ARM identifies barriers to the manufacturing and scale up of regenerative medicines and works with its multi-stakeholder audience to develop and implement solutions.
Over the past decade, advanced therapies have transitioned from academic dream to a clinical reality. Though this field has the potential to dramatically transform the health care landscape and treat patients who have in the past had limited or no treatment options, the shifting treatment paradigm also presents development and marketing hurdles which are different from traditional therapies.
These therapies pose unique challenges for manufacturing and scale up, including shorter shelf lives, greater temperature sensitivities, and increased complexity and cost related to purity and identity testing; producing sufficient supply of starting materials, such as cell lines or viral vectors; autologous manufacturing processes for many products, which requires material from the patient to be collected and modified prior administration; and Chemistry, Manufacturing and Controls (CMC) requirements which are not adequately tailored to the specific needs of regenerative medicines or which create and duplicative processes for testing, reporting, and clinical submissions from country to country.
To ensure broad patient access to these transformative therapies in a timely manner, sector stakeholders must now convene to identify and address these challenges.
ARM is currently developing the “A-Gene” and “A-Cell” projects, intended to create a case study-based reference guide on the best practices for the development of gene therapies and cell therapies, respectively. An expert team, including representation from leading commercial and academic groups, will address key topics in the development and manufacturing of cell and gene therapies, including comparability, critical quality attributes, the product life cycle, the development and use of standards, regulatory implications, and others. The resulting white papers will help advance the principles of Quality by Design (QbD) during the manufacture of gene- and cell-based therapeutics.
ARM’s Manufacturing Workshop Series is intended to convene sector stakeholders, including ARM members, FDA and EMA officials, and others in order to discuss issues relevant to the manufacturing of regenerative medicines, including comparability, international frameworks, and starting & ancillary materials.
