This paper reviews and identifies gaps in existing guidelines on APS for ATMPs; outlines technical limitations and challenges; and provides recommendations on design and how some of the existing challenges may be addressed through risk-based approaches.
Cell & Gene Therapy
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Labelling of CGT clinical drug product (DP)
May 2023 | Benchmarking, Cell & Gene Therapy, Commercialization, Deliverable, Deliverables Report, POI - Cell and Gene Therapy
A BioPhorum member survey examining approaches taken for labelling of allogeneic CGT clinical drug product.
PAT and Rapid Analytics supporting rapid manufacture survey
May 2023 | Benchmarking, Cell & Gene Therapy, Deliverable, Deliverables Report, PAT Monitoring and Control, POI - Cell and Gene Therapy
A BioPhorum member only benchmarking survey focusing on enabling rapid manufacture.
A proposal to align release standards for endonucleases used in nucleic acid removal
May 2023 | Cell & Gene Therapy, Deliverable, Deliverables Report, POI - Cell and Gene Therapy, Publication, Supply Partner
This BioPhorum peer reviewed paper proposes to unify current industry approaches with a single core standard of test methods and criteria necessary for GMP manufacturing. A common testing standard would have multiple benefits including consistency across suppliers, protection of supplier intellectual property, and facilitation of drug development.
Standardizing traceability of personalized cell and gene therapies
Apr 2023 | Cell & Gene Therapy, Deliverable, Deliverables Report, Information Technology, IT for Cell and Gene Therapy, POI - Information Technology, Publication
This document presents a practical vision and reasoning for standardizing the traceability of CGTs across the value chain. A user-centric design perspective leads to an architecture that integrates interrelated services and a model for connecting data across the systems throughout the supply chain.  It considers the current situation and process flows, envisions a series of user stories from the perspective of several key personas, and outlines the benefits sought from a more holistic approach. Â
CGT conceptual facility design
Apr 2023 | Cell & Gene Therapy, Commercialization, Deliverable, Deliverables Report, POI - Cell and Gene Therapy, Publication
A BioPhorum member only deliverable, the BioPhorum Commercialization workstream has developed a conceptual facility design that deliberately disrupts conventions for flow, segregation and cleanroom classification.
Minimizing the impact of bioburden and sterility testing on gene therapy batch yield
Mar 2023 | Cell & Gene Therapy, Commercialization, Deliverable, Deliverables Report, POI - Cell and Gene Therapy, Publication
This paper is part of a series of papers being produced by the CGT Commercialization workstream. It will be available during Q2 2023.
Minimizing the impact of container closure integrity testing on gene therapy batch yield
Mar 2023 | Cell & Gene Therapy, Commercialization, Deliverable, Deliverables Report, POI - Cell and Gene Therapy, Publication
The scope of this paper focuses on CCIT of viral vector gene therapy drug product (DP). Cell therapies are out of scope. The strategy proposed here for CCIT is applicable to all advanced therapy medicinal product (ATMP) drug products that are manufactured in small lot sizes, typically <500 vials.
BioPhorum feedback on ICH Q5A (R2)
Mar 2023 | Cell & Gene Therapy, Deliverable, Deliverables Report, Development Group, Drug Substance, Feedback to agency, POI - Development Group, Regulatory, Regulatory Governance, Supply Partner
BioPhorum’s in-depth feedback includes 70 line-by-line, detailed comments, including the rationale for the points and any proposed changes/recommendations that are deemed necessary by the team to ensure a harmonized implementation across industry and regulatory agencies.
RNA product transfer questionnaire for sponsor and contract organization
Feb 2023 | Cell & Gene Therapy, Deliverable, Deliverables Report, Publication, RNA as a Therapeutics and a Gene-Editing Tool
This BioPhorum member only questionnaire presents a tool for sponsors to ascertain the suitability of the contract organization services and for the contract organizations to understand the scope, requirements, and timelines of the RNA transfer project.Â