapy Medicinal Products support the quest for better and faster development of cell, gene and RNA therapies​ through connecting therapy developers, contract manufacturing, and testing organizations to gain an understanding and respond to the challenges faced by the advanced therapeutics industry.

Development Group

Development Group accelerates and improves the development process of biopharmaceutical medicines for the benefit of the patient. Replacing isolation with collaboration by providing a “safe” space where subject matter experts can work on sharing solutions and best practice on emerging industry trends, implementation of new technologies and common issues whilst ensuring their intellectual property and confidentiality is protected.

Drug Substance

Drug Substance advances excellence in drug substance manufacturing and testing, acting as an industry voice to accelerate technology adoption, implement post-approval change more rapidly and increase confidence in maintaining compliance.

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Fill Finish accelerates development and acceptance of sustainable world-class filling and packaging operations for drug product which meets the future needs of patients. Through innovative solutions resulting from the sharing of expertise, we can overcome common performance challenges and deliver on quality and regulatory compliance.

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Information Technology accelerates digital maturity across the ​global pharmaceutical manufacturing industry.

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BioPhorum Quality provides a dedicated, safe space for quality professionals from biomanufacturers and contract organizations to coalesce and enhance effective collaboration to address shared industry challenges.

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Regulatory CMC brings leaders together who have a common goal – to improve access to medicines through innovation in the Regulatory ecosystem. It provides a dedicated space for strategic leadership and a coordinated industry voice, to move from divergence to convergence.

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Supply Chain to Patient transforms the performance of global pharmaceutical clinical and commercial outbound supply chains through industry collaboration by creating transparent patient-centric, resilient and agile E2E supply chains, that consistently deliver high-quality medicines ensuring an uninterrupted supply to patients.

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