The BioPhorum CGT Environmental Health and Safety (EHS) workstream offers this downloadable template for CGT institutions to use to assess and mitigate biosafety risk for human-derived cell lines susceptible to adventitious agents.
Cell & Gene Therapy
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A downloadable template for CGT institutions to use to communicate workplace hazards and assess the risks posed by CGT materials.
A commentary on the current process analytical technology status and opportunities for cell and gene therapies
This BioPhorum commentary summarizes the CGT industry views on PAT articulated during the virtual event, and highlights key takeaways on the benefits and barriers to PAT, unique considerations for PAT in the context of CGTs, and highlights some near-term options to help accelerate these important therapies to patients prior to implementing PAT.
Demonstrating comparability for clinical cell-based therapy products following manufacturing process changes: an industry perspective
This paper provides joint industry recommendations by Subject Matter Experts in the development and manufacturing of cell-based therapy products to demonstrate comparability for some of the common process changes implemented by manufacturers that may occur during the clinical development lifecycle. Changes with the potential for a significant impact to cell-based therapy products were assessed for comparability recommendations, with the focus on the pre-pivotal and pivotal phases. The resulting recommendations are intended to help manufacturers with the design and approach for comparability studies to streamline study design and execution, as well as organization of data for accelerated regulatory agency approvals.
Defining the required critical quality attributes (CQAs) and phase requirements for mRNA/LNP product development and manufacture
This article identifies not only relevant CQAs, but the stage at which their testing may occur and the category of control strategy (e.g. release, stability, etc.). A justification is provided for each CQA category and for each individual attribute. The justifications provide further information, based on industry experience, on whether these CQAs should be considered for products, as there are many different mRNA products, and each company will have their own in-depth understanding and specific characterization information to inform their quality control strategies
BioPhorum comments on proposed national emission standards for hazardous air pollutants ethylene oxide emissions standards for sterilization facilities residual risk and technology review
Jul 2023 | Cell & Gene Therapy, Deliverable, Deliverables Report, Development Group, Drug Substance, Feedback to agency, Fill Finish, POI - Supply Partner, Publication, Regulatory, Supply Chain to Patient, Supply Partner, Sustainability
This group supports the Environmental Protection Agency’s (EPA) efforts to protect public health and the environment through the Residual Risk and Technology Review (RTR) process and recognizes the
need to develop stringent emissions standards to mitigate any potential hazards posed by Ethylene Oxide (EtO) emissions. However, they are also cautious of the impact these changes may have on the availability and timely delivery of biotherapeutics to patients who rely on them. Biotherapeutics, including vaccines, therapeutics, and other life-saving medications, play a crucial role in improving patient health outcomes and saving lives. For the biopharmaceutical industry, EtO sterilization is a critical process used to ensure product safety and efficacy. The EtO Response team raised several concerns regarding the potential impact of the proposed rule changes on the biotherapeutics market. This document discusses the concerns.
This paper outlines strategies for reducing the volumes required for stability studies with the goal of conserving product for patients, while remaining compliant and delivering data on critical quality attributes across the shelf life of gene therapy products. The recommendations provided include considerations for both drug substance and drug product.
Overview of end-to-end mRNA drug substance and drug product manufacturing processes and scale-up considerations
This paper gives an overview of the mRNA/lipid nanoparticles manufacturing unit operations for drug substance/drug product. It was developed by CGT experts in the RNA workstream of BioPhorum Cell and Gene Therapy to enable future discussions and accelerate the commercialization of mRNA therapies.
This presentation, developed by the BioPhorum Validation team reviews the regulatory challenges faced in cell and gene therapy with a focus to identify and use appropriate analytical controls when dealing with both limited regulatory guidance and limited materials.
This paper evaluates the applicability of the current understanding of viral clearance to the products and processes employed in CGT manufacture to assure the viral safety of these therapies. Unique considerations for preventing contamination through raw material risk mitigation are highlighted. Guidance on viral clearance strategies for inactivation and removal are also provided. This guidance focuses on adeno-associated viral vectors but may also apply to other viral vectors.